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Development of a new patient-reported outcome measure for children with lower limb deformities : concept… Chhina, Harpreet Kaur 2021

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    DEVELOPMENT OF A NEW PATIENT-REPORTED OUTCOME MEASURE FOR CHILDREN WITH LOWER LIMB DEFORMITIES: CONCEPT ELICITATION, ITEM DEVELOPMENT AND CONTENT VALIDATION by  Harpreet Kaur Chhina  M.Sc., Punjab University, 2004 M.Sc., University of British Columbia, 2012  A THESIS SUBMITTED IN PARTIAL FULFILLMENT OF THE REQUIREMENTS FOR THE DEGREE OF  DOCTOR OF PHILOSOPHY  in  THE FACULTY of GRADUATE AND POSTDOCTORAL STUDIES  (Experimental Medicine)  THE UNIVERSITY OF BRITISH COLUMBIA (Vancouver, BC)  April 2021 ©Harpreet Kaur Chhina, 2021  ii  The following individuals certify that they have read, and recommend to the Faculty of Graduate and Postdoctoral Studies for acceptance, the dissertation entitled: Development of a Patient-Reported Outcome Measure for Children with Lower limb Deformities: Concept-elicitation, Item Development and Content Validation  submitted by Harpreet Chhina  in partial fulfillment of the requirements for the degree of Doctor of Philosophy in Experimental Medicine  Examining Committee: Anthony Cooper, Clinical Associate Professor, Department of Orthopaedics, UBC Co-supervisor Jacek Kopec, Professor, School of Population and Public Health, UBC Co-supervisor  Anne Klassen, Professor, Department of Pediatrics, McMaster University Supervisory Committee Member Dr. Stephen Tredwell, Professor Emeritus, Department of Orthopaedics, UBC University Examiner Dr. Carolyn Gotay, Professor Emeritus, School of Population and Public Health, UBC University Examiner  Additional Supervisory Committee Members: John Oliffe, Professor, School of Nursing, UBC Supervisory Committee Member   iii  Abstract  Objectives: To develop a conceptual framework, generate items and scales, and establish content validity for a new patient-reported outcome measure (PROM) for children with lower limb deformities.  Methods: A systematic review was conducted to synthesize the evidence on the use of PROMs to measure Health Related Quality of Life (HRQL) of children with lower limb deformities. A qualitative phase involved semi-structured face-to-face interviews with children with lower limb deformities and their parents from Canada, Ethiopia, India and the USA. Using the knowledge gained from these interviews, a list of items was generated and used to create individual scales for the new PROM. The scales were shown to children with lower limb deformities from Australia, Canada and the USA. Feedback was also obtained from clinical experts. The feedback was used to determine if the content of the scales was comprehensible, relevant and comprehensive (i.e., to determine content validity).   Results: The systematic review confirmed that there was no condition-specific PROM for children with lower limb deformities. Qualitative interviews allowed us to elicit concepts that matter most to children with such deformities and develop conceptual framework covering the most important concerns. A set of independently functioning scales measuring physical function, leg symptoms, hip symptoms, knee symptoms, ankle symptoms, foot symptoms, leg-related distress, appearance, school function, social function, and psychological function were  iv  developed. Items that were difficult to understand were revised. New items were added as suggested by patients and experts.   Conclusions: Lower limb deformities can substantially impact HRQL of children. To date, no condition-specific PROMs exist for this patient population. We have developed items for a set of scales, guided by qualitative interviews with the target patient population that formed the basis of a new PROM. The next phase in the development and validation of this PROM includes a pilot test to ensure the scales work as hypothesized, followed by an international field test study to examine its psychometric properties. Once fully validated, this PROM can be used to measure HRQL of children with lower limb deformities and to evaluate the effectiveness of interventions aimed at improving HRQL in these children.   v  Lay summary  Lower limb deformities in children can affect their health-related quality of life (HRQL). Our review showed that there is no questionnaire to measure how these deformities affect HRQL of children. To develop a new questionnaire, we interviewed children and their parents from Canada, Ethiopia, India and the USA to understand what matters most to these children. Using this knowledge, we wrote items (questions) for a new questionnaire. These items were shown to children with lower limb deformities from Australia, Canada and the USA to test if the items were easy to understand. We asked doctors and healthcare workers for their feedback on the items. This feedback was used to revise the items. These items are now ready for testing in a large number of children. This questionnaire will be used to understand how children feel about their legs and in studies to find the best treatments for lower limb deformities.   vi  Preface Statement of Co-authorship I composed this thesis in its entirety, with guidance and input from Drs. Anthony Cooper, Jacek Kopec, Anne Klassen and John Oliffe.   Statement of research ethics approval This thesis was conducted under ethics approval from University of British Columbia’s Behavioural Research Ethics Board (H15-00514). Separate ethics approval was also obtained at each of the participating sites from Australia, Ethiopia, Canada, India and the USA. The material presented in this thesis is the basis of three manuscripts as listed below. A fourth manuscript will be prepared based on chapters 4-5.  Chapter 2 is based on a published manuscript. The manuscript is reproduced with permission from the journal. I developed the literature search strategy, conducted the systematic review and prepared the manuscript. AK, JK and AC provided guidance to develop the search strategy, reviewed the results and the manuscript. CF was the second reviewer for the selected articles. SP helped in the preparation of the manuscript. Chhina H, Klassen A, Kopec J, Park S, Fortes C, Cooper A. Quality of Life of Children with Lower Limb Deformities: A Systematic Review of Patient-Reported Outcomes and Development of a Preliminary Conceptual Framework. Journal of Limb Lengthening and Reconstruction 2017; 3(1); 19-29.   vii  Chapter 3 is based on a manuscript that is now published in the Journal of Patient Reported Outcomes. I developed the interview guide, conducted interviews at 3 sites, trained interviewers at 2 other sites, analysed all qualitative interviews and prepared the manuscript. AK, JK, JO and AC provided guidance for each and every step of the process for this qualitative study including preparation of the manuscript. CI, ND, AA and TN contributed patients for the qualitative interviews from their respective sites, provided input for the manuscript. Chhina H, Klassen A, Kopec J, Oliffe J, Iobst C, Dahan-Oliel N, Aggarwal A, Nunn T, Cooper. A. What Matters to Children with Lower Limb Deformities: An International Qualitative Study Guiding the Development of a New Patient-Reported Outcome Measure.  Appendix E – A link to the published manuscript is attached.  Chhina H, Klassen A, Kopec J, Oliffe J, Cooper. A. International Multiphase Mixed Methods Study Protocol to Develop a Patient-Reported Outcome Instrument for Children and Adolescents with Lower Limb Deformities. BMJ Open. 2019 May 5; 9(5):e027079. doi: 10.1136/bmjopen-2018-027079.  I wrote the proposal for the development of this new PROM in consultation with the other co-authors and prepared the manuscript.   viii  Table of contents   Abstract .................................................................................................................................. iii Lay summary .......................................................................................................................... v Preface ....................................................................................................................................vi Table of contents ................................................................................................................... viii List of tables.......................................................................................................................... xiii List of figures .........................................................................................................................xv List of abbreviations .............................................................................................................. xvi Acknowledgements .............................................................................................................. xvii Dedication........................................................................................................................... xviii Chapter 1: Introduction ............................................................................................................ 1 1.1 Lower limb deformities  .................................................................................................. 1 1.2 Treatments ..................................................................................................................... 3 1.3 Patient-reported outcome measures and health-related quality of life ................................ 6 1.3.1 HRQL in children with limb deformities  .................................................................10 1.3.2 Importance of measuring PROMs in children with lower limb deformities  ...............10 1.4 Development of a new PROM for children with lower limb deformities ..........................12 1.4.1 Qualitative research for PROM development ...........................................................13 1.5 Objective  ......................................................................................................................16 1.6 Specific aims ................................................................................................................16  ix  1.7 Thesis overview ............................................................................................................16 Chapter 2: Systematic Review.................................................................................................18 2.1 Introduction ..................................................................................................................19 2.2 Methods........................................................................................................................20 2.3 Results..........................................................................................................................21 2.3.1 Physical health .......................................................................................................23 2.3.2 Psychological health ...............................................................................................24 2.3.3 Social health...........................................................................................................25 2.4 Discussion ....................................................................................................................27 2.5 Conclusion....................................................................................................................30 Chapter 3: What Matters to Children with Lower Limb Deformities: An International Qualitative Study Guiding the Development of a New Patient-Reported Outcome Measure ........................35 3.1 Introduction ..................................................................................................................35 3.2 Methods........................................................................................................................38 3.2.1 Sampling ................................................................................................................39 3.2.2 Inclusion criteria .....................................................................................................39 3.2.3 Exclusion criteria ....................................................................................................39 3.2.4 Study setting, recruitment and data collection ..........................................................39 3.2.5 Data analysis  ..........................................................................................................41 3.2.6 Rigor......................................................................................................................42 3.3 Results..........................................................................................................................43 3.3.1 Appearance ............................................................................................................44 3.3.2 Physical health .......................................................................................................45  x  3.3.3 Psychological health ...............................................................................................46 3.3.4 School ....................................................................................................................47 3.3.5 Social health...........................................................................................................49 3.3.6 Mediating factors....................................................................................................49 3.4 Discussion ....................................................................................................................50 3.5 Conclusion....................................................................................................................54 3.6 Update ..........................................................................................................................55 Chapter 4: Item Generation and Scale Formation .....................................................................56 4.1 Introduction ..................................................................................................................56 4.2 Methods........................................................................................................................56 4.2.1 Item length .............................................................................................................57 4.2.2 Item wording ..........................................................................................................57 4.2.3 Reading level .........................................................................................................58 4.2.4 Age-related considerations ......................................................................................58 4.2.5 Response options ....................................................................................................59 4.2.6 Recall period ..........................................................................................................59 4.2.7 Scale formation ......................................................................................................60 4.3 Results..........................................................................................................................61 4.3.1 Physical Function Scale ..........................................................................................61 4.3.2 Symptom scales......................................................................................................62 4.3.3 Leg-Related Distress Scale......................................................................................63 4.3.4 Appearance Scale ...................................................................................................64 4.3.5 School Function Scale  ............................................................................................65  xi  4.3.6 Psychological Function Scale ..................................................................................65 4.3.7 Social Function Scale  .............................................................................................66 4.4 Discussion ....................................................................................................................67 Chapter 5: Establishing Content Validity of LIMB-Q Kids.......................................................71 5.1 Introduction ..................................................................................................................71 5.2 Methods........................................................................................................................72 5.2.1 Sampling ................................................................................................................73 5.2.2 Recruitment............................................................................................................73 5.2.3 Interviews with children..........................................................................................73 5.2.4 Expert opinions ......................................................................................................74 5.2.5 Analysis .................................................................................................................75 5.3 Results..........................................................................................................................76 5.3.1 Summary of cognitive debriefing interviews and expert feedback.............................77 5.3.2 Response options ....................................................................................................78 5.3.3 Recall period ..........................................................................................................79 5.3.4 Physical Function Scale  ..........................................................................................79 5.3.5 Leg Symptoms Scale ..............................................................................................82 5.3.6 Hip Symptoms Scale...............................................................................................84 5.3.7 Knee Symptoms Scale  ............................................................................................85 5.3.8 Ankle Symptoms Scale  ...........................................................................................87 5.3.9 Foot Symptoms Scale .............................................................................................87 5.3.10 Leg-Related Distress Scale ....................................................................................88 5.3.11 Appearance Scale .................................................................................................90  xii  5.3.12 School Function Scale...........................................................................................91 5.3.13 Social Function Scale ............................................................................................92 5.3.14 Psychological Function Scale  ................................................................................92 5.3.15 General expert feedback........................................................................................93 5.3.16 Readability ...........................................................................................................95 5.4 Discussion ....................................................................................................................95 Chapter 6: Challenges and Limitations  ....................................................................................99 6.1 Challenges ....................................................................................................................99 6.2 Limitations ................................................................................................................. 100 Chapter 7: Significance, Implications and Future Directions  .................................................. 103 Tables and figures ................................................................................................................ 109 Bibliography ........................................................................................................................ 156 Appendix A: Supplementary material for Chapter 2 ............................................................... 180 Appendix B: Supplementary material for Chapter 3 ................................................................ 196 Appendix C: Supplementary material for Chapter 4 ............................................................... 207 Appendix D: Supplementary material for Chapter 5 ................................................................ 219 Appendix E: A link to the protocol manuscript ...................................................................... 237     xiii  List of tables  Table 1: Characteristics of the studies included in the systematic review  ................................ 110 Table 2: Patient- and proxy-reported outcome measures from studies included in the systematic review .......................................................................................................................... 112 Table 3: Determinants of quality of life from studies included in the systematic review .......... 114 Table 4: Demographic characteristics of patients participating in qualitative interviews .......... 115 Table 5: Lower limb deformities of patients participating in qualitative interviews  ................. 116 Table 6: Participant quotes from qualitative interviews .......................................................... 117 Table 7: Demographics and clinical characteristics of patients participating in cognitive debriefing interviews..................................................................................................... 129 Table 8: Characteristics of experts who provided feedback..................................................... 130 Table 9: Sequence of cognitive debriefing interviews and expert feedback ............................. 131 Table 10: Number of items retained, revised, dropped and added during each round ............... 132 Table 11: Evidence used to retain, revise, add, and drop items for the Physical Function Scale  136 Table 12: Evidence used to retain, revise, add, and drop items for the Leg Symptoms Scale  .... 139 Table 13: Evidence used to retain, revise, add, and drop items for the Hip Symptoms Scale  .... 141 Table 14: Evidence used to retain, revise, add, and drop items for the Knee Symptoms Scale  .. 143 Table 15: Evidence used to retain, revise, add, and drop items for the Ankle Symptoms Scale . 145 Table 16: Evidence used to retain, revise, add, and drop items for the Foot Symptoms Scale  ... 146 Table 17: Evidence used to retain, revise, add, and drop items for the Leg-Related Distress Scale..................................................................................................................................... 147 Table 18: Evidence used to retain, revise, add, and drop items for the Appearance Scale  ......... 149  xiv  Table 19: Flesch-Kincaid grade reading level of final version of items included in LIMB-Q Kids..................................................................................................................................... 151    xv  List of figures Figure 1: Multiphase mixed methods protocol for development of PRO instruments  ............... 152 Figure 2: PRISMA flow diagram........................................................................................... 153 Figure 3: Preliminary conceptual framework developed as part of the systematic review......... 154 Figure 4: Final conceptual framework developed as part of the qualitative study..................... 155     xvi  List of abbreviations  CAPP-PSI – Childhood Amputee Prosthetics Project-Prosthetics Satisfaction Inventory CAPP-FSI – Childhood Amputee Prosthetics Project-Functional Status Inventory  CFD – Congenital Short Femur COI – Concept of Interest  COSMIN – COnsensus-based Standards for the selection of health Measurement INstruments FDA – Food and Drug Administration HRQL – Health-Related Quality of Life  ISOQOL – International Society for Quality of Life Research ISPOR – International Society of Pharmacoeconomics and Outcomes Research LLSQ – Limb Lengthening Satisfaction Questionnaire  PFFD – Proximal Focal Femoral Deficiency PROM – Patient-Reported Outcome Measure QOL – Quality of Life  SR – Systematic Review  TCA – Translation and Cultural Adaptation   xvii  Acknowledgements  First and foremost, I offer my sincere gratitude to my supervisors and mentors Drs. Anthony Cooper, Jacek Kopec and Anne Klassen for their support and guidance throughout the course of my Doctoral degree. Throughout my PhD, Dr. Cooper has given me his unwavering support and encouragement to pursue research ideas with independence. Dr. Kopec through his discussion sessions on methodology made it an enriched learning experience. Dr. Klassen has inspired me to produce high quality, meaningful work to further the field of patient-reported outcomes and help better the lives of patients. As an expert in PROM development, Dr. Klassen has provided valuable guidance at each and every step of the process. Dr. John Oliffe’s insights for the qualitative phase and his ongoing advice for my thesis and manuscripts were invaluable.   My warmest gratitude goes to colleagues from the department of Orthopaedics at BC Children’s Hospital and the Q-Portfolio team from McMaster University, who have supported me through this long journey.  My sincere thanks to children with lower limb deformities and their families who trusted me by sharing their personal experiences and providing me with this vast knowledge to develop the new patient-reported outcome measure.  This research was made possible by financial support from Department of Orthopaedics at UBC, Canadian Orthopaedic Foundation and BC Children’s Hospital Foundation. xviii Dedication This thesis would not have been possible without the support of my husband Navdeep Chhina. His untiring support gave me strength to keep going during challenging times. A special thanks to my children Malvina and Mirza for being very patient and understanding during mommy’s school years. I would like to acknowledge two very special people in my life, who are my greatest motivation, strength and a continuous source of upliftment, my mama and papa.    Chapter 1: Introduction 1.1 Lower limb deformities Lower limb deformities describe a range of conditions that may result from congenital defects in fetal development, or are acquired during growth [1]. They may also arise from trauma, infection, benign or malignant tumors, or other medical conditions. Lower limb deformities primarily include leg length discrepancy, lower limb deficiency, and associated angular and rotational deformities. Leg length discrepancy (anisomelia, inequality between paired limbs) can be congenital or acquired. Congenital leg length discrepancy can arise from aplasia (defective development or congenital absence of a limb), hyperplasia (an increase in the amount of organic tissue that results from cell proliferation), hypoplasia (underdevelopment or incomplete development of a tissue or organ), hip dysplasia (congenital or developmental deformation or misalignment of the hip) and clubfoot (a congenital deformity of foot where the foot is internally rotated at ankle). Causes of acquired leg length discrepancy could be neurogenic (paralysis, disuse, sympathectomy [the surgical cutting of a sympathetic nerve or removal of a ganglion to relieve a condition affected by its stimulation]), vascular (ischemia, Perthes disease), infection, tumours (physeal involvement, vascular lesions), trauma (physeal injury, fracture stimulation distraction) and disturbance of growth plates [2]. Lower limb deficiencies are congenital and can involve complete or partial absence of one or both of the limbs or a segment of the limb. Tibial deficiency or hemimelia involves a shortened leg along with potentially severe knee and ankle deformities. Fibular deficiency or fibular hemimelia is partial or complete absence of the fibula and is associated with foot and knee deformities along with a leg length discrepancy [2].  2 Congenital femoral deficiency (CFD) is a spectrum of disorders ranging from shortening of the femur (congenital short femur) to complete absence of proximal two-thirds of the femur (proximal focal femoral deficiency [PFFD]) and is often associated with hip and knee deformities [2]. However, the terms CFD, congenital short femur and PFFD are often used interchangeably.  Lower limb amputation could be congenital or acquired. Congenital lower limb amputation is characterized by complete absence of the lower limb at birth, which could be due to intrauterine growth inhibition or disruption due to various reasons, such as exposure to teratogenic agents or amniotic band syndrome [3,4]. Acquired amputations may result from cancer treatment, trauma or infection. An amputation can also be performed for a congenital limb deformity when reconstruction cannot be performed.   Leg length discrepancy can cause angular and torsional deformities as well as soft tissue contractures on the same side or contralateral unaffected lower limb [5]. Leg length discrepancy is also associated with abnormal gait and can cause degenerative arthritis of the lower limb and the lumbar spine [6,7]. Lower limb deformities may vary considerably in etiology, severity and integrity of the surrounding soft tissue [8]. Leg length discrepancy of greater than 2 cm was found in at least 1 in every 1000 people in a retrospective study from France [9]. The incidence of lower limb reduction defects is estimated to be 2 per 10000 live births in the USA [10]. Tibial hemimelia also known as tibial deficiency is a congenital condition where a child is born with a shorter tibia or the tibia is missing all together. The reported incidence of tibial hemimelia is 1 per million live births [11]. Fibular hemimelia is a congenital deficiency of fibula, often associated with leg  3 length discrepancy, foot and knee deformities. It has an estimated incidence between 7.4 and 20 per 1 million live births [12–15]. The incidence of congenital femoral deficiency is 1 in 52,029 [15]. The case rate of deficiency defects in Canada (excluding Quebec) was 3.7 per 10,000 births as reported in 2014 [16]. There is no up-to-date data in the literature on the incidence of these conditions.  1.2 Treatments  Depending on the type of lower limb deformity, treatment options vary from different non-surgical options to complex surgical procedures. Non-surgical treatments may include the use of adaptive devices, prostheses, orthotics or shoe lifts. Surgical approaches may include reconstructive or ablative (amputation) surgery combined with the use of prostheses and orthotics. Distraction osteogenesis principles, which involve surgically moving two segments of a bone slowly apart to allow new bone formation in between to fill the gap, are applied to treat a vast majority of limb deformities. Surgical treatment for lengthening or deformity correction often involves a long, arduous phase during which the leg is in an external fixator device for 6 to 9 months. The process of limb lengthening using an external fixator device has a high complication rate [17]. Common complications include pin site infections, premature consolidation, delayed consolidation, fracture, joint dislocation and contracture formation [18]. The incidence of pin site infections, associated with the use of external fixators has been reported between 11 and 100% [19–24]. Having an external fixator to lengthen a limb has also been found to be psychologically stressful for patients because of its long duration and potential complications [17,25–27].  4 More recently, limb lengthening has also been performed using internal fixation where a lengthening device is inserted inside the bone. This device has a magnet inside it, which is activated by an external remote control device. Using the remote control, the internal lengthening device is lengthened, causing the bone to also lengthen slowly. This technique has some important advantages over external fixation (e.g., no pin site infections, reduced risk of soft tissue tethering, vascular and nerve injuries, axial deviations and joint stiffness due to external fixator devices) [28]. However, internal lengthening cannot be applied to all patients due to clinical indications such as skeletal immaturity of patients with an open physis, small size of the bone canal to accommodate the reaming of the nail, or metabolic bone diseases that prevent adequate locking of the screws [29].  Amputation is a non-reversible surgical procedure and is followed by fitting of prosthesis. In general, leg length discrepancies in one bone larger than 30 percent are considered too large for reconstruction by lengthening of bone and soft tissues, especially when combined with significant joint abnormalities [30]. In cases where limb salvage procedures are not applicable due to specific clinical factors, amputation has an important role to play in creating high functional performance, optimal participation and improved health related quality of life (HRQL) [31]. However, amputation comes with specific problems, such as potential stump overgrowth and pain. The prosthetic limbs require periodic replacement due to patient growth, breakage or fitting problems and lack normal sensation and proprioception [32]. The life-time cost of prosthesis replacement can be substantially higher than the repeat surgical procedures for limb lengthening [33,34].    5 Each of the treatments described above has advantages and disadvantages. At present there is insufficient evidence to support one treatment over the other. Successful outcomes with amputation has been reported in some studies [32,35–38] while other studies have reported successful outcomes with lengthening and reconstruction [39–43]. Comparative effectiveness studies have found no difference when amputation and reconstruction were compared [44–46]. A recent meta-analysis published in 2020 indicates better patient satisfaction, fewer surgical complications, and a smaller number of procedures with amputation for fibular hemimelia as compared to limb reconstruction [47]. Limitations pointed out by the authors include that the meta-analysis was based on only 7 studies and 6 were conducted prior to 2009. With the advancement in reconstructive procedures, the rate of associated complications might be reduced. Patient satisfaction was measured using different questionnaires. The most recent study published in 2019 shows comparable results for amputation versus limb reconstruction using procedures such as the SUPERankle [45].   Given the current advances in reconstructive procedures, the prospective studies or reviews of more recent procedures might show less surgical complications with favourable trends towards reconstructive procedures. However, it will be important for future comparative studies of amputation versus limb reconstruction using more sophisticated reconstructive procedures to measure patient-reported outcomes (PROs) using condition-specific patient-reported outcome measures (PROMs).    6 The lack of evidence demonstrating superiority of one treatment option over the other combined with the impact of each type of treatment on HRQL limits the ability of healthcare providers to counsel families on the best evidence-based treatment option for them.  1.3 Patient-reported outcome measures and health-related quality of life  A PRO is a direct report by the patient, about his or her health, without interpretation of the patient’s response by a clinician or anyone else [48]. PROs can include quality of life (QOL), HRQL and/or functional status associated with health care or treatment [49]. The questionnaires or instruments used to measure PROs are called patient-reported outcome measures (PROMs). With the increased emphasis on patient-centred care, PROMs are increasingly used in clinical practice to inform patient care and in research studies.   QOL and HRQL are among the most frequently measured PROs in healthcare along with the functional outcomes. QOL as defined by the World Health Organisation (WHO) is “an individuals’ perception of their position in life in the context of the culture and value systems in which they live and in relation to their goals, expectations, standards and concerns” [50]. QOL is affected by a person's physical health, psychological state, level of independence, social relationships, personal beliefs and their relationship to salient features of their environment. The terms QOL and HRQL are often used interchangeably in the literature [51,52]. While QOL is an all-inclusive concept incorporating all factors that influence an individual’s life, HRQL includes those concepts that are related to an individual’s health [52,53]. Aspects of QOL such as economic and political factors are usually not considered part of HRQL [53]. For the purpose of this study, the term HRQL will be used (except in chapter 2 which was published before I made this distinction).  7 There are many different conceptual models of HRQL used in research. A systematic review by Bakas et al. found that the most commonly used HRQL models were the World Health Organisation’s International Classification of Functioning, Disability and Health (WHO ICF), Wilson and Cleary’s model of HRQL, and Ferrans and colleagues’ modification of Wilson and Cleary’s model [54–57]. The authors of this review recommended using one of these three models to facilitate future research [54]. The WHO ICF model represents an integration of medical and social models. It has been modified further for children and youth in the WHO ICF-CY (child and youth) model. However, it is a mapping and classification framework rather than a causal model. It may have applications for needs assessments, matching treatments with conditions and evaluating outcomes [55,58].   The Wilson and Cleary model and Ferrans et al’s revised Wilson and Cleary model are the most frequently referenced models in HRQL literature. The revised Wilson and Cleary model provides clear conceptual and operational definitions and clarifies the relationships among different HRQL concepts to guide research and practice. The review by Bakas et al. specifically recommends the use of the model proposed by Ferrans et al. [54]  The original version of the Wilson and Cleary model published in 1995 was a causal model developed to guide the design of interventions/treatments to improve patient outcomes [56]. This model provides an explanation for the relationships between traditional clinical measures and health status measures. The five core concepts in this model include biological and physiological factors, symptoms, functioning, general health perceptions, and overall quality of life. These  8 concepts form a causal path from the cellular level (biological and physiological variables) to the individual level to the interactions between the individual and society.   Symptoms are defined as a patient’s perception of an abnormal physical, emotional or cognitive state [56]. The relationship between the biological and physiological factors and symptoms is complex and often inconsistent. Some biological and physiological variables can be abnormal without causing any symptoms while for some conditions such as depression, there are no biological or physiological abnormalities but there can be a range of symptoms. Due to this inconsistent relationship, other determinants of symptoms, such as psychological factors, patient expectations and social factors need to be considered. Functioning is usually assessed by an individual’s ability to perform a particular task. The four domains of functioning that are commonly measured are physical function, social function, role function and psychological function. Symptom status is an important determinant of functioning. Though there is an evidence for the correlation between symptoms, biological and physiological variables and functioning, the variation in functioning cannot always be explained by this correlation. However, according to the Wilson and Cleary model, comprehensive and disease specific measures of symptoms can be predictors of function and the effect of biological and physiological factors on functioning can be explained by the mediating role of symptoms. Functional status has been shown to be associated with the perceptions of general health. However, general health perceptions have also been associated with biological and physiological factors and hence there will be variation in general health perceptions within each stratum of clinical severity. Patient’s preferences and values play an important role at various levels within the Wilson and Cleary model, specifically in relation to general health perceptions and overall  9 quality of life. The emotional and psychological factors are related bi-directionally to each other and to many other factors in the model. This model was further revised by Ferrans et al. to indicate that biological function is influenced by characteristics of the individual and the environment, delete non-medical factors, and delete labels on arrows that restrict characterizations of the relationships [57].   There are two main types of PROMs that measure HRQL, generic and condition-specific [59]. The generic questionnaires, such as Short Form Health Survey (SF-36), Pediatric Quality of Life Inventory (PedsQL) and Child Health Questionnaire (CHQ) measure common HRQL concerns that apply to any condition or disease as well as to healthy individuals [60–62]. Since generic questionnaires are meant to cover any health condition and can be used to compare patient groups with each other and with the general population, they often fail to ask about issues that matter most to patients with specific health conditions. As a result, generic questionnaires tend to be less sensitive to detecting clinical changes as a result of an intervention [59]. Condition-specific instruments are designed for a particular group of patients that may have unique problems. Such instruments tend to have higher content validity than generic instruments and are thus considered more appropriate for measuring specific concerns of the target patient group. Such tools are especially useful in the measurement of change in a patient’s HRQL after an intervention.     10  1.3.1 HRQL in children with limb deformities  Participation in day-to-day activities, as well as recreational and leisure activities, is considered an important part of normal development and affects health and well-being of all children and adolescents [63–65]. Participation is defined by the International Classification of Functioning, Disability and Health (ICF) as ‘involvement in a life situation’ [58]. Children with limb deficiencies are at risk of reduced participation in social and leisure activities compared to their peers due to the physical limitations caused by their limb deformity [3,66–68]. Such children experience limitations with mobility, difficulties in normal development and the need for assistance with daily activities [69]. Children with limb deformities are also at increased risk of behavioral, emotional, psychological and social adjustment problems [69]. The range of activities in which children and youth participate outside mandated school activities includes artistic, creative, cultural, active physical, sports, play, social, skill-based, and work activities [70]. For children, participation in a wide range of activities is important and has a positive influence on the development of skills and competences, social relationships, and long-term mental and physical health [63–65,71]. The problems listed above, along with the complex treatment procedures that children with lower limb deformities undergo, can have a considerable effect on their HRQL [25,72].  1.3.2 Importance of measuring PROMs in children with lower limb deformities  Measuring outcomes of routine clinical care to evaluate treatment has a long history dating back to Florence Nightingale, who was one of the first clinicians to insist on measuring outcomes of  11 clinical care [73]. PROMs measuring HRQL are now one of the most commonly used approaches to measuring outcomes of treatment in clinical care. HRQL measurement has not only been reported to be useful to physicians in clinical decision making but has also been useful in identifying physical and psychosocial concerns from the parent and child’s perspective, which could otherwise remain undetected [74,75]. HRQL assessment can also facilitate shared decision making by identifying what matters most to patients and by working together with patients to achieve treatment goals. Identifying and prioritising problems from a patient’s perspective can be particularly useful for patients with multiple health problems [76]. Measurement of HRQL concepts, such as mental health, psychosocial health, well-being, pain and vitality has been shown to improve patient satisfaction with medical care in adult patient populations , since it helps identify other important underlying issues [75,77,78]. Using HRQL measurement in a pediatric healthcare setting can also improve parent satisfaction with their child’s medical care and facilitate patient-physician communication [75].  To measure HRQL in children with lower limb deformities, a HRQL measurement tool that measures concepts of interest (COI) identified by the children themselves is needed. Historically, children have been considered to be unreliable respondents for their own HRQL due to a perceived lack of skills (reading, linguistic, and cognitive) needed to provide accurate responses to HRQL measures [79]. This view has been challenged in the literature, and more recent research findings provide evidence that children can be active and contributing partners in their healthcare [80–82]. By 8 years of age, children are able to self-report on their well-being, psychosocial health, and health-promoting behaviors [83–85]. Several researchers have demonstrated that children with  12 chronic illnesses and disabilities were able to describe their experiences in critical and rational ways [86–88].   1.4 Development of a new PROM for children with lower limb deformities To our knowledge, there is currently no validated and psychometrically tested PROM to assess HRQL of children aged 8 years and older with lower limb deformities from their perspective. Hence, there is a need for a rigorously designed, condition-specific PROM for children and adolescents with lower limb deformities. With such a PROM, data collected could be used to inform parents and children about outcomes (physical, social, psychological) associated with different procedures, such as amputation or reconstruction. This information could supplement other objective outcome information (e.g., complication rates, expected appearance, etc.) to help families to come to a more informed decision on a child’s course of treatment [89]. The current study fills this gap by developing a new PROM for children with lower limb deformities.  According to international recommendations, the PROM development process consists of three phases, as briefly outlined below [90] (Figure 1, Page 152). The various phases involve an interactive mixed methods approach with an exploratory sequential design ‘QUAL-quant’ where the qualitative phase is followed by a quantitative phase [91]. As a PhD candidate, I designed the protocol for the development of this new PROM for children with lower limb deformities. However, for my PhD I focused on the completion of Phase 1 only. This phase included the following three steps, aligned with the specific aims of my thesis. Step 1: Development of a conceptual framework of HRQL in children with lower limb deformities. This step involved a) a systematic literature review; and b) qualitative interviews with children with lower limb deformities and their parents.  13 Step 2: Item generation and scale formation for concepts included in the conceptual framework.  Step 3:  Refining the scales through feedback on the new PROM from children with lower limb deformities and clinical and methodological experts.   Phase 2 will involve conducting an international field-test in order to perform item reduction, examine internal consistency reliability and construct validity and develop the scoring algorithms for each scale. Phase 3 will involve further psychometric evaluation of the final item-reduced PROM in a longitudinal study, including test-retest reliability and assessment of responsiveness. A significant portion of Phase 1 was based on qualitative research with children with lower limb deformities. In the development of the PROM, I followed the recommended international criteria [81,82,92–95]. However, developing PROMs in children is more complex than in adults and requires considerable attention to be paid towards the developmental stage of the target population [96,97].  1.4.1 Qualitative research for PROM development Qualitative research has long been considered the crucial foundation in the development of PROMs [98]. However, conducting qualitative research with children can be particularly challenging. Children of the same age can have varying degrees of motor, cognitive and linguistic abilities [99,100]. The ability to screen out irrelevant information and learn new memory strategies are some skills relevant to concept elicitation and cognitive debriefing interviews [96]. Children are required to use these skills to elicit their experiences during the specified recall period for a PROM.    14 Qualitative research plays an important role in the development of a conceptual framework of COI to be included in a PROM, as recommended by the U.S. Food and Drug Administration (FDA) [48]. Qualitative research involving the target respondent population for initial concept elicitation and feedback on a PROM’s items helps ensure content validity of a PROM. Content validity is the empiric evidence that demonstrates that the items and scales of an    instrument are appropriate and comprehensive relative to its intended measurement concept, population, and use [93,101]. The FDA and the equivalent body in Europe (i.e., European Medicines Agency) have both highlighted the importance of establishing content validity for PROMs [48,96]. In fact, the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) states that content validity is the most important psychometric property amongst various measurement properties of a PROM including reliability, validity and responsiveness[101]. Reliability is the degree to which the scores from a PROM are free from measurement error. Measurement error is the systematic and random error of a score that is not due to the true change in the construct being measured. Internal consistency reliability is the degree to which the items of a PROM are consistent with each other. Test-retest reliability is the degree to which person’s responses to items for a measurement instrument are consistent between two separate administrations of the same instrument provided that the construct being measured remains unchanged between the two time intervals. Validity of a measurement instrument is the degree to which it measures the constructs it claims to measure. Construct validity refers to the extent to which the scores of a PROM coincide with existing ideas or hypotheses in relation to the constructs being measured by the PROM. Responsiveness, also known as sensitivity to changes, is the ability of a PROM to measure small changes in the construct being measured that are clinically important [101].  15 The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the COSMIN initiatives provided guidance for establishing content validity of PROMs [94,95,102]. Content validity, as defined by the COSMIN group, is the degree to which the content of an instrument is an adequate reflection of the construct to be measured [101]. Three key aspects of content validity for any PROM are comprehensiveness, relevance and comprehensibility. A lack of content validity can affect other measurement properties of a PROM. Items that are irrelevant to the concept measured can decrease the internal consistency, structural validity and interpretability of PROMs [101]. Missing concepts may decrease both validity and responsiveness. High test-retest reliability and responsiveness do not automatically imply that all items are relevant and no important COI are missing. A measurement instrument can be reliable, but that does not imply that it is also valid. In other words, an instrument can reliably measure a construct but that does not mean that it is necessarily measuring the right construct. The actual COI may be over or underestimated due to having items that are not relevant or as a result of missing concepts. Asking irrelevant questions or not asking the questions that are most important to the target population, can lead to frustration and cause fatigue for the respondents, which can potentially lead to biased responses or a low response rate [103]. These problems can potentially happen more often if the respondents are children due to their young age and a shorter attention span as compared to adults. Hence the purpose of my thesis was to perform careful qualitative work to design a PROM that has high content validity for children with lower limb conditions. In order to ensure content validity, my thesis involved eliciting concepts from a heterogeneous patient population recruited through an international multicentre study.   16 1.5 Objective  To develop a conceptual framework, generate items and scales and establish content validity for a new PROM for children with lower limb deformities. 1.6 Specific aims 1. To develop a conceptual framework for HRQL in children with lower limb deformities 2. To identify concepts of interest important to children with lower limb deformities  3. To generate items for each concept measured and form scales for the new PROM  4. To refine the items and scales through feedback from children with lower limb deformities, clinicians and experts in the field of HRQL and PROMs  1.7 Thesis overview  The steps and methodology to achieve each of the specific aims are briefly described below. Methodology for Aim 1: In order to develop a conceptual framework of HRQL in children with lower limb deformities, I first conducted a systematic review of the literature (Chapter 2) to synthesize the knowledge on the use of PROMs to measure HRQL in children with lower limb deformities. The systematic review confirmed that a condition-specific PROM for children with lower limb deformities had not been developed. A preliminary conceptual framework was generated based on this systematic review. This framework guided the development of an interview guide for the qualitative phase. Based on the qualitative phase, a final conceptual framework was developed (Chapter 3).   17 Methodology for Aim 2: An international qualitative study was conducted with children with lower limb deformities and their parents from five sites in Canada, Ethiopia, India and the USA. This study was used to elicit concepts of interest that matter the most to these children.   Methodology for Aim 3: Based on the concepts elicited in the qualitative analysis, an exhaustive pool of items was generated and used to form the scales for the new PROM (Chapter 4).  Methodology for Aim 4: Cognitive debriefing interviews were conducted with children with lower limb deformities from Australia, Canada and the USA (Chapter 5) who reviewed the scales and provided feedback. Feedback was also obtained from clinicians from the participating sites and from experts in PROM development and HRQL.   18 Chapter 2: Systematic Review  Chhina H, Klassen A, Kopec J, Park S, Fortes C, Cooper. A. Quality of life of children with lower limb deformities: A systematic review of patient-reported outcomes and development of a preliminary conceptual framework. Journal of Limb Lengthening and Reconstruction 2017; 3(1); 19-29. This chapter has been published in the Journal of Limb Lengthening and Reconstruction and is reproduced here with permission from the journal. Please note that this manuscript discusses the progress of the project at the time of this publication in 2017.     19 2.1 Introduction  Lower limb deformities describe a range of conditions that may be congenital or acquired. Congenital lower limb deformities include limb reduction defects, angular deformities, and rotational deformities. Acquired lower limb deformities may result from trauma or infection resulting in mal-unions, non-union, or bone defects. Depending on the type of deformity, treatment options may include reconstructive or ablative surgery combined with the use of prosthetics and orthotics. Distraction osteogenesis principles for limb lengthening are increasingly being applied to treat lower limb deformities [104–111]. Lower limb deformities often cause difficulties in physical functioning; leading to discomfort, pain, and gait disturbance [26,110]. Dissatisfaction with appearance and/or function, along with the complex surgical procedures, can have impacts on quality of life (QOL) [25,105,112].  It has been well-recognized that the focus of any treatment should not only be on physical health but also on mental health and social well-being [113] thus, clinical outcomes need to be complemented by patient-centered outcomes to evaluate interventions [75,114]. Having a patient-reported outcome (PRO) instrument to measure QOL in children with lower limb deformities is crucial for understanding the extent to which their condition affects their QOL, as well as evaluating the success of treatment and tailoring treatment plans to address patient concerns, preferences, and needs [115–122]. The aims for this systematic review were to identify the following: (a) QOL concepts studied in existing literature specific to children with lower limb deformities; (b) PRO instruments used to measure QOL in children with lower limb deformities; and (c) determinants of QOL in children with lower limb deformities as studied in the existing literature.  20 2.2 Methods We conducted our review under the guidance of a medical librarian and followed the PRISMA statement [123]. MEDLINE, EMBASE, CINAHL, and PsycINFO databases were searched from inception of each to January 2016 (full search strategy found in Appendix A, Page 180).  The following inclusion criteria were used: (1) study population included patients with lower limb deformities; (2) mean age for participants was 18 years or under; and (3) study used a PRO or parent-reported outcome instrument to measure one or more aspects of QOL, including any ad hoc PRO instruments. For the purpose of this study, lower limb deformities included lower limb reduction defects, lower limb length discrepancy, and associated angular and rotational deformities. Isolated knee, foot and ankle conditions without any limb length discrepancy or limb deformity were not classified as lower limb deformity for this study. Exclusion criteria were as follows: (1) studies where children with other health conditions were included in the sample, and the results for the lower limb deformities sample were not presented separately; (2) studies that included conditions that could likely affect the lower limb (such as osteogenesis imperfecta, skeletal dysplasia, bone tumors, and multiple exostoses), but results for lower limbs were not presented separately; (3) mean age for the sample was over 18 years; and (4) publication was a review paper, an abstract or a letter to editor.  Titles and abstracts were independently screened by two reviewers (HC, CF). A third reviewer (AC) was consulted to reach consensus when required. Two reviewers independently inspected the full text of the articles that had been deemed potentially applicable and extracted data from those that met the inclusion criteria. References of included publications were inspected for  21 additional papers. No quality assessment was performed since our goal was to identify PRO instruments used and health concepts measured for children with lower limb deformities.  An original copy of each instrument was obtained. Each instrument was examined to generate a list of health concepts and sub-concepts measured. This list was compared with the health concepts and sub-concepts as measured by the publication included in our systematic review. Similar sub-concepts were categorized into one overarching sub-concept. For example, physical activities, walking, recreational activities, transfer and basic mobility, stiffness, and sports were grouped under sub-concept physical function (PF). These sub-concepts were further categorized into 3 major health concepts (physical, psychological, and social health). In the aforementioned example, PF was classified under the concept physical health along with pain and general health (GH).  We also collected the following data on determinants of QOL from the studies included in this systematic review: name of the determinant, direction of its relationship with the health sub-concept, and whether this relationship was statistically significant (i.e., p < 0.05). However, we report only on the determinants that were reported to have a statistically significant relationship with QOL sub-concepts in that study.  2.3 Results Our search identified 938 publications [Figure 2, Page 153]. Five publications met our inclusion criteria. A citation review added 5 additional publications, for a total of 10 publications [Table 1, Pages 110-111] [26,27,66,72,105,112,124,125]. QOL was measured using 30 parent-reported outcome or  22 PRO instruments (Appendix A, Page 195). Three instruments [Children’s Depression Inventory (CDI), Pediatric Quality of Life (PedsQL), and State Trait Anxiety Inventory (STAI)] were used as more than one version (i.e., short/long, different age groups, general/specific diagnoses). For example, two versions (regular and short form) of CDI were used, two versions of PedsQL (core and neuromuscular module) were used, and the STAI was used in 2 papers as Form C and Form Y. Consolidating multiple versions left 27 instruments, of which 1 was an interview, [26] and 2 were non-validated questionnaires [27,105]. Thus, considering only distinct, validated questionnaires, QOL was measured using 24 different instruments [Table 2, Pages 112-113]. Out of the 24 instruments, 14 were patient-reported, 7 were parent-reported, and 3 were both patient- and parent-reported instruments. Two studies used only patient reports [26,126]. Six studies used both patient- and parent proxy-reports [27,66,72,105,112,127] while 2 studies used parent reports only [125,127].  Of these 24 instruments, only 2, AAOS Lower Limb Outcomes Questionnaire [128] and Lower Limb Questionnaire [124] were lower limb specific. However, the Lower Limb Questionnaire was not specific to the pediatric population, and it was only parent-reported. Four instruments [PedsQL, Child Health Questionnaire Parent Form (CHQ PF-28), Pediatric Outcomes Data Collection Instrument (PODCI), and Kidscreen] measured multiple aspects of health. We found that most studies included in our systematic review used the same concepts and sub-concepts as the original instrument used in a particular study with slight modifications in language. The concepts and sub-concepts from the instruments identified were used to develop a preliminary conceptual framework [Figure 3, Page 154]. Three health concepts were identified, physical, psychological, and social health, with 15 sub-concepts.  23 Five studies examined the determinants of QOL for children with lower limb deformities [Table 3, Page 114] [27,72,124–126]. Five determinants that were reported to significantly affect QOL were type of deformity, severity of deformity, complications post-surgery, stage of treatment, and type of treatment.  2.3.1 Physical health Physical health was measured by 10 instruments in 6/10 studies using 3 sub-concepts [66,72,124–127] including PF, GH, and pain. The most frequently measured sub-concept was PF, measured in 6/6 studies, using 9 instruments. GH was measured in 3/6 studies, using 4 instruments [125–127]. Three studies measured pain using 5 instruments [124–126]. PF in children with lower limb deformities was found to be lower than the normative population in 4 out of 6 studies [66,72,125,127].  Determinants of physical health reported in 3 studies [72,124,126] were type of deformity, type of treatment, and stage of treatment. Lee et al. looked at the parental perspectives of their child's limb length discrepancy and found that children with idiopathic leg length discrepancy (LLD) experienced less pain and better global functioning as compared to children with posttraumatic LLD [124]. The authors defined idiopathic limb length discrepancy as limb length discrepancy without identifiable causes (i.e., not infection, tumor, vascular diseases, congenital anomaly, Legg–Calve–Perthes disease, and neurofibromatosis). PF was found to be more affected in the surgery group in comparison with the non-surgery group for children undergoing limb lengthening surgery for achondroplasia [126]. In a study of 52 patients who underwent external fixator treatment for their lower limb deformities, Montpetit et al. found that PF was better  24 preoperatively compared to mid-distraction, whereas at 3 months after removal of an external fixator device, PF was better than at mid-distraction [72].  2.3.2 Psychological health All 10 studies included in this systematic review measured psychological health using 21 instruments measuring 8 sub-concepts, making it the most frequently studied concept. These sub-concepts were emotions, psychological distress, cognition, satisfaction with appearance, self-esteem, treatment expectations, coping, and behavior. Psychological distress was the most frequently studied sub-concept, measured in 8/10 studies using 11 instruments  [26,27,66,105,112,125–127]. Emotions were measured in 4 studies using 4 instruments [66,72,124,126]. Cognition was measured in 4 studies using 5 instruments[66,72,126,127]. Satisfaction with appearance was measured in 2 studies using 2 instruments [26,124]. Behavior was measured in 4 studies using 5 instruments [105,112,125,127]. Self-esteem was measured in 6 studies using 6 instruments [66,112,124–127]. Coping and treatment expectations were measured in 1 study each using Kidcope [26] and PODCI [124] respectively.  Three studies reported that psychological distress levels were normal in children and adolescents with lower limb deformities both pre-[27,112] and post-operatively [105,112]. Another study found psychological distress preoperatively to be higher in children undergoing leg lengthening surgery when compared to children undergoing cosmetic ear surgery as well as a control group of school children [127]. More aggressive behavior and thought-related problems were reported in children undergoing limb lengthening for LLD compared to children in the control group using the Child Behavior Checklist [127]. Patients with LLD were also found to have lower self-esteem and more  25 psychological distress preoperatively compared to a control group of school children and more emotional and cognition issues [72] compared to the normative population. Conversely, 2 studies found behavior to be normal in children with lower limb deformities [105,112]. A study involving 45 children with lower limb deformities found that the behavior of these children, as measured by the parent-reported Post Hospitalization Behavior Questionnaire, was within the normal range [105]. Bond et al. found that children with lower limb deformities awaiting limb reconstruction surgery had normal behavior as measured by the parent-reported Personal Adjustment and Role Skills Scale as compared to healthy children [112].  Determinants of psychological health investigated in 3 studies [27,72,126] were complications post-surgery, type of treatment, and stage of treatment. Significantly higher self-esteem was found in patients with achondroplasia who underwent limb lengthening versus nonsurgical patients [126]. Fewer complications post-limb lengthening surgery were associated with higher self-esteem compared to children who experienced more complications post-surgery [126]. Emotional functioning scores were higher 3 months following frame removal versus preoperatively, suggesting improved QOL in children undergoing external fixator treatment for their lower limb deformities [72]. There was no difference found in the levels of psychological distress pre- versus post-operatively in children and adolescents after Ilizarov leg lengthening [27].  2.3.3 Social health Social health was studied in 7/10 studies using 10 instruments  [68,72,112,124–127]. The sub-concepts measured were family, peer relations (PRs), school, and social function (SF). SF was the most frequently measured sub-concept in 6/7 studies using 8 instruments [66,72,112,125–127]. Five studies  26 measured the sub-concept family using 5 instruments [112,124–127]. Some aspects of family measured in these studies were family activities, family cohesion, family conflicts, parental satisfaction, and family finances. The sub-concepts PR [66,112]and school [66,127] were measured in 2 studies each. PR included aspects such as bullying, social acceptance from peers, support from friends [66] and PRs [112].  SF was found to be significantly lower than the normal population in children with LLD [72] and congenital lower limb deficiencies [66]. However, Bond et al. found SF to be normal in children with lower limb deformities as compared to the normal reference population [112]. Lower limb deformities had no effect on family as measured by the Family Environment Scale [112]. The Child Health Questionnaire (CHQ) showed significant effects on families of children with an LLD [125]. In terms of PR, there was no significant difference between the study population and normative scores, as measured by the parent-reported Personal Adjustment and Role Skills Scale [112]. Children with lower limb deformities were not found to have significantly different scores relative to a control group of school children in academic performance at school [127].  Determinants found to be significantly related to social health in 2 studies [72,128] included severity of deformity, type of deformity, and stage of treatment. SF was found to be better 3 months after removal of an external fixator device as compared to preoperatively in children undergoing treatment with an external fixator for their lower limb deformities [72]. Children with non-idiopathic lower limb deformities scored significantly lower than children with idiopathic lower limb deformities on the sub-concept family, suggesting higher impact on parental emotions and family activities as measured by the CHQ-Parent form [125]. This study also found  27 that a limb length discrepancy of 2 centimeters or more had a negative impact on SF, parental emotions, and family activities [125].  2.4 Discussion This systematic review was conducted to synthesize the available evidence on the assessment of QOL of children with lower limb deformities using PRO instruments. We found that there were no validated PRO instruments specifically designed to measure QOL of children with lower limb deformities.  The existing instruments that are widely used in pediatric orthopaedics either measure the physical/functional aspects of the lower limb and are developed for an adult population [128]or are not lower limb specific [129]. QOL of children with lower limb deformities has been measured using either a generic [72,125] or a parent-reported QOL instrument [124]. More recently, Fabricant et al. have modified and validated the Scoliosis Research Society instrument for assessing QOL of adults with lower limb deformities [130]. In spite of this, there is still a need for a patient-reported QOL questionnaire for children and adolescents with lower limb deformities.  We identified one instrument for parents of children with LLD [124]. The content for this 34-item questionnaire was generated from a literature review and interviews with 58 parents of children aged 6–20 years. This questionnaire measures parental satisfaction, effect of LLD, preferred treatment, parent's mood, and concerns and parental expectations regarding the treatment. There is currently limited psychometric information (content validity and internal consistency only) about the performance of this questionnaire. More research is needed that looks at other scale  28 characteristics, for example, test-retest reliability, construct validity, or responsiveness [82,92].   Giving voice to children to express their concerns about their life with lower limb deformities is important in developing an instrument to measure their QOL. The assumption that children are unreliable respondents about their QOL, due to a lack of linguistic, reading, and cognitive skills, is increasingly being challenged [82]. In general, good agreement has been found between child and parent reports for domains of physical activity, functioning and symptoms, but for emotional and social domains, poor agreement has been found [131]. Discrepancies known as cross-informant variance [132] between parent's and patient's own perspective of QOL has been consistently documented in several studies of QOL in both children with chronic health conditions and healthy children [133,134]. Hence, both children and parent proxy-reports should be incorporated to completely understand the complex and multidimensional construct of QOL [135,136].  Our systematic review identified determinants reported to have a statistically significant relationship with some aspect of QOL of children with lower limb deformities. However, there could be a number of other factors not identified in these previous studies that influence the QOL of these children. Social support, family functioning, self-perceptions of appearance, school, behavior, athletics, and social competence are some of the determinants affecting the self-esteem of children with limb deficiencies in general [132]. As such, there is a lack of information on determinants of QOL of children with lower limb deformities in the existing literature. Hence, future studies need to specifically aim at collecting information on determinants of QOL for this specific population.  29  We identified that 24 instruments used to measure QOL in children with lower limb deformities measured a wide range of concepts (3 concepts and 15 sub-concepts). This variation indicates the lack of consensus on sub-concepts important for assessing QOL for these children. Even for the most frequently measured sub-concept of psychological distress, there were 11 different instruments used. The heterogeneity in instruments used for QOL measurement also highlights the potential difficulty encountered while generalizing results across studies. Furthermore, there are other previously identified challenges while conducting PRO research with children and adolescents which makes outcome assessment and generalization of the results more challenging in general [137]. Some of these challenges are due to developmental differences in various age groups, age-related vocabulary, and comprehension of health concepts [137]. Outcomes assessment is further complicated in lower limb deformities due to the lack of a universally accepted classification system for assessing lower limb deformities. However, the classification system developed by the Limb Lengthening and Reconstruction Society (LLRS-AIM classification) has been introduced to address this deficiency [138].   Hence, we aim to develop a new patient-reported QOL instrument, which has been systematically developed by following international guidelines for PRO development, [92,93] psychometrically tested and is clinically meaningful. Development of a PRO instrument is an iterative process with the first step being the development of a preliminary conceptual framework of concepts and sub-concepts important to the target population [93]. We used the preliminary conceptual framework derived from our systematic review to inform our interview guide for qualitative interviews with children with lower limb deformities and their parents. We  30 have established an international multidisciplinary collaboration involving five centers across Canada, Ethiopia, India, and the USA to develop our QOL questionnaire. Our qualitative phase is well under way with 37 interviews conducted to date. Our qualitative phase also consists of novel methodologies such as photo elicitation interviews to overcome linguistic barriers of our young patient population. Item generation and scale development will be followed by cognitive debriefing interviews with children from the five participating centers. Expert opinion will be obtained from our established multidisciplinary collaborative team which consists of orthopaedic surgeons, nurses, occupational therapists, physiotherapists, psychologists and experts in the field of PRO development and QOL studies. Expert opinion from members of relevant specialist societies will be sought. Field testing, psychometric analysis and cross-cultural validation will be done across all five participating centers. Following the ISPOR PRO, good research practice guidelines and acknowledging the challenges associated with using one outcome assessment for both children and adolescents, we aim to develop age-appropriate questionnaires [137]. We will develop separate QOL questionnaires for age groups 8–11 years and 12–18 years. Once developed, this PRO instrument could be used to measure the success of various treatment options for children with lower limb deformities to aid clinical decision-making, including type and timing of surgery, and patients/parent counseling.  2.5 Conclusion  Existing parent-reported outcome and PRO instruments measure 3 QOL concepts in children with lower limb deformities. There were no validated PRO instruments specifically designed to measure QOL of children with lower limb deformities.    31 2.6 Update Since the completion of this systematic review in 2016 and subsequent publication in 2017, there were some modifications to the study protocol as discussed below. At the time of completion of this systematic review, incorporation of photo-elicitation to the qualitative phase was planned. However, it was not carried out due to resource constraints. The qualitative study was multicentre, and to incorporate photo-elicitation for the initial qualitative interviews was found to be a very resource-intensive procedure for the participating sites.   As suggested by the ISPOR guidelines, we planned to develop separate versions of the PROM with age-appropriate content and vocabulary for the age groups 8-11 and 12-18 years [137]. However, it was found during the qualitative phase that the concepts of interest were similar across the age groups. If adequate attention is given to select age-appropriate vocabulary and reading level for the target patient population, development of two separate versions can be avoided. Hence, item generation and scale development (Chapter 4) focused on writing the items at the lowest possible grade level, appropriate for an 8-year old. Items were further edited as indicated during the cognitive debriefing interviews and expert feedback (Chapter 5) to ensure that the items are comprehensible and relevant to children aged 8-18 years.  New studies relevant to this topic have been published in the past four years. There was one study published in 2016, right after the cut-off for systematic review literature search and one more recent publication pertinent to this topic, as discussed below. These two studies used 8 instruments to measure HRQL and behaviour related issues in children with congenital limb deficiency [45,139]. These instruments included an in-house questionnaire developed for children  32 with limb deficiencies (both upper and lower limb), PedsQL generic core scale, PedsQL Family impact Module, PedsQL Pain Questionnaire, Piers-Harris Children’s Self-Concept Scale, Behaviour Assessment System for Children, Limb Lengthening Satisfaction Questionnaire (LLSQ) and Childhood Amputee Prosthetics Project-Prosthetics Satisfaction Inventory (CAPP-PSI) [45,61,139–144]. No new HRQL concepts were identified in these two studies that have not been previously identified in our systematic review in 2016. These studies did not specifically mention any determinants of HRQL.  Johansen et al. looked at HRQL and school-related issues in children with congenital limb deficiencies [139]. They used an in-house questionnaire and PedsQL to measure HRQL. As stated in this study, the in-house questionnaire was developed due to the lack of a standardised questionnaire applicable to this patient population. However, the questionnaire development only included input from organisations representing this diagnosis, parents of children with limb deficiencies, and adults with limb deficiencies. This questionnaire when developed was completed only by the parents of the children with limb deficiencies. Their study found that children with multiple limb deficiencies including lower limb deficiencies have more challenges as compared to children with upper limb deficiencies. Children with upper and lower limb deficiencies were able to participate in activities at school even when some of them were using assistive devices. However participation in physical activities presented a challenge for the children and their teachers and required planning and cooperation.  Birch et al. in their paper published in 2019, compared amputation with reconstruction for children with fibular hemimelia [45]. This study showed no significant functional and  33 psychological differences between both groups at mid-childhood (average age of study participants being 9 years). There are several limitations to this study. They have compared children who had amputations and reconstruction procedures prior to 2004. There have been significant advancements in the field of limb reconstruction that can reduce complications associated with these procedures. Similarly, there have been improvements in the field of prosthetic limbs as well. Hence the results from this study that are based on procedures done more than 17 years ago should be interpreted with caution. The study used several questionnaires for functional and prosthetic assessment and psychosocial assessment. Psychosocial assessment was conducted using generic questionnaires such as PedsQL, Piers-Harris Children’s Self-concept Scale and The Behaviour Assessment System for Children. Two condition specific questionnaires were used to look at satisfaction with both procedures. The LLSQ used in this study is a 5-item questionnaire that uses a 10 cm visual analogue scale to assess a child’s satisfaction with their limb lengthening. The LLSQ was first developed as a parent-reported questionnaire to assess the parental satisfaction with their child’s limb lengthening procedure and parental report of their child’s satisfaction with the limb lengthening procedure. The parental version of the LLSQ was adapted from the parent version of CAPP-PSI [144]. There is no further information available on the validation of the LLSQ. Childhood Amputee Prosthetics Project- Functional Status Inventory (CAPP-FSI) is a standardised measure of parent-reported functional status of children ages 3-17 years with upper and lower limb deficiencies only. Content was developed based on a literature review and input from clinicians. There was no involvement of children with limb deficiencies in any phase of content development.    34 The Patient-Reported Outcome Measurement Information System (PROMIS) includes a number of person-centred measures to evaluate and monitor physical, mental, and social health in adults and children.[145]. Self-report measures for children between the ages of 8-17 years are available. PROMIS measures are psychometrically sound and can be used as complementary measures along with condition-specific instruments for children with lower limb deformities.   35 Chapter 3: What Matters to Children with Lower Limb Deformities: An International Qualitative Study Guiding the Development of a New Patient-Reported Outcome Measure  This chapter in its current form is published in the Journal of Patient Reported Outcomes. Chhina H, Klassen A, Kopec J, Oliffe J, Iobst C, Dahan-Oliel N, Aggarwal A, Nunn T, Cooper. A. What Matters to Children with Lower Limb Deformities: An International Qualitative Study Guiding the Development of a New Patient-Reported Outcome Measure  3.1 Introduction  Lower limb deformities include conditions such as leg length discrepancy, lower limb deficiency and associated angular and rotational deformities of the hips, knees, ankles and feet. Lower limb deformities can be separated into two broad categories: 1) congenital deformities, which may result from defects in fetal development, and 2) acquired deformities, which arise from trauma, infections, tumours and other medical conditions [1]. Epidemiological data vary among individual deformities. For example, in a retrospective study from France, leg length discrepancy of greater than 2 cm was found in at least 1 in every 1000 people [9]. In the USA, the overall incidence of limb reduction defects is estimated to be 2 per 10000 live births [10]. The rate of limb deficiency defects in Canada (excluding Quebec) in 2014 was 3.74 per 10,000 births [16]. Fibular hemimelia, a congenital deficiency of the long bones, has an estimated incidence between 7.4 and 20 per 1 million live births [12–15]. The incidence of tibial hemimelia is reported to be 1 per million live births [11,146]. The incidence of congenital femoral deficiency is 1 in 52,029 [15]. However, there is  36 a lack of up-to-date data on the epidemiology of these congenital conditions and lower limb deformities.   Children with lower limb deformities often have physical limitations due to gait irregularities and pain. These differences in the appearance and function of their lower limbs can discourage participation in social, recreational and leisure activities, which may result in behavioural, emotional, psychological and social adjustment problems [3,66,68,69,147]. The health-related quality of life (HRQL) of these children is often impacted due to the anatomic and functional factors discussed above, as well as by the complex surgical procedures for their treatment [25,27,72,105].  Treatment options depend on the type of limb deformity, as well as the clinician’s skills and institutional resources available at the treating centre. Non-surgical treatment options include shoe-lifts, braces and step-in prostheses. Surgical treatment options for limb deformities in children vary from limb lengthening and reconstruction to amputation. Choice of treatment is usually dependent on a number of factors including clinical presentation [31], the desires of the patients and parents, the predicted HRQL, cultural influences, cosmetic preference and socio-economic status of individual families [66,68]. Socio-economic status has been known to be the fundamental cause of health disparities between high-income, lower-middle and low-income countries. Hence, the availability and access to treatments vary considerably between these countries. Furthermore, the high-income countries are using more sophisticated surgical treatment options, such as internal lengthening devices and latest hexapod external fixators with the ability to correct deformities more precisely, whereas the majority of low-income and lower-middle- income countries are still using traditional external fixators when available. Overall,  37 there is less specialist surgical workforce in low- income and lower-middle- income countries [148].  Each treatment option has advantages and disadvantages. Amputation is a non-reversible surgical procedure followed by fitting of prosthesis. Amputation is associated with potential stump overgrowth and pain. Prosthetic limbs require periodic replacement due to patient growth, breakage or fitting problems and lack normal sensation and proprioception [32]. The life-time cost of prosthesis replacement can be substantially higher than the repeat surgical procedures for limb lengthening [33].   Reconstruction by lengthening corrects leg length discrepancy and deformity by utilizing distraction osteogenesis [108]. This approach is more involved and may require multiple procedures. An external or internal fixation device is applied to the leg and remains in place for months. The child experiences multiple hospital visits and extensive physiotherapy and rehabilitation. This treatment is psychologically stressful due to its duration, impact on schooling and the potentially high rate of complications [17,25–27].   There is insufficient evidence in the literature to support one treatment option over the other. Some studies show successful outcomes with amputations [32,35–38], while others have also reported similar successful outcomes with lengthening and reconstruction [39–43]. Some comparative effectiveness studies have found no difference when comparing amputation and reconstruction [44,45].   38 The lack of evidence demonstrating the superiority of either treatment options and its effect on HRQL limits the ability of healthcare providers to counsel families on the best evidence-based treatment option for them. Consequently, there is a need for a rigorously designed patient- reported outcome (PRO) instrument to collect data from patients with lower limb deformities. These data could be used to inform parents and children about outcomes (physical, social, psychological) associated with these procedures. This information could supplement other objective outcome information (e.g., complication rates, how the leg will look, etc.) to help families to come to a more informed decision on a child’s course of treatment [89].  Our systematic review identified the lack of a validated and psychometrically tested PROM for children with lower limb deformities [149]. To fill this gap, our team is developing a new PROM called LIMB-Q Kids [90]. A rigorous qualitative phase is crucial to the development of a new PROM [98]. Qualitative interviews can provide a deeper understanding of what is important to a patient and establish the instrument’s content validity [94,95]. In this chapter, we describe the international qualitative study which guided the development of LIMB-Q Kids. Our aim was to identify concepts of interest important to children with lower limb deformities and use this knowledge to develop a conceptual framework of HRQL for these children.   3.2 Methods Individual semi-structured face-to-face interviews with children with lower limb deformities and their parents were conducted at five international sites: Canada (2 sites), Ethiopia, India and the USA. Ethics approval was obtained at the primary study site (CW15-0215 / H15-00514) and  39 each of the participating sites. A written informed consent was obtained from the parents and assent was obtained from the children.  3.2.1 Sampling Purposive sampling was used to recruit a maximum variation sample of children with lower limb deformities that included patients who varied by type of lower limb deformities, treatment type, stage of treatment, age, gender and country of residence.  3.2.2 Inclusion criteria Children aged 8 to 18 years with a confirmed diagnosis of a lower limb deformity were eligible. Parents of these eligible patients were also invited to participate in individual interviews.  3.2.3 Exclusion criteria Patients with lower limb deformities along with other medical conditions which may have a confounding effect on their HRQL and conditions such as cognitive or developmental delay affecting their ability to communicate were excluded from this study. Isolated deformities of hip and foot were excluded.  3.2.4 Study setting, recruitment and data collection Recruitment took place at hospitals in Canada, the USA (high-income country), India (lower-middle-income country) and Ethiopia (low-income country). The two sites from Canada and 1  40 site from the USA were tertiary care hospitals providing specialised orthopaedic care to children across their respective provinces or states. The site from India was a leading tertiary care hospital providing care to patients from several states in India. The site from Ethiopia was a pediatric orthopaedic teaching hospital that provided care to physically disabled children. Our two centres in Ethiopia and India primarily catered to patients with low socio-economic status with limited access to medical care.   Recruitment at these sites was done by their respective research teams in consultation with the primary study site (senior author’s site). An interview guide was prepared in English (attached in Appendix B, Pages 196-206) and translated into French, Amharic, Hindi and Punjabi. The interview guide was based on the preliminary conceptual framework derived from our systematic review [149]. Feedback from the orthopaedic surgeon (senior author) and an expert in PROM development (AK) also informed the interview guide. The interview guide was modified during the process to gain understanding of the emerging themes.   All English interviews in Canada and the USA, and Hindi and Punjabi interviews in India were conducted by the first author (HC). The first author is a native speaker of both Hindi and Punjabi languages and is also fluent in English. Interviews at the French-speaking Canadian site and Amharic-speaking site in Ethiopia were conducted by the local research assistants who were trained by the first author. Both the researchers from the French-speaking Canadian site and Amharic-speaking site in Ethiopia were native speakers of French and Amharic, respectively. They were both fluent in English as well. Interviews were conducted until data saturation was  41 reached, i.e., no new information or thematic variation emerged in the subsequent interviews [150,151]. 3.2.5 Data analysis All interviews were recorded and transcribed verbatim. A pragmatic approach to translation was used in this study. Hindi and Punjabi interviews were transcribed and translated into English by the first author (HC). French interviews were translated and transcribed by the same researcher from the French-speaking site in Canada who conducted the interviews. Amharic interviews were translated into English and transcribed by the research assistant from the Amharic-speaking site who also performed the interviews. Triangulation was achieved by interviewing the parents or caregivers of the children as a second source of data [152,153]. While analysing the interviews, variables such as age at the time of the interview, country of residence, and child or parent interview were included to see if the concepts were relevant across country, age and parent versus child.  This study adopted an interpretive description (ID) approach [154]. ID is a non-categorical qualitative approach which aims at developing knowledge for the clinical context of applied health disciplines [154]. Transcripts were analysed using a line-by-line coding approach to label and identify recurrent themes and patterns. Coding involved an inductive (from the interviews) and deductive process (preliminary conceptual framework from our systematic review). Constant comparison was used to compare interview data and categorise the concepts of interest (COI) into major and minor themes [155]. The results of this analysis were used to refine the preliminary conceptual framework from our systematic review [149].   42 3.2.6 Rigor We used Guba and Lincoln’s factors to assess four criteria of rigor as suggested by Sandelowski [156,157]: truth value, applicability, consistency and neutrality. Truth value of a qualitative study is tested by the credibility of the results. Our study findings are supported by relevant quotations from the participants. We also used triangulation as a way of ensuring credibility of our results. Triangulation involves collecting data from multiple sources/multiple points of view in an attempt to address the subjectivity in qualitative work and as a way of validating the findings [158]. We achieved triangulation in our study by interviewing the parents/caregivers of the children separately as a second source of data. According to Guba and Lincoln, the applicability of the results of a qualitative study is determined in terms of fittingness [157]. Fittingness refers to the ability of the research findings to fit the data from which they were derived. Quotations from the study participants demonstrated the fit between the data and study findings. The consistency of the qualitative findings is evaluated by their auditability [157]. A description of the coding process and ongoing discussion with the research team members contributed towards the auditability of the findings. Peer debriefing, which involved discussing the ongoing analysis with the research team, was also conducted on a regular basis to verify the study findings [155]. To further support auditability, close contact was maintained with the study team members with expertise in qualitative research and PROM development. We used confirmability as the criterion for neutrality, which means that we were reflexive about the research process and the findings [157,159]. Reflexivity involves awareness of researchers’ biases and beliefs that can influence decisions during the data collection and analysis, including categories not ultimately borne out by the data [160]. The first author also kept a journal to signal thought processes and interpretations, in accounting for how individual patient or parent stories were selected as  43 representative and thematic. The authors involved in developing the coding schedule were non-clinician researchers. Individual investigators from the participating sites were clinicians who were directly involved in the clinical care of children with lower limb deformities. These investigators were involved in the preparation of the manuscript, including a detailed review of the qualitative analysis. The involvement of multiple perspectives and discussions to reach a consensus reduced individual biases.  3.3 Results Seventy-nine interviews (39 children and 40 parents) were conducted with children with lower limb deformities and their parents from four countries (Canada, Ethiopia, India and the USA) (Table 4, Page 115). Interviews were conducted at individual sites between 2015 and 2018. Out of the 39 interviewed children, 2 had amputation, 7 had no treatment at the time of the interview, 20 were in the active treatment phase (external fixators, internal lengthening nails, osteotomy) and 10 had some treatment done in the past (external fixators, internal lengthening nails). A list of lower limb deformities of the interviewed participants is included in Table 5 (Page 116). Five main themes emerged from the qualitative interviews and formed the basis of the conceptual framework (Figure 4, Page 155). These five themes were: 1) appearance, 2) physical health, 3) psychological health 4) school and 5) social health. These themes formed the basis for the development of outcome scales for LIMB-Q Kids. Other factors such as coping, emotional and instrumental support were identified to be the mediating factors influencing HRQL. Below we describe each theme and provide illustrative quotes (Table 6, Pages 117-128) from interviewed children (labelled as ‘C’) and their parents (labelled as ‘P’). It is important to note that while we  44 conducted individual interviews with children and their parents separately, no new themes emerged from the parent interviews only.   3.3.1 Appearance Leg appearance was an important theme for the interviewed children.   Appearance of specific body parts and scars: Appearance was discussed in terms of the leg overall, specific parts including hips, knees, feet and surgical scars (Quotes 1-11). The children talked specifically about the shape, symmetry, size and overall appearance of their legs (Quotes 1-4).  Clothing: Children also discussed how their leg looked when wearing different types of clothing and foot-wear (Quotes 12-16). Children talked about the type and fit of clothes and overall appearance with certain clothes (Quotes 13-17). They discussed how they were not able to wear different types of clothes as they would like. Some children talked about not being able to wear shorts or short skirts and they wore long pants to hide their leg. They also described having to wear specific types of clothes when they had an external fixator device (Quotes 13 and 14). Adolescent children were also concerned about the appearance of their legs in photographs (Quote 19).  Shoes and other devices: Children talked about their appearance when wearing treatment related devices such as splints, crutches, external fixator devices, braces, prostheses, and shoe-lifts (Quotes 17 and 18).   45 3.3.2 Physical health Children talked about their limitations with physical function and their symptoms.  Physical Function: Under physical function, the children discussed their limitations with mobility, balance, sports and recreational activities, activities of daily living (ADL), and their adaptations for specific activities. Most children were unable to walk or run long distances without discomfort or pain (Quotes 20-23). The mobility limitations were either due to their limb deformity or due to the shoe-lifts or external fixator devices.  Children also mentioned losing balance, feeling uneven and falling (Quotes 26 and 27). They described limitations in the type of sports they could participate in due to their limb deformity and treatment related devices such as external fixators and shoe-lifts (Quotes 30-32). Some children mentioned choosing alternate sports or adapting themselves accordingly (Quotes 28 and 29). Most children were able to perform ADLs without any limitations even when they were wearing their prosthesis. However, some children mentioned having problems with ADLs (putting on or taking off clothes, etc.) while they were in the external fixator devices (Quote 24).  Symptoms: Children talked about the symptoms they experienced due to their lower limb deformity. Symptoms included pain, feeling tired and limping (Quotes 33-36). Pain involved the leg as well as other parts of the body. Pain was described in terms of its frequency and severity (Quotes 37-41). Pain was reported during the time period when they had the external fixators on. They also discussed interference of pain during walking and sleeping at night (Quotes 33-34, 41).  46 3.3.3 Psychological health Lower limb deformities impacted the psychological health of children. Children shared their concerns about their body image, talked about the distress due to their leg-related problems, how their leg-related problems impacted their confidence and self-esteem and how they developed coping strategies. Some children indicated how their leg problems had a positive impact on them.  Body image: Children with leg deformities perceived their body image diversely. Some children with congenital deformities perceived themselves as being ‘normal’ since they were born with the lower limb deformity. At the same time children who acquired the deformity due to trauma, infection, tumours or other medical conditions later on in their life perceived themselves as not being ‘normal’ and experienced challenges adapting physically and psychologically (Quotes 42-44).  Children described a range of strategies to conceal their leg with the deformity or their leg with the external fixator device. Some avoided wearing certain types of clothes while others would not go out with clothes that revealed their deformity (Quotes 45 and 46). Some children with congenital deformities walked with an altered gait to conceal their limp (Quote 46). Some children felt ashamed or embarrassed about their body image with their limb deformity (Quote 45). Children described that they felt self-conscious due to their leg deformity, limping, external fixator devices or the shoe-raises they had to wear (Quotes 47-50).  Distress: Most children had experienced leg-related distress to some extent either due to their physical limitations, appearance related issues due to the deformity, external fixator devices or  47 shoe raises, complex surgical procedures, and limitations in social participation. Their distress was expressed in various emotions such as being worried, feeling down, crying, feeling irritated, and angry (Quotes 57-68). Children were worried about their surgeries and how their leg problems would affect them in the future (Quotes 53-56). They were worried about limping, other people staring, not being able to walk and the leg length difference happening again after they had received the treatment. While some adolescents were also worried about the kind of jobs they can do in future with their leg problem, younger children worried about their leg or foot hurting while playing. Children also felt being down, sad, upset and discouraged at times due to the challenges they were facing either due to the deformity itself or due to the treatment procedures such as having the external fixator device on for months (Quotes 57-68).  Confidence and Self-esteem: Children talked about how their leg problems and external fixator devices affected their confidence and self-esteem (Quotes 69-75). Some of them didn’t feel comfortable getting their pictures taken while others felt inferior to other kids (Quote 75).  3.3.4 School Most children talked about the impact of having a limb deformity on their school life. They discussed their limited participation in activities at school, having to miss school, the isolation they felt at school, emotional and instrumental support from their friends and teachers and some school-based environmental barriers.  Function: Children mentioned having to miss school because of their frequent appointments with the doctors, physiotherapists and occupational therapists and recovery period after surgeries.  48 While some children were ok going to school with the external fixator devices on others were scared to go to school (Quotes 83-92). Most children interviewed from Canada and the USA were able to go to school while some children from India and Ethiopia had to miss a lot of school or were not able to go to school at all during the treatment phase. They either had to travel to different cities for treatment or had physical limitations getting to school with their deformity because they couldn’t afford the treatment (Quotes 87-92). Some children missed school due to pain and other complications of their surgical procedures (Quotes 91 and 92).  Participation: Children talked about their limited participation at school in academics, sports and recreational activities. Most participants did not describe any impact on their academic performance due to their limb deformity. However, a few children from India and Ethiopia mentioned missing an entire year of school due to treatment (Quotes 93-95). Participation in sports and other activities at school was impacted for most children. Some children found alternate activities that were physically less demanding to accommodate their lower limb deformity or while they were in the external fixator devices (Quotes 93-95).  Isolation: Children felt isolated and left out at times due to being teased by their peers, being asked about their legs all the time, having to tell other people about their leg, shoe-lifts and external fixator devices (Quotes 104-108).  Environmental barriers: Children mentioned having problems at school due to the environmental barriers such as taking the stairs with their external fixator devices and problems when they were in a wheelchair (Quotes 102 and 103).   49 3.3.5 Social health All children interviewed were school-age so most of their social life was around friends at school or school-related social activities. However, some children did talk about the impact of their deformity on the social life outside of school.  Social Function: Children talked about how their social function was affected in terms of making new friends, going out with friends and family (Quotes 109 and 110). Their limited physical function and concerns about their appearance directly impacted their social function in terms of being unable to participate in social activities with friends and family (Quotes 111 and 112). Adolescent children mentioned how they would want to post pictures on social media. Some adolescent children also shared their difficulties in finding jobs, especially the children with prosthesis (Quotes 112, 116-117).  Isolation: Children felt socially isolated and left out when their friends and other people noticed their limping, stared at their leg and asked them about their leg. Some experienced teasing from peers. They felt lonely and isolated when they couldn’t go out and play with their friends and siblings (Quotes 125-136).  3.3.6 Mediating factors  The mediating factors, as identified based on the qualitative analysis, were the factors impacted by the deformity that influenced the children’s perceptions of their HRQL. Coping, emotional and instrumental support were the three mediating factors found in this study.   50 Coping: It was evident from the interviews that some children with lower limb deformities had developed their own coping strategies to deal with their challenges (Quotes 76-82). One child mentioned keeping a journal to document their feelings while another accepted their deformity philosophically, suggesting that everything happens for a reason. Another child also mentioned feeling better thinking that their problem was not as bad as some other people’s. Some children coped with their scars by considering them to be reminders of what they have been through. There was a positive impact of their lower limb deformity seen on some children. They felt stronger and more motivated to do certain activities which they couldn’t do during their treatment especially if it had involved an external fixator device. Strong religious beliefs and acceptance of their deformity as god’s will were also seen in interviewed participants from Ethiopia.   Emotional and instrumental support: Children talked about the emotional support they had at school from their friends and teachers (Quotes 96-98). They were also getting help with physical activities, such as carrying a backpack (Quotes 99-101). Children also talked about the emotional and instrumental support from friends, people and family (Quotes 118-124).  3.4 Discussion The purpose of conducting this qualitative study was to identify the prevailing concepts representative of an international sample of children with lower limb deformities as a means to developing items for a new PROM. The framework of HRQL concepts guided the development of items and scales for an internationally applicable PROM for this patient population (Chapter 4).  51 The preliminary conceptual framework developed based on our systematic review identified three overarching health concepts including physical, psychological and social health [149]. These concepts identified in our systematic review were also consistent with studies looking at the impact of tumours in the lower limb on HRQL [161–167]. A systematic review by Bekkering et al. summarizes 12 studies, looking at HRQL, functional ability and physical activity in children and adults with bone cancers of the lower limb [168]. While the overarching health concepts identified in our international qualitative study remain the same as identified in our systematic review, we found certain themes in our qualitative study that were not emphasized in the literature but were important to the children with lower limb deformities. For example, appearance of the leg was an under-represented theme in the literature that was found to be an important theme in the final conceptual framework derived through the interviews. Children talked about the appearance of their legs, knees, hips and feet. This is now reflected in the conceptual framework emphasizing the importance of appearance for these children. A saturation table (found in Appendix B, Pages 204-205, supplementary material for Chapter 3) shows how each theme was endorsed in interviews.  Similarly, school was a major theme based on the qualitative interviews. Given that our target patient group is between the ages of 8 and 18 years when children typically go to school, it is not surprising that most children talked about school-based challenges. Our findings of lower participation in physical activities at school and missed days of school are in line with the similar findings from previous studies [139,169].    52 Our findings of concealment by wearing specific clothes or avoiding more revealing clothes, and hiding their affected leg were similar to strategies for hiding upper limb deficiencies [170,171]. Children discussed several issues with the external fixator devices. These issues were found to be temporary and only encountered during the time they used the external fixators. However, it is common for children to require treatment with an external fixator multiple times over the course of their childhood with the duration of use as long as nine months each time.   The strength of this study is the heterogeneity of the sample for interviews, in terms of age, gender, type of deformity, type of treatment, stage of treatment, country of residence and socio-economic status. This diverse sample enabled us to identify a range of concepts important to an eclectic international population of children with lower limb deformities, increasing the generalizability of the PROM. The overall thematic findings resonated with and fairly represented the entire sample included in this study.   With the aim of understanding what’s important to children with lower limb deformities, we also interviewed the parents of these children. A recent review indicating the current recommendations for proxy reports suggests that observable concepts are often reliable while the subjective concepts such as emotional well-being and social functioning are found to be unreliable when measured using the proxy reports [172]. Similar discrepancies between child and parent-proxy reports have been reported in a study looking at HRQL in children with congenital lower limb deficiencies using generic HRQL instruments [66]. The analysis of parent data in our study did not find any new themes other than those found from the interviews with children. This  53 is also supportive of the previous literature suggesting that by 8 years of age children can reliably report on their well-being, psychological health and health promoting behaviours [83–85].  There are some limitations to this study. We acknowledge the potential for sampling bias given the centres selected for qualitative interviews. Since our centres in Ethiopia and India primarily serve patients with low socio-economic status, some of these children might have reported substantially higher impact of their limb deformity on HRQL. However, since we did not collect any information on the socio-economic status of the participating children, this conjecture is only based on the author’s knowledge of the individual participating sites. For this study, we were only able to interview children from four countries. The number of children with amputations included in this qualitative study was small. Hence, we might have missed some concepts relevant to children with amputations.   We recognise the importance of establishing trustworthiness of translating and transcribing verbatim qualitative interview data. The methodological challenges in cross-language qualitative research are well recognised [173]. A strength of our study is that members of the study team who translated the interview guide, performed interviews in local languages, and translated and transcribed them into English were native speakers of the local language and were fluent in English as well. A pragmatic approach to translation was used in this study. While there are limitations in not using a certified translator at this step, using researchers from the study team with bilingual skills as translators offers the unique advantage of not bringing in an ‘outsider’ to conduct, translate and transcribe these qualitative interviews. The interviewers had sufficient background knowledge about their individual sites and the orthopaedic conditions of the  54 children, were well aware of the study objectives, and were able to probe efficiently using conceptually equivalent terms as needed.   3.5 Conclusion Our study describes the impact of lower limb deformities on HRQL of children. This impact is further influenced by health inequities including low socio-economic status and access to healthcare in lower-middle-income and low-income countries. Nonetheless, the COI identified were similar across children from all countries. This detailed knowledge gained from interviewing children with lower limb deformities provided us with first-hand information about what matters most to these children. Physical health, psychological health, social health, school and appearance were the areas most important to children with lower limb deformities. This framework guided the development of outcome scales specific to these patients. Next steps included item generation (Chapter 4), and cognitive debriefing interviews with children with lower limb deformities and feedback from clinicians and experts in HRQL and PROM development (Chapter 5).   55 3.6 Update Since the publication of the manuscript based on this chapter, a more detailed framework adapted from the modified Wilson and Clearly model of HRQL and showing the relationships among the concepts important to children with lower limb deformities has been developed (Figure in the Appendix B, Page 206) [57]. In this framework, the physical deformity itself is seen as the primary cause of leg symptoms and limitations in physical function and hence physical participation in children with lower limb deformities. The limited physical function and participation in turn impacts the children’s ability to participate in social activities with their family, friends, and peers at school and outside of school. This can cause psychological health concerns in these children. The physical deformity also leads to appearance-related concerns which can further exacerbate psychological health issues and impact the children’s HRQL. Other factors that can impact HRQL are experience of care and access to healthcare, family support (emotional and instrumental) and social support. As identified through our systematic review, determinants of HRQL in this population included type of deformity, severity of deformity, type of treatment, stage of treatment and complications post-treatment. This proposed model encompasses the core elements of the revised Wilson and Cleary HRQL model, with symptoms and impaired physical function (functional status) due to physical deformity directly influencing general health perceptions and overall QOL [57].    56 Chapter 4: Item Generation and Scale Formation  4.1 Introduction  Development of a PROM is a complex, multi-step and iterative process (Figure 1, Page 152). Key steps for the development of a PROM include: 1) literature review; 2) concept elicitation; 3) development of a conceptual framework ; 4) item generation and scale development; 5) cognitive debriefing interviews and expert input for establishing content validity; 6) field-testing and evaluation of the psychometric properties of the PROM  [50,93,137,174]. Item generation and scale formation is a key step in the development process. With the aim to develop an internationally applicable questionnaire for children with lower limb deformities, it was important to ensure that the items were generalizable across patients from different countries. As per the FDA guidance for PROM development, ‘item generation is incomplete without patient involvement’ [48]. Hence the item generation and scale development for LIMB-Q Kids was guided by the qualitative interviews with the target patient population from different countries (Chapter 3) and the final conceptual framework developed based on the patient interviews (Figure 4, Page 155). In the process of item generation, appropriate guidelines from the FDA and ISPOR for PROM development, were followed [48,94,95,137]. In addition, an orthopaedic surgeon with expertise in treating lower limb deformities and experts in PROM development and HRQL assisted in content development. This chapter provides details of item generation and the formation of clinically relevant scales. 4.2 Methods  The final conceptual framework refined during the qualitative phase formed the basis for the development of the items. This framework had five main themes including appearance, physical  57 health, psychological health, school and social health (Figure 4, Page 155). For each of these themes, the codes developed during the qualitative analysis of interviews with the children (Chapter 3) were closely reviewed and items were written to cover the concept of each code until an exhaustive list of items was generated for the entire dataset. Items were developed in consultation with the team members who have extensive experience in HRQL studies and developing PROMs in children and adults. While writing items and selecting response options, the following guidelines for item generation were followed.  4.2.1 Item length The items were made short and concise to reduce the demand on verbal memory which is especially important for children [99,175].   4.2.2 Item wording In developing draft items and scales, the wording of the patients was used as much as possible to ensure that the items resonate well with the experiences of the children with lower limb deformities completing the items. The language of the items was kept simple and straightforward. Any colloquial terminology, complex, ambiguous, and double-barrelled items (items that combine two topics and could be interpreted differently by different respondents) were avoided. Positive or neutral wording of the items was used whenever possible. Negatively worded items, especially those involving logical negations, were avoided because such items could be difficult for children to understand [175]. As an example, consider the following two items: “I feel unhappy about my leg” vs. “I am not happy due to my leg problems”. In this  58 example ‘unhappy’ is preferable to ‘not happy’. Also, if a concept overlapped with another PROM developed, cognitively tested and validated by our team for children (e.g., CLEFT-Q a PROM for children and young adults with cleft lip and palate[176,177]), rather than creating new wording for that concept we re-used the already validated item. Permission to use items from CLEFT-Q Kids was obtained from Dr. Anne Klassen. The CLEFT-Q, authored by Drs. Anne Klassen and Karen Wong, is the copyright of McMaster University and The Hospital for Sick Children (Copyright ©2017, McMaster University and The Hospital for Sick Children).  4.2.3 Reading level It was ensured that the items had the lowest possible Flesch-Kincaid (F-K) readability level. The F-K readability level indicates the grade-reading level of an item (U.S. grade level). Readability cut-offs were determined as per the reading comprehension literature [178–180]. For our population ranging from 8-18 years, the majority of the items were written at or below grade 3 reading level.   4.2.4 Age-related considerations Recommendations from the ISPOR Task Force for Good Research Practices for Assessment of Children and Adolescents suggest that separate scales may be considered for children (8-11 years) and adolescents (12-18 years) [137]. However, during the qualitative analysis, it was found that concepts of interest most important to patients were similar for all age groups. Therefore, no separate items were created for different age groups, which will facilitate longitudinal research. Special considerations were given to selecting health-related vocabulary, reading level, response scale, and recall period appropriate for children [137].  59 4.2.5 Response options As suggested in the available literature, the number of optimal response options to get a reliable response from children is four [175]. Four-labeled response options are also in line with the guidelines published by Khadka et al. indicating that PROMs should have simple question formats, only a few (4 to 5 at most) response options and that all response options should be labeled [181]. We used a Likert-type scale with four-labeled response options with mutually exclusive, conceptually exhaustive categories. No neutral mid-point for response options was used. Children with their developing cognitive, communication and social skills can be more tempted to pick the neutral mid-point for several reasons, including satisficing, superficial responses, social desirability and a non-extreme response style [175]. Considerable attention was paid to the labels for the response options. Using familiar labels can lead to higher endorsement frequencies and is an important determinant of response behaviour. Labels with extreme intensity are less preferable. Feedback on the response options was obtained during the cognitive debriefing interviews (Chapter 5).   4.2.6 Recall period Shorter recall periods are usually recommended while balancing out the recall bias and respondent burden. The FDA PRO guidance indicates that longer recall periods requiring participants to rely on their memory can undermine content validity [48]. A one-week recall period was chosen for all scales included in this PROM, except the Appearance Scale, where the patients were asked about how much they like the appearance of their leg now. Same response options and recall period were chosen for scales measuring similar constructs as in other pediatric PROMs developed by our team (CLEFT-Q and FACE-Q Kids). These PROMs  60 (CLEFT-Q and FACE-Q Kids) were developed and cognitively tested with 153 children and field-tested with 4839 children, i.e., we know that they work with children in this age group [176,177,182].  4.2.7 Scale formation For the theme Physical Health, items were written for Physical Function sub-theme which formed the ‘Physical Function Scale’. To assess symptoms, items were generated to form separate scales for the leg and for anatomic parts of the leg, including the hip, knee, and foot. The first draft of each symptom scale was developed based on symptoms mentioned by the children during the qualitative interviews and feedback from a limb reconstruction surgeon (AC). Input from the limb reconstruction surgeon was crucial to make sure that the items cover a full set of clinically relevant symptoms for full clinical spectrum of lower limb deformities. For the theme Appearance, items were generated around the appearance of specific body parts, clothes and shoes. These items formed the ‘Appearance Scale’. For the Psychological Health theme, items were written for the sub-themes Body Image and Distress. Items related to both these themes were used to form the ‘Leg-Related Distress Scale’. A ‘School Function Scale’ was formed based on the items under the theme School. Similarly, a ‘Social Function Scale’ was formed based on the items covering the Social Function theme from the qualitative interviews. Items were written for the sub-themes Confidence and Self-esteem, which formed the basis of the ‘Psychological Function Scale’.    61 4.3 Results Ten scales, including Physical Function, Leg Symptoms, Hip Symptoms, Knee Symptoms, Foot Symptoms, Leg-Related Distress, Appearance, School Function, Social Function, and Psychological Function were formed. Version 1 of the scales used for the first round of cognitive debriefing interviews is found in Appendix C, Pages 207-218, supplementary material for Chapter 4. Version 1 of LIMB-Q Kids had 124 items. Below we describe how we used data from the interviews from children to guide the development of items within each of the scales. To this end, we provide examples of quotes from the interviews and examples of items that were informed by these quotes. In the quotes below, ‘I’ is the interviewer and ‘C’ is the child.   4.3.1 Physical Function Scale Version 1 of the Physical Function Scale had 28 items focusing on physical function required for day to day activities including sports and recreational activities for children. All items asked “Are you able to…?” The response options were: I CANNOT do this; I have A LOT of trouble doing this; I have A LITTLE trouble doing this; I CAN do this. The recall period for this scale was past one week. Examples of patient quotes and some items Quotes Items C: I can’t really bend my knee and my ankle doesn’t move at all so, I wear the brace to keep my ankle straight. …bend your knee? C: I don’t do much because of limping. …walk without limping? C: When I go up the stairs, I fell one day. I: How did you fall? C: I didn’t have balance. …walk up stairs? …run without falling? C: In my high school I played for the girls’ flag football team. I couldn’t even do that because I would get either tired running fast or at the end of practice I would feel way more …run as fast as you want?  62 Quotes Items uneven than I was. I cannot run really fast like other children. C: I have a problem changing my clothes when I am in frame, so I had to ask for help. …put on or take off clothes?  4.3.2 Symptom scales  Version 1 of the symptom scales was developed for leg, hip, knee, and foot. These scales had 7, 5, 5 and 6 items, respectively. The response options for all symptom scales were: Never, Sometimes, Often and Always. The recall period for all symptom scales was past one week. Examples of quotes and some items Leg Symptoms Scale: All items asked ‘How does your leg feel?’ Quotes  Items  C: It used to hurt to walk and I always cried when I walked. My leg hurts when I walk. C: When we do a running thing, I usually just sit out coz as I got older when I started to run it got more painful and harder, so now when they are doing, like, a running activity, I usually just don’t do it coz it hurts. My leg hurts when I run. C: Sometimes I have to go and, like, from the first floor to the second or third floor a bit slower about it and tire me out more. My leg gets tired when I walk.  Hip Symptoms Scale: All items asked ‘How does your hip feel?’ Quotes  Items  I: Did you also have any pain? C: Yes, I did have pain too. I: Where was the pain? C: In my leg and hips. My hip hurts. C: After, like, about 10 – 15 minutes it will start hurting. Ah, with sports, like I said with soccer, I play goalie. so it’s easier for me. Ah, sometimes I’ll just get, like, pain in my hip  My hip gets tired.     63 Knee Symptoms Scale: All items asked ‘How does your knee feel?’ Quotes  Items  C: Firstly, when I walk or when I play, when I throw the ball or when I run then I have pain in my knees. C: I walk really slowly and sometimes I’ll be able to walk faster but if my knee hurts, then I’ll walk slower. My knee hurts.   Foot Symptoms Scale: All items asked ‘How does your foot feel?’ Quotes Items C: Sometimes if I run too long, the foot hurts so much and then the next day I couldn’t put any pressure on it, because it hurt so much. My foot hurts when I walk. My foot hurts when I run. C: No, I can’t stand for a long time. I: What happens when you stand for a long time? C: My leg and foot get tired and my foot gets swollen. My foot gets tired when I walk.  My foot is puffy or swollen.  4.3.3 Leg-Related Distress Scale  Version 1 of this scale had 15 items about the psychological distress related to leg including different emotions, concealing the leg with the deformity or the leg with a special device (external fixator device, splints, etc.) and issues around body image. The items asked ‘How do you feel about your leg?’ The response options were: Never, Sometimes, Often and Always.  The recall period for this scale was past one week. Examples of patient quotes and some items  Quotes  Items  C: when I go to school wearing shorts or skirt cazuse when my leg shows in those clothes, I usually wanna cover it, like wrap a thing around it, so nobody can see it. I cover up or hide my leg when I go out.  C: I have to wear long dresses to cover my legs. I avoid wearing shorts or skirts. C: Sometimes I do worry about my leg. Will it get better; will I be able to walk straight. That it doesn’t get worse. I worry about my leg. C: Sometimes I would be mad if people over I get upset when people ask about my leg.  64 Quotes  Items  react and ask “How are you? How are you?  C: I get mad sometimes because when I wear shorts the scar is visible and people ask me what’s wrong with me and I don't like that. C: I would just post pictures of my upper body. Because my leg would look bent in pictures. I avoid having photos taken that show my leg. C: I’ll look at myself when I’m walking. It just feels different from the others. And as I started realizing, as I started getting older, I started freshman year, 9th grade year, it was high school, everything was different and I started feeling different about myself. I feel different from other people because of my leg. C: I’m in high school, people are looking at me and they start to make fun of you. I get teased about my leg.  4.3.4 Appearance Scale  Version 1 of the Appearance Scale had 18 items focusing on the length and size of the leg, appearance of hips, knees and feet, how their leg looks while walking, running, in specific clothes and photos. All items asked ‘How much do you like…’. The response options were: Not at all, A little bit, Quite a bit, Very much.  The items in this scale asked about how the leg looks now. Examples of patient quotes and some items Quotes  Items  C: My leg was like 7 cm shorter than the right leg. …the length of your leg? C: I can find clothes but you can clearly see my leg problems when I wear shorts and I always feel ashamed wearing shorts so I don't prefer wearing them. …how your leg looks in shorts or a skirt? C: This leg would be bigger that this one and this one was always skinny. …how well your legs match each other (look the same)? C: My leg would look crooked. …how straight your leg looks (not crooked or lopsided)?     65 4.3.5 School Function Scale  Version 1 of the School Function Scale consisted of 17 items asking about the children’s school life, including items about being able to go to school, feeling accepted, feeling safe at school (not bullied), friends at school and being asked about their leg problems at school. Ten items were borrowed from the School Function Scale for the CLEFT-Q kids with permission from the developers. The response options were: Never, Sometimes, Often and Always. The recall period for this scale was past one week. Examples of patient quotes and some items Quotes  Items  C: I stopped school for a year and I was not able to play with my friends either. I am able to attend school. C: My friends help me in any way they can. Like, if I just feel like a lot of pain, my teachers will try and help me out of my school, help me get around the school and stuff. My teachers help me at school. Other students help me at school. C: I’m in high school, people are looking at me and they start to make fun of. I get asked why do you limp? What’s your problem? But I was pretty used to that cause it happens since like kindergarten. Other students are nice to me. I feel accepted at school. I feel safe at school (not bullied because of my leg). I am ok if students ask about my leg. C: Well, I mostly play sports like football. Most of my classmates are very competitive and a lot of times they fall down so I can’t join them in the play. I couldn’t compete in anything for the sports competition at school. I just wish I could, like, put my own effort in it but I couldn’t. I get asked to join activities and games at school.  4.3.6 Psychological Function Scale  Version 1 of the Psychological Function Scale had 10 items about the overall psychological well-being of children with lower limb deformities including confidence and self-esteem. These 10 items were borrowed from the Psychological Function Scale for the CLEFT-Q kids with  66 permission from the developers. The response options were: Never, Sometimes, Often and Always. The recall period for this scale was past one week.  Examples of patient quotes and some items Quotes  Items  C: I get experience from my leg problem and I get to grow from this, I overcame, like, this whole problem by myself. I’m proud of it. I am proud of myself. I: so how did it affect your confidence? C: [Sigh] Well, like I said, it all goes with that. I didn’t even wanna get dressed in the mornings. Like I didn’t even wanna get ready for anything anymore. So it did affect my confidence somewhat and my self -esteem. I like myself. I feel confident. C: If I was to take a picture of you standing up, I wasn’t able to do that because I didn’t feel comfortable, like you would see me shifting. I would look like to the side and I didn’t like that because obviously people are gonna notice. I never like to be taken pictures of like full body. I feel good about myself.  4.3.7 Social Function Scale The Social Function Scale had 13 items about the children's social life including feeling accepted by friends, going out with friends and people staring or asking about their leg. Ten items were borrowed from the Social Function Scale for the CLEFT-Q kids with permission from the developers. The response options were: Never, Sometimes, Often and Always. The recall period for this scale was past one week. Examples of patient quotes and some items  Quotes  Items  C: No I don’t go out for weddings. I: When did you stop going? C: When I started limping. I: Do rest of your family go? C: Yes I feel confident when I go out (like to a party). I: Why do you think it’s difficult sometimes to make new friends? C: Just because they look at me differently than other people. C: And, um, they don’t wanna socialize, like, It’s easy for me to make new friends.  I get asked to go out with friends  67 talk and stuff. (like to a party). I: What happens when someone new meets you for the first time? Do they ask you questions about your leg? C: They ask me question, like what’s wrong with my leg, how did it happened. I: How does that makes you feel? C: Sometimes I answer the questions and sometimes I won't say anything to them and I will just walk away from them. I get angry because some of the people, I don't know them and they just stop me and start asking me questions.  I: Does limping and not being able to walk like your friends bother you? C: It worries me a lot, some people stare at me, and kids laughs It’s okay when people look at my leg.  It’s okay if people ask me about my leg.  4.4 Discussion The development of items for LIMB-Q Kids required a careful and creative synthesis of knowledge gained from direct input from the target patient population, clinical experts and experts in PROM development, while closely following the available item development guidelines. We developed items for 10 independently functioning scales, including physical function, leg symptoms, hip symptoms, knee symptoms, foot symptoms, leg-related distress, appearance, school function, social function, and psychological function. Version 1 of LIMB-Q Kids had 124 items. This version was used for the cognitive debriefing interviews with patients with lower limb deformities and to obtain expert feedback (Chapter 5).   While writing the items for LIMB-Q Kids, we applied lessons learnt from the development of other similar PROMs for children such as the Q-Portfolio instruments (CLEFT-Q, FACE-Q, ACNE-Q, LIMB-Q Adults, EAR- Q and WOUND-Q [176,182–184]. These PROMs have been developed to measure what matters most to the patients following a rigorous PROM development methodology and engaging the target patient population at all steps of development [177,183–185]. While writing items for the School Function, Social Function and Psychological  68 Function scales for LIMB-Q Kids, we used the wording of the items for similar concepts in CLEFT-Q. These items have already been rigorously tested during cognitive debriefing interviews with children as young as 8 years of age and field-tested in an international sample of 2434 patients with cleft lip and palate from 12 countries [176,177]. Choosing a recall period of 1 week is supported by the US FDA’s recommendations for a shorter recall period [48] and a clinical rationale, as well as the fact that this recall period was tested in PROMs validated by our team in large international samples of children within the same age range [176,177].  Two other condition-specific questionnaires that have been used previously in children are the Limb Lengthening Satisfaction Questionnaire (LLSQ) and the Leg Length Discrepancy questionnaire [45,124]. While these questionnaires were reviewed earlier in chapter 2 as part of the systematic review findings, we describe them again here to contrast their content with that of LIMB-Q Kids given that these questionnaires are the closest in comparison. Both of these questionnaires were developed using input from parents and clinical experts. No attempt was made to engage patients in the development of items for these questionnaires. The LLSQ is only applicable to children with lower limb deficiencies while the Leg Length Discrepancy questionnaire is only applicable to children with leg length discrepancy. There are no items in these questionnaires focusing on children with angular and rotational deformities of the lower limb. The child version of the LLSQ has 5 items with a visual analogue scale that ask about the appearance of the leg, length of the leg, function of the leg, child’s ability to do things and satisfaction with the lengthening procedure. While these are all important and relevant questions to be asked, there were several other concerns that were raised by children during our qualitative interviews. Furthermore, a single item per domain that attempts to capture a broad concept may  69 be difficult to answer for younger children and may not be sufficient psychometrically. Being able to capture a wide range of concerns of children using multiple items, as opposed to one item for each domain, is important. Single items are more prone to random measurement errors which are more likely to be cancelled out with multiple items. Single items can also lead to more biases in the meaning and interpretation of the item. Multi-item scales can cover a wide range of meanings of the items to cover a full range of the construct being measured. A single item often leads to more ambiguity in its interpretation [186]. The Leg Length Discrepancy questionnaire is a 34-item questionnaire using a 5-point Likert scale. This questionnaire was developed based on a literature review, clinical input and semi-structured interviews with the parents of children with leg length discrepancy. The questionnaire focuses on parent satisfaction, the effect of leg length discrepancy, parents’ mood, concerns, and expectations with regards to the treatment. There is limited evidence in the literature around the development, content validity and other psychometric properties of this questionnaire.  There is a need for a more holistic approach to the management of lower limb deformities by assessing the impact of the deformity and its treatment on all domains of HRQL. Having a comprehensive set of independently functioning scales in a single PROM that resonates with patients is useful in both research and clinical practice. As evident from our systematic review [149] and a recent publication by Birch et al. in this field [45], clinicians often use multiple questionnaires to measure overall physical function, symptoms and treatment satisfaction. This approach can be challenging for various reasons, including requiring multiple licenses to use different questionnaires, patient and administrator burden, and not being able to assess the impact of the medical condition on other domains important to this patient population.  70 Appearance, school, social and psychological well-being are all key aspects of HRQL that are important to patients and should be measured along with physical function and symptoms, but currently are not included in other PROMs. LIMB-Q Kids will provide a comprehensive set of simple yet meaningful scales that will facilitate the collection of evidence-based outcomes data from children.   71 Chapter 5: Establishing Content Validity of LIMB-Q Kids  5.1 Introduction Content validity is the empiric evidence that demonstrates that the items and domains of a measurement   instrument are appropriate and comprehensive relative to its intended measurement concept, population, and use [48,93,187]. Once items and scales are developed, the next important step in the development of any measurement instrument is to establish its content validity. This chapter presents the methods and results used to establish content validity of LIMB-Q Kids. Guidance for establishing content validity of PROMs is outlined by the COSMIN initiative and ISPOR [94,95,102,187]. Two steps in establishing and documenting content validity of LIMB-Q Kids were: 1) initial qualitative interviews for concept elicitation (Chapter 3) and item generation (Chapter 4); and 2) cognitive debriefing interviews to receive feedback from children and expert input to revise the items (Chapter 5). The objectives of cognitive debriefing interviews are to assess a respondent’s comprehension of the items with respect to their intended meaning, comprehensiveness of the content included in the PROM and relevance of the items to the participants. This step is important for understanding a child’s perspective, thought patterns and vocabulary and identification of any format or wording problems or difficulties with the instructions, item stems, recall period, or response options  [83,95,188]. These interviews can also be useful in identifying any additional items that have not been developed based on the initial concept elicitation and item generation phase to ensure the PROM is comprehensive.   Using children as content validity experts to provide feedback on self-report instruments for children is a critical step in developing psychometrically sound measures for children [189]. Using  72 children as content validity experts aligns with the qualitative principle of member checking (inviting qualitative informants to verify a researcher’s interpretations to enhance the credibility of the results) and is an important step in establishing content validity of an instrument [189].  The aim of this study was to establish content validity of LIMB-Q Kids by conducting cognitive debriefing interviews with children with lower limb deformities, and obtaining expert input from clinicians directly involved in the care of children with lower limb deformities.  5.2 Methods Individual semi-structured interviews were conducted with children with lower limb deformities from sites in Australia, Canada and the USA. Ethics approval was obtained at the primary study site (CW15-0215 / H15-00514) and at each of the participating sites. A written informed consent was obtained from the parents and assent was obtained from the children.  Our study followed the good practices in using cognitive debriefing interviews to evaluate patient understanding of a PROM as outlined by ISPOR [94,95]. Expert input was obtained at the same time from the clinicians and allied healthcare professionals at each of the participating sites. Version 1 of LIMB-Q Kids used for cognitive debriefing interviews and expert feedback consisted of ten independently functioning scales, including Physical Function, Leg Symptoms, Hip Symptoms, Knee Symptoms, Foot Symptoms, Leg-Related Distress, Appearance, School Function, Social Function and Psychological Function. Three key concepts of content validity were evaluated, that is, comprehension, comprehensibility and relevance.   73 5.2.1 Sampling Sites were instructed to use purposive sampling to recruit a maximum variation sample of children with lower limb deformities that included patients who varied by type of lower limb deformities, treatment type, stage of treatment, age and gender. Children aged 8 to 18 years with a confirmed diagnosis of a lower limb deformity who could speak and read English were eligible. Patients with lower limb deformities along with other medical conditions which may have a confounding effect on their HRQL and conditions such as cognitive or developmental delay affecting their ability to communicate were excluded from this study. Isolated deformities of hip and foot were excluded.  5.2.2 Recruitment Recruitment of eligible participants was done by the participating sites in consultation with the primary study site. All interviews were conducted by one interviewer (HC) who was trained by a researcher with expertise in conducting cognitive debriefing interviews for PROM development (AK). Face-to-face interviews were done either in person or virtually through Zoom.  5.2.3 Interviews with children The interview process followed the approach suggested by Willis [190,191] and used a semi-structured interview guide (Appendix D, Page 219-223). At the beginning of the interview, children were made aware of their role as critical reviewers of the items rather than just responding to the items. This approach has been suggested as an important step in establishing content validity of self-report instruments for children using children as experts [189]. A ‘Think  74 Aloud’ protocol along with verbal probing was used [190]. We used a concurrent verbalisation method, whereby children were given the instructions to think-aloud while completing the items. Specific verbal probes, such as asking the respondent to reformulate an item, or to define some of the key terms in their own words were used [192]. During the interviews, children were asked to comment on the ease of completion, appropriateness of the instructions, recall period, items, and response options. They were asked to read each item out loud and explain what they understood the item was asking about. Children were then asked to explain the reasons for choosing their answer for each item. This method of interviewing allowed us to query a participant’s understanding of the item and their interpretation of the instructions and response options. If children identified certain items or words that they didn’t understand, or the interviewer noted any hesitation from the children while reading a specific word or item, the child was asked to suggest alternate wording. To assess comprehensiveness, at the end of the interview children were asked to tell us anything about their leg that was important to them but was not included in the items they reviewed. To assess relevance, children were also asked about the importance of the items to them and whether they can relate the items to their own experiences of having a lower limb deformity.   5.2.4 Expert opinions The items and scales were also shown to experts at each of the participating sites. The experts for this study were orthopaedic surgeons treating children with limb deformities and the allied health care professionals, including nurses, physiotherapists, occupational therapists, psychologists and prosthetists from the participating sites who were involved in the care of children and adolescents with lower limb deformities. All expert interviews were individual one-on-one  75 interviews either in person or virtually through Zoom. The purpose of seeking feedback from the clinicians and the allied health care professionals was to make sure that we had not missed any clinically important items. All experts were asked to comment on the comprehensibility of the instructions, items and response options. They were specifically asked to identify items that they thought would be difficult for their patients to understand. In addition, they were asked to suggest alternate wording for any items they identified as including words ‘not commonly used’ by their patients. They were also asked about any items that they thought were not relevant for this patient population and could be potentially deleted. The experts were also asked to suggest any new items relevant for this patient population and those they think are important to be included in the scales. Finally, they were asked whether they thought these items would be able to measure change before and after treatments that they provide.  5.2.5 Analysis All child and expert interviews were recorded and transcribed verbatim. Analyses were conducted using the reparative approach representing the ‘Inspect and Repair’ model [191]. This approach included closely examining the transcribed interviews and summarising edits after each interview. Cognitive debriefing interviews and expert input was conducted in rounds. The number of patients and experts during each round varied and was based on recruitment and scheduling at each site. A summary of feedback from patients and experts was prepared at the end of each round and discussed with the three members of the research team and used to make changes to the scales. The research team members included an orthopaedic surgeon with expertise in treating children with lower limb deformities (AC) and experts in PROM  76 development and HRQL (AK, JK). In an iterative manner, LIMB-Q Kids was revised through feedback until content validity was achieved.  5.3 Results Forty patients and experts reviewed LIMB-Q Kids between December 2019 and January 2021 (Tables 7, 8, Pages 129, 130). Mean age of 17 children at the time of cognitive debriefing interviews was 13 years (range 8 to 17 years). Nineteen experts and 4 parents provided feedback on the draft of LIMB-Q Kids (Table 8, Page 130). The largest group of experts were orthopaedic surgeons (39%) and half of the experts were from Canada (57%). Though cognitive debriefing interviews were conducted to receive feedback from children only, four parents volunteered to provide feedback as well.  Five rounds of interviews were conducted with children and experts (Table 9, Page 131). Table 10 (Pages 132-135) shows the number of changes made during each round for each of the scales. Overall 37 new items were added in all 5 rounds. Tables 11-18 (Pages 136-150) summarise the evidence used to retain, revise, add, and drop items from each scale.  Version 1 of Limb-Q Kids had 10 scales and 124 total items. The Physical Function Scale had 28 items. The leg, hip, knee and foot symptom scales had 7, 5, 5, and 6 items, respectively. The leg-related distress, appearance, school, social function and psychological function scales had 15, 18, 17, 13 and 10 items, respectively.    77 5.3.1 Summary of cognitive debriefing interviews and expert feedback After each round of interviews with the patients and expert feedback, a detailed consultation was done with three members of the research team, an orthopaedic surgeon specialising in treating children with lower limb deformities and experts in PROM development and HRQL (Table 10, Pages 132-135).  Version 1: In round 1, cognitive debriefing interviews were conducted with 5 patients with lower limb deformities and feedback interviews with 3 clinical experts. Overall, 91% of the items remained unchanged after the first round of interviews. Five new items were added. By the end of round 1, LIMB-Q Kids consisted of 129 items.  Version 2: In round 2, version 2 of LIMB-Q Kids was shown to 8 children, 5 clinical experts and 1 parent. Eighty two percent of the items remained unchanged after the second round of interviews. Twelve new items were added and 4 items were dropped. A new scale, the Ankle Symptoms Scale with 8 items was added after round 2 as suggested by the experts. By the end of round 2, LIMB-Q Kids consisted of 145 items.  Version 3: In round 3, 2 patients and 9 experts were interviewed. Ninety percent of the items remained unchanged after round 3. Seventeen items were added and 7 items were dropped. Out of the 17 items added, 15 were for the individual symptom scales. 14 out of 15 edits required during this round were small changes to simplify the items further and reduce the F-K grade level. At the end of round 3, LIMB-Q Kids consisted of 156 items.   78 Version 4: Round 4 included 3 patients with amputations, 3 parents of children with amputations, and 7 experts. Patients, parents and experts only reviewed the Leg Symptoms Scale for any additional items that need to be added for children with amputations. Three items were added to the Leg Symptom Scale at this time.   Version 5: During round 5, the final version of LIMB-Q Kids had 159 items and was tested in one patient. No further changes were required based on patient feedback at this time.   5.3.2 Response options All participants, including the youngest child who was 8 years old, were able to understand the response options very well. I: Can you tell me what do you understand from these response options or answers?  Patient: I have a little trouble doing this saying that you still need the persons help but you don’t need it that much but you still have a little trouble and then I can do this is you can perfectly do it with no body’s help. (9-year-old patient with hemi-hypertrophy and leg length discrepancy)  There were no changes made to the response options for all scales in the final version of LIMB-Q Kids. As suggested by 1 expert during round 2, we replaced the response options for the symptoms scales from frequency (Always, Often, Sometimes, Never) to severity (Not at all, A little bit, Quite a bit, Very much). However, upon further probing during round 3 of interviews, it was found that frequency response options were preferable. Response options were changed back to a frequency scale in the final version of LIMB-Q Kids.  Physiotherapist: I think generally from a follow up perspective we tend to ask more about um… how often, how much, more than how much. Because like um… the how often kind of gives you a better idea of how limited they would be in their function. You know because, is it like… is it like all the time then they’re probably more likely to have more functional impairment.    79 Physiotherapist: Yeah, and I was going to say the same thing. I – looking at the scale, uh, yeah, I mean knowing just a few of the patients, like the 9-year-old, that I just treated, I’m not sure that she’s going to understand – like she might just say well, you know, it happens you k now 2 times a week .That seems it’s much more easy to understand versus this where they might even know like oh well is 4 times a week quite a bit or is it… you know?   Surgeon: …I think they will relate more closely to frequency. Yeah, I find that for example when patients give me back a PROM, I usually do it on paper just because it’s easier….., these types of questions can cause some confusion like sort of question on it, like I’m not sure I understand, or they’ll draw an arrow and make it sort of in between two of them because it’s much but it’s not very much so yeah, definitely frequency would be more objective from a patients point of view.   5.3.3 Recall period There were no changes made to the recall periods for any of the scales. The recall period of one week was found appropriate for all scales except for the Appearance Scale where the patients were asked to think about their appearance now. As evident from the interviews with the children, none of them had any difficulty in thinking about the items during the past week.  No specific concerns were raised about the recall period for the appearance scale.  5.3.4 Physical Function Scale  This scale consists of items measuring the physical function required for day- to- day activities of daily life, sports and recreation.   Instructions: Instructions were edited after round 1 and round 2. One out of 5 children (a 9-year old) interviewed during round 1 needed help to understand the words ‘past week’ (line 2). No edits were made after round 1. Probing was done during round 2 and 7/8 participants understood these words. Hence, no edits were made to this instruction. Instruction line 3 ‘Do you have problems with both legs? If yes, please answer thinking of the leg that is worse’ was edited to  80 ‘Do you have problems with both legs? If yes, please answer thinking of the leg that causes more problems’.  Patient: I don’t want to say worse leg, because it isn’t worse but like the one that had a little more problems. (12-year-old patient with fibular hemimelia)  After round 2, this instruction was edited to 'These questions ask about the leg you are seeing the doctor or nurse about'. These edits were done to avoid any confusion as to which leg is causing more problems in patients with bilateral lower limb deformities. One interviewed parent did not like calling it the leg that causes more problems. Experts also suggested making this instruction more clear to avoid any confusion. Parent: It’s like if we frame their leg that is theirs and they’re answering questions about their leg and they’re hearing that their leg is a problem. It’s not the kindest way that we can describe their physical challenge.   The instructions were changed based on patients and expert feedback. After round 1, the following instruction was added: ‘If you use a special shoe, shoe-lift, splint, prosthesis and any other special device or have a frame on, please answer thinking about how your leg feels with those devices on’. However after round 2, this was modified to ‘If you wear a special shoe, shoe-lift or brace or have an artificial leg (prosthetic leg), please answer the questions thinking of when you have them on’. The words ‘devices’ and ‘prosthesis’ were found to be difficult to understand. Other words such as special shoe, shoe-lift and prosthetic leg were found familiar. The phrase ‘artificial leg’ was suggested instead of ‘prosthesis’ by an interviewed child.  Patient: Maybe instead of prosthesis because it’s definitely harder to say. Maybe like prosthetic or maybe artificial leg. Yeah either of those would be better. I feel like that one’s kind of easier to understand. (13-year-old patient with leg length discrepancy and dysplastic femoral condyle)  No further edits were made to the instructions after round 3.   81 Items: This scale had 28 items at the beginning of the cognitive debriefing interviews and 32 items in its final version. For this scale, 1 item was revised after round 1, 5 after round 2 and 9 items were revised after round 3 of interviews and expert feedback. The number of items retained, revised, dropped or added during each round is presented in Table 10 (Pages 132-135). Details of the revisions to all items and evidence used to retain, revise, add, and drop items in the Physical Function Scale are presented in Table 11 (Pages 136- 138).   Overall, 16 out of 28 initial items remained unchanged throughout all five rounds (items 1, 2, 3, 6, 13, 14, 18, 19, 22, 24, 25, 26, 27, 28, 29 and 30). (Please refer to the final version of LIMB-Q Kids in Appendix D, Pages 224-236). After round 3, 9 items were slightly edited to simplify the item and reduce the F-K grade level (Items 7, 9, 10, 11, 12, 15, 16, 17, and 23).   Item 5 ‘… sit cross-legged on the floor’ was edited during round 2 from ‘…sit cross-legged’ to 'sit cross-legged on the floor' as clarification was sought by one participant. Item 7 ‘lift one leg while you stand on the other’ was revised three times to get to its current format. It was edited from ‘lift your leg (e.g. to kick a ball) to ‘lift your leg while standing' after round 1. This item was further edited to ‘lift one leg while standing on the other’ during round 2. It was further edited slightly after the third round of interviews to achieve a lower F-K grade level (from 2.2 to 1.0). Examples of patient quotes supporting the edits to this item are shown below.   Patient: If it was just lift your leg everybody would understand, like can you just lift your leg? If you’re asking somebody of younger ages it would kinda be uh kick a ball it would be like two things so I think if you just left it as lift your leg they would more understand it.(12-year-old patient with fibular hemimelia)  Patient: Just lifting my leg yeah but kicking a ball to me implies force like bringing my leg back and then swinging it forward a little bit.(16-year-old patient with hemi-hypertrophy)  82 No new items were suggested by the children or experts for the Physical Function Scale during round 1. Interviewer: Ok so these 28 questions were about the things you can do with your leg. Do you think anything that you think is important in terms of your physical activity with your leg that we haven’t included here? You can take a moment to think about it. Patient: yeah I think it’s pretty comprehensive, I can’t think of anything else. (17-year-old patient with leg length discrepancy, congenital posteromedial tibial bowing)  Interviewer: Which one of these questions do you think, um, resonate with your own experience that you have experienced those problems in your day to day activities. Because some, not all the questions will be relevant to you. But if you have to think of few questions, which ones do you think are relevant to you? Patient: Uh, well all of these do affect me, and quite um, so in the past week I couldn’t do a lot of that. I think they’re all quite relevant. (17–year-old patient with fibular hemimelia and leg length discrepancy)  However, items 31 ‘jump up and down on one leg’ and 32 ‘stand on one leg’ were added after round 2 and round 3, as suggested by experts, to better capture the concept of ‘balance’. Following round 3, one new item was added (item 4) to capture the physical function required for using the toilet: ‘Are you able to sit on a toilet?’  5.3.5 Leg Symptoms Scale  This scale consists of items measuring symptoms in the leg.   Instructions: Based on the feedback for the Physical Function Scale, instruction lines 2 and 3 were edited after rounds 1 and 2 for all symptom scales.  Items: This scale had 7 items in version 1 and 14 items in its final version. For this scale, 1 item was revised after round 1, 3 after round 2 and no items were revised after round 3 of interviews and expert input. Overall 4 out of 7 items remained unchanged throughout all 3 round (items 4, 5, 6 and7). The number of items retained, revised, dropped or added during each round is  83 presented in Table 10 (Pages 132-135). Details of the edits to all items and evidence used to retain, revise, add, and drop items for the Leg Symptom Scale are presented in Table 12 (Pages 139-140). Below are selected examples of the edits and quotes from the patients, parents and clinical experts.  Item 11 was edited twice. After round 1, this item was changed from 'My leg is puffy or swollen (bigger than normal)’ to 'My leg is swollen' as 1 patient asked for clarification on whether its comparison to the other leg or another person's leg. The word 'puffy' was removed to test whether one word, 'swollen', would be sufficient for patients to understand the item. After round 2, the word 'puffy' was added again, as suggested by experts. One patient was not familiar with the word ‘swollen’ but understood the word 'puffy' when probed. Patient: ok so is it talking about if your leg has gotten more swollen in the past week or is it more swollen compared to the other leg? (17-year-old patient with leg length discrepancy, congenital posteromedial tibial bowing)  Four new items (items 2, 3, 9 and 10) were added as suggested by the clinical experts ‘My leg hurts when I sleep’, ‘My leg hurts when I stand for a long time’, ‘My leg feels numb (cannot feel it)’ and ‘My leg feels tingly (pins and needles feeling).  Surgeon: I mean, you know, you don’t talk about, like, uh, like a sensory deficit type of thing. Like – like, “I feel pins and needles in my legs.”  During round 4, additional input was sought from patients with amputations, their parents and experts on the leg symptoms scale. Three additional items were added at this time. These items were: ‘My leg hurts when I touch it’; ‘My leg is itchy’ and ‘My leg has a rash (looks red)’.   84 5.3.6 Hip Symptoms Scale   This scale consists of items measuring symptoms in the hip.  Instructions: Line 2 of the instructions was edited similar to other symptom scales. After round 2, instruction line 3 was edited from ‘Do you have problems with both hips? If yes, please answer thinking of the hip that is causing more problems’ to ‘These questions ask about the hip that is part of the leg that you are seeing the doctor or nurse about’. This change was made to make it clear which hip the children are supposed to think about.  Items: This scale had 5 items in version 1 and 10 items in its final version. Three items were revised after round 1, 2 after round 2 and no items were revised after round 3 of interviews and expert input. The number of items retained, revised, dropped or added during each round is presented in Table 10 (Page 132-135). Details of the edits to all items and evidence used to retain, revise, add, and drop Items in the Hip Symptom Scale are presented in Table 13 (Pages 141-142). Below are some examples of edits and supporting quotes.   Item 1 was edited from' My hip hurts' to 'My hip hurts when I am resting or relaxing' as suggested by the clinical experts to separate the hip symptoms while resting, walking and running. This item was further edited after round 2 to ' My hip hurts when I rest or relax'.  Item 9 went through revisions twice. After round 1, this item was edited from ‘My hip locks (gets stuck)' to 'My hip gets stuck'. The word ‘locking’ was understood as ‘stopping’ or ‘being stuck’. This item was further edited after round 2 to ' My hip gets stuck (not able to move it)' as described by a patient.   85 Patient: My hip locks, gets stuck what I understand from this is like I guess also movement you can feel your hip like lock likes get stuck in this area or whatever.(12-year-old patient with fibular hemimelia and leg length discrepancy)  Interviewer: ok so what do you understand by locks? Patient: um like locking, like you can’t move it, like it temporarily gets stuck. (17-year-old patient with leg length discrepancy, congenital posteromedial tibial bowing)  Two items on hip symptoms, asking about ‘Clicking’ and ‘Popping’, were consolidated to one item since they were described as similar symptoms by some participants. Clinical experts also suggested consolidating these items as the symptoms are very similar clinically. Clicking or popping with pain was suggested to be a clinically relevant symptom by the experts. The final edited item 8 reads ‘My hip hurts when it makes a sound (clicks or pops)’. Surgeon: Clicking and popping would be very hard to differentiate.   Five additional items were included: ‘My hip hurts when I sit for a long time’, ‘My hip feels weak (shaky)’, ‘My hip hurts when I stand for a long time’, ‘My hip hurts when I walk up the stairs’ and ‘My hip hurts when I walk down the stairs’. Surgeon: It would be just like the sitting with a severe hip deformity, um… Surgeon: Sitting at school or sitting for specific tasks. And it’s not like that just popped up in my mind because so many of the parents are happy because the kids aren’t complaining when they get home from school. And they’re not getting phone calls from the teachers saying, “Oh your kid can’t even sit straight, you gotta get this hip sorted out”.   5.3.7 Knee Symptoms Scale  This scale consists of items measuring symptoms in the knee.   Instructions: Revisions to the instructions were similar to those in the Hip Symptoms Scale. Line 3 of the instructions was edited to ‘These questions ask about the knee that is part of the leg that you are seeing the doctor or nurse about’.   86 Items: This scale had 5 items in version 1 and 13 items in its final version. Three items were revised after round 1, 4 after round 2 and no items were revised after round 3 of interviews and expert input. The number of items retained, revised, dropped or added during each round is presented in Table 10 (Page 132-135). Details of the edits to all items and evidence used to retain, revise, add, and drop items in the Knee Symptom Scale are presented in Table 14 (Pages 143-144). Below we show examples of revisions and patient quotes, and list new items that were added following the interviews.   Item 11 ‘My knee feels wobbly (like it is going to give out)’ was modified twice. After round 1, this item was edited from 'My knee gives way when I move (feels wobbly) to 'My knee feels wobbly'. Patients related more to word 'wobbly' than ' gives way'. This item was further edited after round 2 to 'My knee feels wobbly (like it is going to give out)' as suggested by the experts. The item in its current form was understood by 2 patients and approved by 9 experts in round 3. Patient: Um, I don’t know, I am not sure if a younger kid would understand gives way that might be hard to understand, so that might be easier for them to understand like my knee feels wobbly.  (17-year-old patient with leg length discrepancy, congenital posteromedial tibial bowing) Patient: My knee gives way when I move, meaning feeling wobbly. Interviewer: so what do you understand with this question? Patient: it’s like when you’re walking and your knee suddenly turns wobbly. (9-year-old patient with hemi-hypertrophy and leg length discrepancy)  The following new items were added as suggested by clinical experts and a parent: ‘My knee feels weak (shaky)’, ‘My knee is swollen or puffy’, ‘My knee hurts when I bend it’, ‘My knee hurts when I stand for a long time’. ‘My knee hurts when I walk up the stairs’, ‘My knee hurts when I walk down the stairs’ and ‘My knee hurts when I straighten it’.  Parent: How does your knee feel? Maybe asking them if their knee hurts when they bend it   87 5.3.8 Ankle Symptoms Scale  This scale consists of items measuring symptoms in the ankle. As suggested by the clinical experts, a separate scale for ankle symptoms was added after round 2 of interviews and expert feedback.   Instructions: Instructions for this scale were kept similar to other symptom scales.  Items: This scale had 10 items in its final version. The 8 items added for this scale were similar in wording to the other symptom scales. These items were tested in round 3. Two new items were added to the ankle symptoms scale based on the interviews and expert feedback in round 3: ‘My ankle hurts when I stand for a long time’ and ‘My ankle hurts when I walk on a bumpy road’. Details of the edits to all items and evidence used to retain, revise, add and drop items in the Ankle Symptom Scale are presented in Table 15 (Pages 145).  5.3.9 Foot Symptoms Scale  This scale consists of items measuring symptoms in the foot.  Instructions: The instructions were revised in a way similar to other symptom scales. Line 3 of the instructions was edited to ‘These questions ask about the foot on the leg that you are seeing the doctor or nurse about’.   Items: This scale had 6 items in version 1 and 9 items in its final version. Two items were revised after round 1, 2 after round 2 and no items were revised after round 3 of interviews and  88 expert input. The number of items retained, revised, dropped or added during each round is presented in Table 10 (Page 132-135). Details of the edits to all items and evidence used to retain, revise, add, and drop items in the Foot Symptom Scale are presented in Table 16 (Page 146). As suggested by the clinical experts, 2 items about foot getting tired were deleted after round 3 as they were not considered clinically relevant. Items around foot pain (hurting) were suggested to be more clinically relevant symptoms. Six new items were added as suggested by the clinical experts: ‘My foot hurts when I walk with my shoes on’, ‘My foot hurts when I walk bare foot (no shoes on my feet)’, ‘My foot hurts when I stand for a long time’, ‘My foot hurts when I walk on a bumpy road’, ‘My foot gets stuck (not able to move it)’, and ‘My foot feels weak (shaky)’. Surgeon: I have had patients like talk a lot about like problems getting shoes on and I don’t know if that should be included anywhere. But you know sometimes you do surgery, they swell a lot, or something like that or they just have….or even if they have a lot of deformity and then you improve it then they can get into shoes just like um   5.3.10 Leg-Related Distress Scale This scale is composed of items measuring the distress the children feel due to their leg.   Instructions: The items ask about how the children feel about their leg. After round 1, 3/5 participants were not clear that it was about how they feel about their leg. One participant suggested underlining ‘you feel’. As a result, line 1 of the instructions was edited to underline ‘you feel’ to emphasize that it is about how the person feels rather than how the leg feels (as in the symptoms scales). The final instruction read: How do you feel about your leg? Instruction lines 2 and 3 were edited similar to the Physical Function Scale.   89 Items: This scale had 15 items in version 1 and 14 items in its final version. One item was revised after round 1, 2 after round 2 and 1 after round 3 of interviews and expert input. The number of items retained, revised, dropped or added during each round is presented in Table 10 (Page 149-152). Details of the revisions to all items and evidence used to retain, revise, add, and drop items in the Leg-Related Distress Scale are presented in Table 17 (Pages 147-148). Some examples of edits supported by quotes from the patients, parents and clinical experts are shown below.  Overall, 7 out of 15 initial items remained unchanged throughout all five rounds (items 3, 4, 5, 6, 7, 9 and 12). Item 1 was edited after round 1, from 'I cover up or hide my leg when I go out' to 'I hide my leg when I go out'. Two patients in round 1 didn't understand that the item is asking about covering up the leg to hide it. Item 2 was revised from ‘I avoid wearing shorts or skirts’ to 'I avoid wearing shorts or skirts that show my leg'. This was edited as suggested by the experts to make it clearer.  Patient: I just don’t understand why someone would not wear shorts or skirts . (13-year-old patient with posteromedial tibial bowing and leg length discrepancy)  Item 10 was edited after round 3 from 'I dislike how I look when I walk (e.g. limp)' to 'I dislike how I look when I walk'. While the item was found relevant to the interviewed patients, different patients described how they walk differently. One patient described looking 'funky' not necessarily 'limping' when walking.  Patient: um I guess I look a little misaligned but don’t necessarily think I limp I just look a little funky.    90 The item ‘I feel unhappy about the scars on my leg’ was included in rounds 1 and 2 of cognitive debriefing interviews and expert input. This item was found relevant only to patients who had scars. Hence, this item was removed from this scale. It was kept as a stand-alone item to be asked only of patients who had scars due to their surgeries.  5.3.11 Appearance Scale  This scale consists of items asking about the appearance-related concerns of the children.   Instructions: After round 2 of interviews and expert feedback, line 3 of the instructions, ‘Do your legs look different from each other? If yes, please answer thinking of the leg that you like the least’ was edited to 'These questions ask about the leg you are seeing the doctor or nurse about'. Participants and experts suggested not using ‘the leg that you like the least’. Two participants couldn’t decide which leg they like the least. Line 4 of instructions, ‘If you use a special shoe, shoe-lift, splint, prosthesis and any other special device or have a frame on, please answer thinking about how your leg feels with those devices on’ was edited to ‘If you wear a special shoe, shoe-lift or brace or have an artificial leg (prosthetic leg), please answer the questions thinking of when you have them on’. Details of the revisions to all items and evidence used to retain, revise, add, and drop items in the Appearance Scale are presented in Table 18 (Pages 149-150).  Items: This scale had 18 items in version 1 and 17 items in its final version. For this scale, 4 items were revised after round 2 of interviews and expert input. Thirteen out of 18 items (1, 2, 3, 6, 7, 8, 9, 10, 11, 12, 14, 15, and 17) remained unchanged during all five rounds of interviews  91 and expert input (Please refer to the final version of LIMB-Q Kids in Appendix D, Pages 224-236).   Item 4 ‘How much do you like the size of your leg’ was edited after round 2 from 'the size of your leg (not too skinny)' to 'the size of your leg'. One parent objected on using word 'skinny'.  Parent: how much do you like your legs… the length of your legs, the size of your leg… um… get rid of “not too skinny”. I don’t like the word skinny. Girls shouldn’t be looking at that word “skinny”, you know, it’s not appropriate.   Item 13 was edited from ‘how straight your leg looks (not crooked or lopsided) to ‘how straight your leg looks’. The words 'crooked or lopsided” were removed' from the brackets as suggested by a parent and an expert. While some participants understood the item without the content in the brackets others were not familiar with the words in the brackets. It is worth mentioning that the words ‘crooked’ and ‘lopsided’ were originally taken from the qualitative interviews as used by the children during concept elicitation phase.  An item about the appearance of scars was included in rounds 1 and 2 of cognitive debriefing interviews and expert input (a similar item was initially included in the Leg-Related Distress Scale). This item was removed from this scale and kept as a stand-alone item that is only applicable to patients with scars.  5.3.12 School Function Scale This scale consists of items about the school.   Instructions: No edits were made to the instructions.  92 Items: One item was edited, one deleted and one added during all five rounds of cognitive debriefing interviews and expert input. Item 9 ‘I feel safe at school (not bullied because of my leg)’ was changed to ‘I feel safe at school’. However, it was edited again to ‘I feel safe at school (not bullied)’ to avoid any confusion with the physical safety. Item ‘I am able to attend school’ was deleted after round 2. One new item was added: ‘My teachers treat me well’. This scale has 17 items in its final version. (Please refer to the final version of LIMB-Q Kids in Appendix D, Pages 224-236).  5.3.13 Social Function Scale This scale consists of items about the social function of children.  Instructions: This scale did not require any edits to the instructions.   Items: This scale has 12 items in its final version. This scale did not require any edits to the items. After round 2, the item ‘My friends treat me the same as others’ was deleted since it was found to be similar to the item ‘People treat me the same as everyone else’. (Please refer to the final version of LIMB-Q Kids in Appendix D, Pages 224-236).  5.3.14 Psychological Function Scale  This scale has items about the psychological function of the children.  Instructions: This scale did not require any edits to the instructions.  93 Items: This scale has 10 items about the overall psychological well-being of children including confidence and self-esteem. This scale did not require any edits to the items. (Please refer to the final version of LIMB-Q Kids in Appendix D, Pages 224-236). 5.3.15 General expert feedback  Overall, the experts found the scales to be very comprehensive and the items in each scale easy to understand and relevant to this patient population. The majority of the additional items suggested were for the symptom scales. Some quotes from expert interviews are below:  Surgeon: It’s a pretty – a pretty um, detailed list. Overall, it’s very comprehensive.   Surgeon: I think these are great questions because they cover the gamut of the symptoms that I would be asking about and usually the patients come in with pain, or having weakness, or some neurologic type symptoms and pain at rest is obviously an important one too because it may point to other things so I think these are all very good questions and they’re all unders tandable too, you know putting the word shaky, cannot feel it, pins and needles, I think those are all very helpful.   Surgeon: So… let’s see… get out of bed… sit in a chair – I don’t – get up from a chair. These are all – I mean, these are all things that I’ve had people complain of, so this is good.   One surgeon specifically commented on the importance of School Function Scale. Surgeon: so yeah the school is certainly very important.  This is a challenging section for sure to put together, but I think these are all very good questions.  When specifically asked whether they think these items will be able to measure change, they all thought that most of the items will allow them to measure change when administered before and after treatment.  Interviewer: So we were talking about measuring change, right? Do you do think… that these items can measure change? Physiotherapist: Yeah, I think so. Uh it’s covering the things we like to look at, like um… yeah e they’re good functional measures.    94 Surgeon: I think, this reasonably covers most areas possible which might show a change for us in our final analysis if we do surgery or whatever treatment you do and you want to come back in two years’ time and ask the same questions, yeah.   Interviewer: Thinking about the social life and the school scale, do you think you can measure change with these items. Surgeon: I would think so yeah, because I’ve observed myself with many of my patients, I keep asking them as well, actually many of these questions I ask  especially at the end of treatment. I ask many of them what they think of themselves and what their friends think.   Physiotherapist: I think so and I think I’m hoping that it actually would be really good to pick up change because a lot of patients like maybe not prior to a procedure but even – no even prior, and then having a procedure done. The after – kind of the aftermath of it, I have a feeling this will measure change actually quite well.   Interviewer: Do you think like, these items or some of these items are able to measure change? Surgeon: Yeah, yeah I do think that there is some, you know, certain things like “bend your knees,” sometimes that’s a surgical goal right? Um… yeah, some things so those are like range of motion things, “walk without limping,” we’re often times trying to fix a limp… um, speed and distance is certainly the way a lot of people um… gauge whether their surgery has helped, um, just in a very informal way, so having it like more quantified as you have here would be helpful.   Experts were asked whether they will potentially be using this new PROM in their clinical practice. Most of them were very interested in using LIMB-Q Kids when it’s finalized and validated. Interviewer: Do you see yourself using these scales? Surgeon: oh yeah, definitely, yeah one of the complaints that I would have about a lot of the scales that I use are that they don’t capture a global picture about how this affects somebody. I really appreciate those sections in terms of quality of life and things like that because those are the ones that often children find the most like you said, the most problems they have is the distress they have because of whatever physical problem they have and it’s interesting to see both like in terms of how their functional sections look in terms of and how their quality of life section looks because it’s very commonly their function isn’t as impacted as, maybe their score isn’t very low in that section but their quality of life is terribly low and you know they worry about. So definitely for me, that’s one of the big advantages I think of having a deformity specific questionnaire, this stuff is as important as the functional stuff.   Physiotherapist: I think it’s something very useful. It’s a really interesting tool and hopefully it will change our practice too. For us to know what’s going on in their life while they are going through a treatment and before.    95 5.3.16 Readability Mean F–K readability for the 159 items was 1.04 (range, 0–5.2) (Table 19, Page 151). The readability scores for 90% of items were at or below grade 3 F-K reading level. The 10% items that were over grade 3 F-K reading level were still understood well by the patients interviewed during the cognitive debriefing interviews. 5.4 Discussion Cognitive debriefing interviews with an international sample of patients and feedback from clinical and methodological experts have established the content validity of LIMB-Q Kids. We were able to refine the instructions, response options and items for each scale. Expert feedback from a diverse group of experts involved in the care of patients with lower limb deformities helped establish the comprehensiveness of each scale and ensure the clinical relevance of the items to this patient population.   Following the COSMIN guidelines for establishing content validity of PROMs, we focused on three key components of content validity, i.e., comprehension, comprehensiveness and relevance. Items and instructions were understood by all interviewed patients after making relatively minor edits. There was a preference both from the patients and experts for a frequency scale as opposed to severity scale for the response options. Our aim was to develop items applicable to and understood by patients with lower limb deformities between the ages of 8-18 years. This presented a challenge during item writing to make sure that the items are understood by young children. The majority of the items were at F-K grade level of 3 or lower. A low grade reading level ensured the ease of reading for the children. A few items were at a higher level than grade 3 but were still understood by the interviewed children. The majority of the children did not  96 suggest any new items and found the items relevant to their experience with their lower limb deformities. Overall, the experts found the scales to be comprehensive and covering areas relevant to this patient population. Most of the new items suggested by experts were for the symptom scales. Most of the experts interviewed in this study indicated their interest in using LIMB-Q Kids, once validated, in their practice due to the lack of such a comprehensive and rigorously developed condition-specific tool.  A well-designed and rigorously conducted study for the development of a PROM can help to ensure its content validity  [101,193–195]. There are guidelines to perform concept elicitation by conducting qualitative studies to obtain patient input for good content coverage [48,95,193,196]. We conducted a systematic review and an international qualitative study for concept elicitation following the recommended guidelines [149]. Item generation was carried out following the relevant guidelines in the literature and guidance from experts in PROM development [48,94,137]. Next we conducted cognitive debriefing interviews with the target patient population and obtained feedback from clinical experts involved in the care of this target patient population. In doing so, we followed the COSMIN guidelines for establishing content validity of PROMs [102]. While some studies may compute quantitative indices to assess content validity (e.g., Content Validity Ratio or Content Validity Index) [197,198], the COSMIN and ISPOR taskforce do not recommend the use of such indices [95,102].  Based on our systematic review in 2016 and our recent literature search, there is still a lack of a rigorously developed PROM for children with lower limb deformities [149]. In the absence of condition-specific PROMs for this patient population, previous studies have used generic  97 outcome measurement tools or parent-reported measurement instruments [149]. A paper published in 2019 discusses the use of a 5-item Lower Limb Satisfaction Questionnaire for children with lower limb deformities [45]. This child version was developed from a parent-reported version of the Limb-Lengthening Satisfaction Questionnaire, which was adapted from a parent questionnaire for assessing prosthesis satisfaction in children with limb deficiencies only (CAPP-PSI) [144]. However, there are no available reports on the detailed development and validation of the child version of the Lower Limb Satisfaction Questionnaire. Hence the need for a PROM that is developed by engaging the target patient population during concept elicitation and content validation still exists.  The inclusion of an international sample of patients and experts is a strength of this study. Clinical experts from various international sites, directly involved in the care of children with lower limb deformities, were consulted for their feedback. The study team consisted of experts in PROM development and HRQL studies. All cognitive debriefing interviews with patients and interviews with experts were conducted by one interviewer to ensure consistent methodology. The detailed one-on-one interviews with the patients and clinical experts provided rich feedback for the items included in each scale.  Our study to assess content validity of LIMB-Q Kids has some limitations. The number of patients interviewed in each round was relatively small and some items that were slightly revised or added after round 3 were only tested in 1 patient. However, the last few revisions made by the team were very minor and the F-K grade reading level was low. While the cognitive debriefing interviews were not conducted with children from Ethiopia and India, inclusion of clinical experts from these countries was useful to ensure that the items reflected the perspective of  98 children in these countries. Some important points were raised by the clinical experts about the items in relation to their applicability in these countries. For example, an item in the Physical Function Scale asks if the children are able to ride a bike. The word ‘bike’ often means a motorbike or a motorcycle in India and Ethiopia. However, in Canada, the UK and the USA, ‘bike’ is a bicycle. These points were noted and need to be carefully probed for during the future translation and cultural adaptation (TCA) of LIMB-Q Kids for use in these countries.   5.5 Conclusions This study demonstrates content validity of the scales included in LIMB-Q Kids, a new PROM for children with lower limb deformities. At this stage in the development of LIMB-Q Kids, the focus was to have a comprehensive set of scales that are easily understood, comprehensive and relevant to the patient population. The next steps (phases 2 and 3) will involve a pilot field-test study at a centre in Canada to test the scales, followed by an international field test study and psychometric analysis. Scoring algorithms will be developed, following which the scale could be used to inform clinical practice and research.    99 Chapter 6: Challenges and Limitations  6.1 Challenges A major challenge in carrying out this study was the international scope of the work. With the aim to develop a PROM that is applicable internationally, the initial qualitative study for concept elicitation, the cognitive debriefing interviews and expert interviews for content validity were conducted at 8 sites, including Australia, Canada (two sites) Ethiopia, India, the UK and the USA (two sites). This involved getting ethics approvals from different research ethics boards and data transfer agreements from these sites. This process was long and there were significant delays due to different approval requirements from different countries. Conducting interviews (with both patients and clinical experts) from international sites in different time zones was also challenging. International work also presents challenges due to language barriers. A substantial amount of time was required to translate and transcribe the interviews into English for analysis. At the sites where I was not able to conduct qualitative interviews myself in a local language, training was required to ensure the local interviewer understood the purpose of the study and was familiar with the interview guide before conducting the interview.  A large portion of my thesis work was qualitative in nature. This involved initial qualitative interviews and cognitive debriefing interviews that lasted an average of 90 minutes. Recruitment for these interviews was slow as many children and their families did not want to take part in the study due to the time commitment. Scheduling one-on-one interviews with the clinical experts was also challenging. Nonetheless, one-on-one interviews with the clinical experts provided rich data and an extensive feedback on the items.  100 Conducting research with children can be challenging in a number of ways. It was difficult to develop a rapport with some children and to reduce the unequal power relations between an adult researcher and a child participant. I found this particularly challenging when conducting interviews with children in India. This difficulty could be due the researcher being considered as an outsider, and the relatively low socio-economic status of the family. However, one-on-one interviews provided rich data and offered advantages over other methods of concept elicitation, such as focus groups. Although focus groups may seem to be a faster way to obtain data, they can pose challenges whereby participants might feel shy discussing their personal experiences in a larger group. With our target participants being children, one-on-one interviewing was preferred.   The cognitive debriefing interviewing stage started in December 2019. However, due to the COVID-19 pandemic, research activities were curtailed at the primary study site in March 2020. Face-to-face cognitive debriefing interviews at the primary study site were put on hold, and ethics applications and data transfer agreements for other participating sites were stalled. To overcome these challenges, the protocol for cognitive debriefing interviews was amended to include virtual interviews. The approval of this amendment at the primary study site and other participating sites delayed the recruitment and conduct of the cognitive debriefing interviews.  6.2 Limitations When conducting research with children, especially qualitative research, it is important to incorporate child-friendly research methodologies. One such method is photo-elicitation interviews (PEI) for concept elicitation, which was initially planned to be part of my thesis.  101 However, grant funding for the study was limited and PEI would have required significant resources (time, funding and trained personnel). Participation in PEI would have also required more involvement from the patients and their families. This increased time commitment from the patients and families was also anticipated as a potential limitation to recruitment, especially in the context of a multi-centre, international study. Therefore, a decision was made to drop the PEI from the study.   There could be a sampling bias due to the choice of study sites. The selection of study sites from Australia, Canada, Ethiopia, India, the UK and the USA was based on prior collaborative relationships between members of the research team and clinicians at these sites. These existing collaborations facilitated the conduct of the study at these sites. In order to develop a PROM that is applicable to low-, middle- and high- income countries, it was important to have representation from these countries during the concept elicitation phase. The two centres from Ethiopia and India, where qualitative interviews were conducted, primarily served patients from low-socio economic status. Some of these patients had limited access to healthcare resources, which led to late diagnosis and treatment for some of the interviewed children. Hence these children might have reported a higher impact of their lower limb deformity on HRQL. Similarly, for the cognitive debriefing interviews, we were only able to perform interviews with children who spoke English, thus primarily from Australia, Canada and the USA. However, the study team plans to conduct a translation and cultural adaptation of LIMB-Q Kids for use in low-income and middle-income countries as part of the international field-test study. During the TCA, cognitive debriefing interviews will be conducted with children to ensure that LIMB-Q Kids is easy to understand from their perspective. Inclusion of children from a range of countries in the field-test  102 sample will allow us to ensure that the final set of items in each scale of LIMB-Q Kids is determined using a truly international sample. A relatively small number of patients with amputations were involved in the development of this PROM. Specifically, 5 patients with amputations were involved in the concept elicitation interviews and the cognitive debriefing interviews. While this small number of patients with amputation might have limited us in capturing all the concerns of children with amputations, clinical experts, including orthopaedic surgeons, physiotherapists, occupational therapists, nurses, and prosthetists, provided additional input to ensure relevance of the items to these children. Parents of children with amputations also provided input. An advantage of the modular approach to PROM development that our team has taken is that new concepts of interest that are not part of the initial PROM can be developed into scales to fill gaps that are identified in the future.     103 Chapter 7: Significance, Implications and Future Directions  This study fills an important gap in HRQL research by developing a PROM that measures the concerns that matter most to children with lower limb deformities. The PROM (LIMB-Q Kids) developed as part of my doctoral research (phase 1) is now ready for field-testing (phase 2) in a large sample of children with lower limb deformities from various participating countries.  Traditionally, PROMs are developed in one language and usually in one country [199]. These PROMs are later translated into other languages as needed. Very few PROMs have been designed for use in low-income and lower-middle-income countries [200]. Though there are guidelines for translation and cross cultural adaptation of PROMs [201,202], a more rigorous approach is to involve target participants from different countries of varied socio-economic status in the development and initial validation stage, as opposed to conducting translation retrospectively [203]. Developing PROMs cross-culturally can help facilitate international research and increase their uptake.   The international qualitative study for concept elicitation has also drawn attention to the impact of a lower limb deformity on HRQL of children. Sharing the results of qualitative interviews with the respective participating sites was a reminder for those sites about the challenges these children face in their day-to-day activities. The concerns of children about their appearance and the need for social and psychological support, specifically when they are going through complex surgical procedures for the treatment of their deformities were also brought to the forefront. While some sites already had existing services or pathways to refer these children to adequate services as needed, other sites became more aware of these needs. One example was the primary  104 study site at BC Children’s Hospital, where after the qualitative interviews with children and their families, the clinical team recognised the need to follow the children and their families more closely in order to refer them to the appropriate psycho-social support services as needed, especially at the time of diagnosis and during surgical procedures. While the sites from the low- and lower-middle-income countries do not have the support services readily available to these children, the study has highlighted the importance of such services.   The impact of lower limb deformities on HRQL has been studied in the past. However, none of the previous studies included an international sample of children. Our international sample of children with lower limb deformities was heterogeneous in terms of age, gender, type of deformity, stage of treatment, country of residence and socio-economic status. The concepts of interest used to create LIMB-Q Kids were identified from this diverse sample of children, which increases the generalizability of this new PROM. Appearance, physical health, psychological health, social health and school were the five themes that were important to the interviewed children with lower limb deformities. The findings from the qualitative study were similar across children from various international sites. Since the analysis was not focused on specifically looking at differences in HRQL across various participating sites, future studies in this area can focus on a country-by-country analysis.  By looking at HRQL in an international sample, this study was also able to draw attention to how socio-economic status and access to healthcare can impact HRQL in low-income and lower-middle-income countries. Low socio-economic status directly translates to limited access to healthcare, including timely diagnosis, treatment, and access to auxiliary services, such as  105 occupational therapy, physiotherapy, nursing and psychological support, all of which are crucial for children with lower limb deformities.   Certain themes were under-represented in the literature when looking at the impact of lower limb deformities in children. For example, leg appearance emerged as an important theme based on the qualitative interviews. Interviewed children were concerned about the appearance of their legs, hips, knees and feet due to the deformity, as well as their overall appearance due to treatment-related devices, such as the external fixator devices, shoe-lifts, splints and braces. As a result, appearance is now a separate theme in our conceptual framework and this has led to the development of a separate scale for this concept. This scale can be used to assess the impact of lower limb deformities on the perception of appearance among children at various stages of treatment and to evaluate if and how different treatments may improve appearance in children with deformities. Understanding a child’s perception of their appearance with their deformity can also help patients, families and clinicians in shared decision making in which appropriate consideration is given to what is most important to a child with lower limb deformities.   Similarly, school function was a major theme based on the qualitative interviews. As a result, we developed a separate scale for school function. Our target patient population were children between the ages of 8 and 18 years, who typically go to school. Hence, it is not surprising that this was a major theme in the qualitative interviews. Children discussed their challenges regarding participation in school activities at various levels, including physical and social activities. Participation varied depending upon the individual child and also the country of residence. Some children from Ethiopia were unable to attend school for a long period of time  106 due to limited access to treatment and their family’s low socio-economic status. Nonetheless, school was a common theme for the interviewed children across all the sites. Findings of lower participation at school and missed days of school among children with lower limb deformities are consistent with previous studies.  There are important clinical implications of this work. The PROM developed as the end product of the larger project will help assess HRQL of children with lower limb deformities and will potentially benefit future patients with these deformities by helping measure their health outcomes. In the long term, LIMB-Q Kids could be used by health care professionals to look at the effectiveness of relevant interventions for these children in terms of their HRQL. This process will eventually help develop or modify interventions/treatment plans, health care services and target care, to achieve a better HRQL for children with lower limb deformities. LIMB-Q Kids can also help future patients in making important decisions about treatment choices, by comparing HRQL between different interventions (such as amputation versus reconstruction) and comparing HRQL pre and post-intervention.   Beyond the clinical contributions of this research, there are also methodological contributions. There is still a paucity of condition-specific PROMs in the field of pediatric orthopaedics. This research may serve as an example of how to use qualitative methods in the development of PROMs for a pediatric patient population. Patients with a wide range of orthopaedic conditions leading to lower limb deformities were interviewed in the development of LIMB-Q Kids. Some scales from LIMB-Q Kids may be applicable to other orthopaedic conditions (after some testing and validation studies) without going through the time-consuming process of concept elicitation.  107 Examples of such conditions include hip disorder like Legg-Calve-Perthes disease or slipped capital femoral epiphysis (SCFE) and specific pediatric deformity conditions, for example, Blount’s disease. The scales included in LIMB-Q Kids are independently functioning scales which, upon full validation, will provide the users with the flexibility to use individual scales as per their clinical and research needs. Due to the lack of condition-specific PROMs, generic measures or PROMs developed for adults are used in children with specific medical conditions. Although generic instruments allow for comparisons between the impacts of different diseases or between healthy and diseased populations, they may fail to ask about issues specific to children with orthopaedic conditions. The development of internationally applicable PROM following standard guidelines is a resource- and time-intensive task. Nonetheless, there is great value in having a PROM that measures what matters most to the target patient population across different cultures and levels of socio-economic development. Rigorously developed PROMs with established content validity have the potential for improving overall outcome assessment and facilitating shared decision-making between patients, families, and clinicians. Furthermore, current surgical practice in pediatric orthopaedics is based on poor quality evidence [204–207]. Hence, there is clearly a need to improve the quality of evidence about various surgical procedures using PROMs that focus on what’s important to the patients.  There is limited data available on the cost-effectiveness of amputation versus reconstruction procedures for children with limb deformities. Availability of a rigorously developed and validated condition-specific PROM for children with these conditions will also allow for cost utility analysis. This will further help develop evidence supporting the treatment choices for patients, their families, and clinicians.   108 Future directions for this research project are to conduct a pilot field-test in Canada followed by an international field-test study and psychometric evaluation of LIMB-Q Kids. Translation and cross cultural adaptation will be done to make the PROM suitable for non-English speaking sites so they can take part in the field-test study. Data will be collected from an international sample of patients. Item reduction will be based on the field-test data to remove items with poor psychometric performance and to reduce respondent burden. It is especially important to keep the length of PROMs manageable specifically for children since they get distracted and lose attention quickly [136]. Longer PROMs tend to increase the chances of children not answering all the items, skipping items or not reading the items carefully, which can lead to unreliable results. In conclusion, my thesis involved a rigorous approach to completing phase 1 of the development of a new cross-culturally relevant PROM called LIMB-Q Kids. My thesis also establishes content validity of this instrument. When validated, LIMB-Q Kids will be useful for informing both research and clinical practice.    109 Tables and figures  110  Table 1: Characteristics of the studies included in the systematic review Author Year Country Study Design Type of deformity Gender (%) Age range (Years) Sample size Type of Intervention Type of comparison sample  Average Length of follow-up (months) PRO  used Bond 1999 England Prospective observational study Short stature (13/53); other congenital or acquired deformity (40/53) Not given 6 -17 53 Not applicable Normative data Not applicable CDI-S, CFSEI,  STAI, STAIC, PARS -III, FES Ghoneem 1996 Canada Prospective cohort study 30 congenital, 15 acquired (tibia: 29; femur:23; foot:12) Male (26/45) Female (19/45) 3 - 18 45 Deformity correction by Ilizarov Not applicable 36 PHBQ,  CDI, Not validated In House Satisfaction Questionnaire Kim 2012  South Korea Retrospective Review with Prospective assessment of QOL Achondroplasia Male (7/22) Female (15/22) 8 - 25 22 Bilateral lower limb lengthening using Ilizarov Not applicable 58.8 AAOS Lower limb score, SF-36, Rosenberg self-esteem questionnaire, PedsQL Lee 2012 South Korea Prospective observational study Post traumatic or idiopathic leg length discrepancy Male (35/58) Female (23/58) 6 - 20 58 Not applicable Not applicable Not applicable PODCI, In-house  parental questionnaire Martin 2003 England Prospective longitudinal study Lower limb deformity Male (9/15) Female (6/15) 11 - 18 15 Ilizarov leg lengthening Not applicable 1 month post frame removal OSIQ, CDI, Kidcope, Interview Michielson 2011 Netherlands Cross sectional study Congenital lower limb deficiency (LLD) Male (34/56) Female (22/56) 8 - 18 56 Not applicable 158 Dutch children, aged 6 to 18 years (mean age 12 years; standard deviation (SD) 3), recruited from 4 mainstream schools in the Netherlands Not applicable CAPE, Kidscreen Montpetit 2009 Canada Case series Leg length discrepancy, angular deformity, or Male (37/52) Female (15/52) 5 - 21 52 External fixation, Botulinum toxin-A norms of healthy children 3 months post frame removal PedsQL  111 Author Year Country Study Design Type of deformity Gender (%) Age range (Years) Sample size Type of Intervention Type of comparison sample  Average Length of follow-up (months) PRO  used clubfoot Niemela 2008 Sweden Prospective Study Leg length discrepancy Not reported 7 - 15 30 Ilizarov leg lengthening Patients with prominent ears; Age and gender-matched control group 12 months post surgery RCMAS, I think I am ,  CDI, SPIQ, Youth self-report, CBCL, STAI (X) Ramaker 2000 Sweden Prospective Study Leg length discrepancy (17 congenital, 5 infection, 3 fracture, 1 unknown) Male (14/26) Female (12/26) 6 - 17 26 Ilizarov leg lengthening Not applicable 16-67 months post surgery Dutch self-inventory for depression assessment, Zung Depression Scale for adolescents, Spielberger test for State and Trait  anxiety, Not validated in house questionnaire. Vitale 2006 USA Prospective Study Leg Length discrepancy Male (49/76)  Female (27/76) 5 - 18 76 Not applicable Published norms from CHQ Not applicable CHQ PF-28     112  Table 2: Patient- and proxy-reported outcome measures from studies included in the systematic review  Constructs Measured No. of studies that used this Instrument Name of instrument Type of report Physical Function Pain Gen. Health Social Function School Family Peer relation/friends Beha-viour Cog-nition Co-ping Emo-tions Psycholo-gical distress Self-esteem Satisfac-tion with appear-ance Treatm-ent Expect-ations AAOS LL outcomes [128] Patient + +              1 CBCL[127] Parent +  + + +   + +   +    1 CHQ[125] Parent + + + +  +  +    + +   1 CAPE[66] Patient and parent +   +     +       1 CDI[26,105,112,127] Patient            +    4 CFSEI-2 Form B[112] Patient             +   1 Dutch self-report inventory for children[27] Patient            +    1 FES[112] Parent      +          1 SPIQ[127] Patient         +       1 I think I am[127] Patient             +   1 Kidcope[26] Patient          +      1 KIDSCREEN[66] Patient and parent +   + + + +    + + +   1 Offer Self-Image Scale (OSIQ)[66] Patient              +  1 PODCI[124] Parent + +         +   + + 2 PedsQL[66,12] Patient (34), Patient and (38) +   +  +   +  +     2 PARS III[112] Parent    +   + +    +    1 Post- Parent        +        1  113  Constructs Measured No. of studies that used this Instrument Name of instrument Type of report Physical Function Pain Gen. Health Social Function School Family Peer relation/friends Beha-viour Cog-nition Co-ping Emo-tions Psycholo-gical distress Self-esteem Satisfac-tion with appear-ance Treatm-ent Expect-ations Hospitalization Behavior Questionnaire (PHBQ)[105] Revised Children's Manifest Anxiety Scale (RCMAS)[127] Patient            +    1 Rosenberg[126] Patient             +   1 SF-36[126] Patient + + + +        +    4 Specific LLD questionnaire[124] Parent + +    +     +  +   1 STAI[112,127] Patient            +    2 Youth Self-Report[127] Patient   + +    + +   +    1 Zung Depression Scale[126] Patient            +    1 No. of instruments measuring this sub-concept N/A 9 5 4 8 2 5 2 5 5 1 4 11 6 2 1   114 Table 3: Determinants of quality of life from studies included in the systematic review Determinant Construct where relationship with determinant was statistically significant  Severity of deformity Social [125] Complications post-surgery Psychological [126]  Type of deformity Physical [124] Social Health [125] Stage of treatment Physical, Social, Psychological [27] Psychological [72] Type of treatment Physical, Psychological health [126]       115 Table 4: Demographic characteristics of patients participating in qualitative interviews  Canada - 1 Canada -2 USA India Ethiopia Total Interviews (parent and patient) Total 16 10 21 20 12 79 Interviews Patients 7 5 11 10 6 39        Congenital Lower Limb Deformity 6 5 11 7 5 34 Acquired Lower Limb Deformity 1 0 0 3 1 5        Average Age (in years for patients) 13.7 11 12.3 13.7 13 12.9 Age Range (in years for patients) 11 to 18 8 to 18 9 to 16 11 to 18 10 to 17 8 to 18        Gender       Female 2 2 7 2 2 15 Male 5 3 4 8 4 24 Interview in English       Yes 16 0 21 0 0 37 No 0 10 0 20 12 42     116 Table 5: Lower limb deformities of patients participating in qualitative interviews  Acquired genu valgum post treatment of Ewing's Sarcoma, leg length discrepancy due to growth disturbance due to 8 plates for treatment of genu valgum  Acquired leg length discrepancy secondary to a tibial fracture  Congenital A/K amputation  Congenital femoral deficiency  Fibular hemimelia  Congenital pseudarthrosis of tibia  Genu valgum  Infantile Blount’s Disease  Infection induced proximal tibial growth plate disturbance, proximal tibia vara, shortening and recurvatum deformities  Leg length discrepancy secondary to congenital posterior medial tibial bowing  Leg length discrepancy, Perthes  Leg length discrepancy, osteogenesis imperfecta  Leg length discrepancy secondary to left hemi-hypertrophy  Leg length discrepancy, post traumatic avascular necrosis of hip  Leg length discrepancy, arm deformities secondary to meningococcemia  Leg length discrepancy acquired due to post traumatic malunion of femur  Leg length discrepancy secondary to septic hip, osteomyelitis, avascular necrosis of hip  Leg length discrepancy  Leg length discrepancy, osteoporosis  Leg length discrepancy, osteogenesis imperfecta, osteoporosis  Leg length discrepancy, neuroblastoma at birth  Leg length discrepancy, congenital patella dislocation  Tibial deficiency (Gallop-wolfgang complex)  Right femoral hypoplasia  Right hemihypertrophy, KT syndrome  Right external tibial torsion, miserable mal-alignment syndrome, osteochondritis dissecans  Rickets, severe bilateral leg deformities  Proximal femoral focal deficiency  Post traumatic partial distal tibial physeal closure with severe deformity of the ankle   117  Table 6: Participant quotes from qualitative interviews Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme India Male  15 1 C: I didn’t like how my leg was not straight and my knee was popping out. Appearance Leg - shape US Female 14 2 C: My one leg was longer than the other. Appearance Leg - size Ethiopia Female 17 3 C: It means by nature, my right leg was bent in ward and the left one doesn’t even touch the ground, it was short. Appearance Leg - symmetry Canada Female 11 4 P: something that really bothers me is.., because her left leg is so much shorter than her right, P: we have to sometimes get a lift in her shoe.  Appearance Leg - symmetry US Female 14 5 C: Because my leg would look bent in pictures. Appearance Leg - photos Canada Male  14 6 C: I don’t like how it looks coz it just looks weird. Appearance Leg - Overall US Female 16 7 C: They would only notice how my hips were different. One was like more shaped and the other one was higher, like kind of like a balance. Appearance Hip India Male  15 8 C: I didn’t like how my leg was not straight and my knee was popping out. Appearance Knee Ethiopia Male  13 9 C: I don't like my foot because it is bent inward.  Appearance Foot India Female 11 10 C: I just want them to disappear soon. They look bad. Appearance Scars Canada Female 13 11 P: She does have some quite noticeable scarring on her lower left leg. And in like 2 or 3 years, if there is still a lot of scarring, there is some kind of post-operative thing that they can do to kind of reduce the appearance of the scar. Appearance Scars Canada Female 8 12 C: I cannot wear short dresses and shorts in the summer. Appearance Clothes - Type Canada Male  13 13 P: Right now, he doesn’t really enjoy wearing the shorts and all the people to see the frame. Appearance Clothes - Type US Male  16 14 C: I have to wear either stretchy pants or shorts when I am in frame Appearance Clothes - Type Canada Female 11 15 P: With the frame she can only wear certain things. She knows that this is hanging out, it’s visible. Appearance Clothes - Type  118 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme India Female 11 16 P: She has to either wear long pants or jeans. But now it’s even visible when she wears jeans. Appearance Clothes - Fit US Female 14 17 C: Shoe- lifts was probably the one thing that I probably wouldn’t go to school and, it just seemed weird cause I thought there would be inside the shoe but it wasn’t and that’s a huge thing… everybody would notice. Appearance Shoe-lift Canada Female 11 18 P: I think she wore it to school once and then she didn’t wanna wear the shoes anymore. P: it was because people were kinda pointing, you know. If it’s more visually noticeable, she’s less likely to want to do that so you know. Appearance Shoe-lift US Female 16 19 C: ...if I was to take a picture of myself standing up, I wasn’t able to do that because I didn’t feel comfortable … like you would see me shifting. I would look like to the side and I didn’t like that because obviously people are gonna notice. Appearance Pictures  US Female 16 20 C: Running? Oh, I couldn’t. It would feel like I’m—one leg is hitting the floor faster than the other, so you  would just end up like—like falling pretty much until you—unless you know how to prepare yourself. But I couldn’t run. Physical  Function - mobility US Female 11 21 C: I’m slow and bad at running. Physical  Function - mobility Canada Male  14 22 C: My leg problems don’t allow me to walk like a longer period of time. After, like, about 10 – 15 minutes it will start hurting. Physical  Function - mobility Canada Male  16 23 C: Well, with the frame on, obviously I feel immobile and it’s not easy or fun to not be able to do much.  Physical  Function - mobility US Female 8 24 C: I could not take off the clothes when I am in frames and I’m wanting to go to the mall or something, so my mom helps me take them. Physical  Function - ADL  Canada Male  11 25 C: sometimes it was like uneven if I skated since one leg is longer than the other. Physical  Function -  Balance US Female 14 26 C: I stood on this leg and put this on the air then I fall over. Physical  Function -  Balance US Female 16 27 C: I would lose my balance and I would lean to the side. Physical  Function -  Balance India Male  16 28 C: I couldn’t run much when my knees used to rub against each Physical  Function -  119 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme other. I. How much were you able to run I.I could run as long as I wanted to by keeping my knees apart. I had to intentionally keep them apart. I had to force my knees to stay apart while running. Sports - Adaptation Canada Male  14 29 C: Ah, with sports, like I said with soccer I—I play goalie…P: … so—so it’s easier for me. Physical  Function - Sports - Adaptation India Male  12 30 C: When I have the frame on, I can’t play. Physical  Function - Sports  US Female 8 31 C: Well, I don’t really like doing sports because I feel uncomfortable with my leg in the frame. So, I could hurt myself doing other things. Physical  Function - Sports US Male  14 32 C: Well, soccer I couldn’t do. Basketball I can do because it wasn’t as much as running as soccer. Physical  Function - Sports US Male  11 33 P: because of the leg length. If he put too much pressure or if he—walked or he biked too long, his legs would hurt, he would have to stop. Because his legs would hurt or his hip.  Physical  Symptoms - Pain US Female 16 34 C: I had pain when I was in school and I was walking a lot.   Physical  Symptoms - Pain India Male  18 35 C: No, I can’t stand for a long time. My leg gets tired and my foot gets swollen. Physical  Symptoms - Tired India Male  18 36 C: I don’t do much because of limping. Physical  Symptoms - Limping US Male  16 37 C: My back always hurts and sometimes I’d have to stop doing whatever I’m doing. If my knee hurts, then I’ll walk slower. Physical  Symptoms - Pain Canada Male  14 38 C: After, like, about 10 – 15 minutes of walking it will start hurting. Physical  Symptoms - Pain Canada Male  18 39 C: ...before I got this treatment done, like it was really bad, like I had trouble sleeping at night. Physical  Symptoms - Pain Canada Male  18 40 C: Because my back has already starting to get more and more painful before I got this treatment done, like it was really bad. Physical  Symptoms - Pain Canada Male  8 41 C: I don’t like how. It has the fixator on and sometimes when it's functioning it hurts, it ends up aching and pounding. Physical  Symptoms - Pain Canada Male  18 42 C: It’s a little deformed, coz it’s not normal. Psychological  Body image  Canada Male  11 43 C: I’m pretty normal. I like to go to schools, which sounds kind of weird sometimes, but I like to sometimes I like to do sports and stuff Psychological  Body image   120 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme at school. I like quality time with my family. Canada Male  16 44 P: when he was going to school he wore shorts, he would wrap the shorts to fully cover: … he wanted it all wrapped up so he will get an extra T-shirt and wrap around so you couldn’t see that the bottom front of his frame and just use safety pins. He was a little bit self-conscious. Psychological  Body image  Ethiopia Male  16 45 C: I can find clothes but you can clearly see my leg problems when I wear shorts and I always feel ashamed wearing shorts so I don't prefer wearing them. Psychological  Body image  Canada Female 13 46 C: I’m really used to walking around with it and I even like—I knew how to walk without having a limp, so people didn’t even notice at all.  Psychological  Body image  Ethiopia Female 17 47 C: I don’t go anywhere. I only go to school and church. I: why? C: Because I don't want to go anywhere with my stick, I just don't feel happy doing that.  Psychological  Body image  Ethiopia Male  13 48 C: Yes, I feel so self-conscious. I look people around if they are staring at me or not. Psychological  Body image  Canada Female 11 49 P: she’s conscious of her appearance. She wants to be as normal, like I mentioned, if she has a lift on her leg or on her shoe, she’s very conscious about that, because you know, kids are aware of it and she is aware that they are aware of it. P: … she’s self-conscious about how she looks to the other kids.  Psychological  Body image  US Male  16 50 C: I feel little self-conscious about the appearance of my leg with the fixator on because people may think differently of me and ask about it.  Psychological  Body image  US Female 10 51 C: Yeah, I worry. It’s like would I still be able to move around when I grow up? Would I be able to get agile when I grow up? Will I be able to move around and agile? What type of job do I have to get so—with my bends legs? So, I thought about it a lot.  Psychological  Distress - worry US Female 9 52 C: Sometimes before I go to bed I worry about I’m never gonna be able to walk and that I might just be in the wheelchair for the rest of my life. Psychological  Distress - worry India Male  14 53 C: I don’t play.  I am worried that I will hurt my foot while playing I: What do you think will happen to your foot C: It will be painful if? It Psychological  Distress - worry  121 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme gets hurt while playing I: Is that why you don’t play C. Yes. P: There was a huge effect on him. He would cry watching other children. He wouldn’t be able to wear clothes. Ethiopia Male  16 54 C: Limping worries me a lot, some people stare at you, and kids laughs. Psychological  Distress - worry Ethiopia Male  16 55 C: Yes, I worry because they told me I might not even stand by my self after a couple of years and might need a wheel chair.  Psychological  Distress - worry US Female 16 56 C: I always hear that like something a leg discrepancy would happen again. You never know I’m still 16. What if my leg decides to grow again? Then what?   Psychological  Distress - worry Ethiopia Female 17 57 C: I don’t go anywhere, I only went to school and church. I Because I don't want to go anywhere with my stick, I just don't feel happy doing that. Psychological  Distress - emotions Ethiopia Male  13 58 C:  I get mad sometimes because when I wear shorts the scar is visible and people ask me what’s wrong with me and I don't like that.  Psychological  Distress - emotions US Female 10 59 C: Sometimes it makes me feel sad but all that time I’m okay with it, like, when I’m not able to do things with my friends, that’s when I feel sad—that’s—why is my leg like this? I start questioning myself sometimes. Psychological  Distress - emotions Canada Male  8 60 C: sometimes sadness, yeah. Because I keep wanting to come out of it. Psychological  Distress - emotions Canada Female 11 61 C: Like, sometimes this thing (frame) makes me really angry [chuckle]… and then I become mad at the world and just everything. Psychological  Distress - emotions India Female 11 62 C: Yes, I do get angry because I want it to get better quickly. Psychological  Distress - emotions Canada Female 13 63 C: In the beginning, ah, like, before I had it, I was like so mad, like, I was like wondering why I had to have this. None of my friends had anything like it so I felt like really alone and stuffs.  Psychological  Distress - emotions US Female 16 64 C: I would get kind of upset and bring myself down. Psychological  Distress - emotions India Male  18 65 C: Yes, I feel angry.  I: Who do you get angry at? C: At my leg that limps. Psychological  Distress - emotions India Male  14 66 C: Yes, I would get angry when I was not able to play. Psychological  Distress - emotions  122 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme US Female 16 67 M: she was concerned about, “Mommy how is—how it’s gonna be after surgery, how …” She was thinking about how—how she’s gonna look after the surgery. Psychological  Distress - emotions US Male  14 68 C: I felt a little bit annoyed because, like, it was kind of normal as, like, people are, like, always asking. So, it’s just, like, when can you stop asking?  Psychological  Distress - emotions US Female 16 69 C: I didn’t even wanna get dressed in the mornings. Like I didn’t even wanna get ready for anything anymore. I wanted to wear like baggy clothes. So it did affect my confidence somewhat and my self-esteem. Psychological  Confidence and Self-esteem Ethiopia Male  16 70 C: Always get very sad, seeing every kid play and not playing. And ask question why this happened to me and feel inferior than the other kids. Psychological  Confidence and Self-esteem US Male  16 71 C: Well, right now with the frame on, I feel, like, not very confident in my abilities because I really can’t do as much because I don’t have my full range of motions.  Psychological  Confidence and Self-esteem Canada Female 11 72 P: when the frame is on, her self-confidence kind of gets a little less….Just because she is aware of it and she knows that they’re looking at her. P:  I know she doesn’t like that as well. She doesn’t like to draw attention to it. Psychological  Confidence and Self-esteem US Female 10 73 C: And I’m not confident to do a lot of things most of the time I will —like if it’s a big events  I’m not really confident to go with something short. I try to find something long to cover my legs. So, it reduces my confidence. Psychological  Confidence and Self-esteem US Female 9 74 C: Yes, my confidence is affected. I: Yes. In what way? C: Usually when I go to school wearing shorts, skirt cause when my leg shows I usually wanna cover it, like wrap a thing around it, so nobody can see it. Psychological  Confidence and Self-esteem US Female 16 75 C: If I was to take a picture of you standing up, I wasn’t able to do that because I didn’t feel comfortable like you would see me shifting. I would look like to the side and I didn’t like that because obviously people are gonna notice. So I couldn’t even do that. I never like to be taken pictures of like full body and then I would l look at myself and be like, “Wow, like this is tremendous, this is”. So imagine Psychological  Confidence and Self-esteem  123 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme somebody that has a bigger difference than me, that’s even worse.  US Female 8 76 C: To feel better, I bought a journal so I can write about it sometimes.  Psychological  Coping US Male  16 77 C: Sometimes I would be sad because it’s like, “Why me?” but then it’s, like, everything happens for a reason. Psychological  Coping Canada Male  14 78 C: Ah, I just think that there are some people who are far worst who have no legs. I: So that makes you feel better then, right? C: Well it makes my life, makes me feel like my problems are not as bad. Psychological  Coping US Male  16 79 C: It’s a reminder of what I have been through. How far I’ve come. And just a part of me and sometimes people notice them and they ask me what happened. Psychological  Coping US Male  14 80 C: but sometimes I think like, it is better because I get experience from this and I get to grow from this, like I overcame, like, this whole problem by myself and I’m proud of it.  Psychological  Coping US Female 16 81 C: I also didn’t want that to affect me and what I wanted to be. I wasn’t gonna let it take over me. Psychological  Coping US Female 14 82 C: Usually if you find out that something’s wrong with you, it kinda push you down but it actually brought me up. It made me wanna do things better.  Psychological  Coping Canada Male  11 83 C: But we do have appointments for like physio and going to the doctor’s appointment – that would kind of affect my school because on school days, I would have to go to the hospital sometimes, so I would have to minimize my going to school, and then I’d have to work a bit harder, because of the days that I miss school. I have to get the homework for that.  School Function Ethiopia Male  11 84  C: No I don't go to school when I have the frame, I’m scared kids might push me and if I fell there will be a lot of problem..  School Function US Female 14 85 C: I couldn’t go to school if I’m on medication. So that’s why it took me so long to go to school because I don’t wanna go to school in pain and I couldn’t take Advil. School Function Canada Male  11 86 C: It’s bit nervous going to school with the frame because I’m a bit nervous because there’s a lot of kids at school and a lot of curious kids that might want to like touch it and I don’t want them to touch it in case of infection.  School Function Ethiopia Male  10 87 C: When I was sick we went to different hospitals and miss a lot of School Function  124 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme school and I forgot most of it so I start from grade one.  Ethiopia Female 11 88 C: Ohh, I stopped school for a year and I was not able to play with my friends either. School Function Ethiopia Female 17 89 C: Yes a lot, for my first surgery I stayed in the hospital for a couple of weeks and for the second one I stayed here for a couple of months. School Function Ethiopia Male  11 90 C: I had to quit school and that was hard because it takes a lot of time to get the treatment.  School Function India Male  15 91 C: This has affected my studies a lot. My whole school year has been wasted. School Function Ethiopia Male  11 92 C: I could not be equal to my friends, not being able to be in the grade I want to be in my age. School Function India Male  12 93 C: I don’t play at school. I am worried that I will hurt my foot while playing. School Participation Canada Male  14 94 C: when we do a running thing… ah, ah, usually I just sit out coz as I got older when I started to run it got more painful and harder, so now I just kinda when they are doing, like, a running activity I usually just don’t do it coz it hurts. School Participation India Male  12 95 C: No, I don’t play outdoors when I have the frame. I play indoor game that I can play sitting down. My friends also come and play with me.  School Participation US Female 8 96 C: Well, when I wasn’t ready to keep-up with the class, the teacher sent me a kid to walk with me. School Emotional support US Female 12 97 C: Um, they were really good. They helped me if I needed it. They helped against bullies and stuff like that. School Emotional support India Male  12 98 C: Yes, I have a lot of friends. In my last school, the entire school became my friend because they all would come to see me after my operation. School Emotional support Ethiopia Female 17 99 C: When I go to school I used a stick to walk and had to take a stair to get to class and my hands always get bloody and I can’t carry my text books I have to ask my friends to carry them for me.  School Instrumental Support Ethiopia Female 11 100 C: They are supportive. In our break or after school they always let me out first or stay with me until the crowed is gone so that I won’t fall and hurt myself again.  School Instrumental Support Canada Male  16 101 C: Usually teachers just make sure someone brings my backpack to School Instrumental  125 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme me and overall it’s fine so far. Support Ethiopia Female 17 102 C: And if there is a makeup class the teachers always give that in the top of the building, because all of the classes will be occupied and I can’t take all of the stairs so I had to miss all of the makeup classes.   School Environmental Barriers US Female 16 103 C: It made it so much harder to keep up with all the work and just like, even now, I feel so overwhelmed because I’m in a wheelchair, I don’t have a desk, like my papers are everywhere. I am…where am I gonna write.  School Environmental Barriers US Female 16 104 C: I have to deal with whatever I have. So in the negative aspect as people, like, kids my age these days, like I’m in 11th grade, I’m in high school, people look at me like, “What is wrong with you?”  School Isolation US Female 14 105 C: Some people were really mean to me. Some people would start out dress code to me because I physically could not put on pants.  Because the brace came up to here.  School Isolation Canada Female 8 106 C: It’s an ongoing problem with the kids at school who always ask me “what do you have in your feet”. I don’t like that. School Isolation Canada Female 13 107 C: Oh ah [pause]—I don’t—well, yeah, coz sometimes we went on field trips and stuff and I was kind of stuck in my crutches watching everyone. I was kinda left out sometimes coz everyone was riding around and I was just sitting there. School Isolation Canada Male  11 108 C: But sometimes people would be curious about my leg problems, and sometimes they’d wonder, ‘Oh why is your shoe bigger than the other shoe?’  School Isolation US Male  16 109 I: Did you ever hesitate to make more friends or new friends because you were worried that they were going to notice your leg? C: Occasionally, I might, cause I think before like, analyzing people to see how they may react to it and if they were good person and they will look back, well I would be more open to them but as opposed to a person who might not be as supportive. Social  Function US Female 16 110 C: But the moment when I would walk again, you could notice. That made me feel very like uncomfortable. So I never wanted to go out. If my mom told me, “Oh, let’s go somewhere and let’s go to the grocery store.” Anywhere, like I didn’t wanna go. I just wanted to stay home in comfortable clothes where nobody would know what I Social  Function  126 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme had. US Female 16 111 C: I wouldn’t wanna go out with friends because they were normal. My friends were normal. They look even and I didn’t. And I always like thought and I was to the side. Social  Function US Female 16 112 C: I’m in high school. I would like be on the social media and see everybody else taking picture of like, their body and stuff like, you know somebody else I guess. Social  Function US Male  16 113 C: Sometimes I’ll be sad because I wasn’t able to go to the beach with my friends or go to amusement parks. Social  Function India Male  12 114 C: Can’t play catch with my siblings. I: What do you do when they are playing catch? C: sit on one side. Social  Function Canada Male  11 115 P: A little bit inconvenient right now for me because he has to almost all the time stay at home, not going out with us, –well, we didn’t going out on weekends. We cannot have a long trip for a while, because we have to take care of the alignment, cleaning of pins, those kind of things so after the surgery, we stay home more than before. Social  Function Canada Male  18 116 C: It’s harder to get a job. It’s been a couple of months and I’m looking and even places that are hiring, uh, they don’t call back.  Social  Function Canada Male  18 117 C: I worked in sales. It’s all standing-up jobs. Ah um, I would have done apprenticing if it weren’t for the high demand of electrical apprentice, if it weren’t for the amount of standing that I had to do. Social  Function India Male  12 118 C: No, My other friends tell the new friends not to ask me about it because I might feel bad about it.  Social  Emotional support Canada Female 13 119 C: They're really, really helpful, especially when I had my frame on.: Um, they would carry my bags, they would carry my instrument, and they didn’t care that I was, like coz I was in a wheelchair. So it’s like, when I had to out places. So then, they even took me to concerts in my wheelchair and then they took me trick or treating.  Social  Emotional support US Female 16 120 I: Do you—do you—so you did feel the need of support groups? P: Yeah. Of course and they need to have people that understand what they went through or what they’re going through.  Social  Emotional support Ethiopia Female 17 121 C: My friends are so understanding, starting from grade one until grade nine they are the one who carries my textbooks. And I never went to school alone, they always come in turn to my home and go to Social  Emotional support  127 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme school with me. Canada Male  16 122 C: A lot of strangers open doors for me when I had the frame on, then people would open a door if they saw me with my crutches. Social  Emotional support US Female 14 123 C: My mom was the only person that was helpful. She was so supportive. Um, I definitely think it was stressful. She had to take a lot of time off of work to take me and pick me up. Social  Emotional support Canada Female 13 124 C: They're really, really helpful, especially when I had my frame on. P: Um, they would carry my bags, they would carry my instrument, and they didn’t care that I was, like coz I was in a wheelchair. So it’s like, when I had to out places. So then, they even took me to concerts in my wheelchair and then they took me trick or treating. Social  Instrumental Support India Male  14 125 C: Yes, everyone knew and even though some would tease me, the rest were okay with it.  Social  Isolation India Male  18 126 C: Yes, they do make fun of me. They call me lame. Social  Isolation US Female 10 127 P: She would go to church, she would like to sit inside the church because people were looking at her P: She would like to want that she would sit outside.  Social  Isolation Canada Male  13 128 C: I get upset and like leave when my siblings make fun of me.  Social  Isolation Ethiopia Female 11 129 I:  What happens when someone new meets you for the first time? C: They pity me and always have a sad face. I: What does that make you feel? C: It makes me very mad. Social  Isolation US Male  16 130 C: They would go call me cyborg.  Social  Isolation Canada Male  8 131 C: yeah, and just when we're in places he's really looking at your eyes, he pays attention to people staring at him.  Social  Isolation Canada Female 13 132 C: In the beginning, ah, like, before I had it, I was like so mad, like, I was like wondering why I had to have this. None of my friends had anything like it so I felt like really alone and stuffs.  Social  Isolation Canada Female 11 133 C: Um, kinda just because they look at me differently than other people.  And, um, they don’t wanna social, like, talk and stuff.  Social  Isolation Canada Female 13 134 C: …So I just started showing my leg when we went somewhere and I just kind of find it funny how people would stare at me and like they would make like really funny faces when they looked at my leg. Social  Isolation India Female 12 135 C: Yes, my friends would notice and ask what happened. C: They would say there is a bit of a limp. Social  Isolation  128 Country Sex Age (years) Quote # Child Quote (C), Parent Quote (P) Theme Sub-theme US Male  14 136 P: because at this point, you know, mostly everybody that he came close to will have something to say about him and about his leg and about the way he looked. Social  Isolation  129  Table 7: Demographics and clinical characteristics of patients participating in cognitive debriefing interviews Demographic and Clinical Variables  Number (%) Country Australia 5 (29) Canada 11 (65) USA 1 (6) Gender Male 10 (59) Female 6 (35) Age (Years) Average 13 Range 8 to 17 Type of deformity Fibular Hemimelia 6 (35) Amputation 4 (24) Hemi-hypertrophy 2 (12) Ollier's Disease 1 (6) Spondylocostal dysostosis and Scoliosis 1 (6) Posteromedial Tibial Bowing and Leg Length Discrepancy 1 (6) Congenital Short Femur 1 (6) Leg Length Discrepancy and Dysplastic Lateral Femoral Condyle 1 (6)     130 Table 8: Characteristics of experts who provided feedback Speciality Australia Canada Ethiopia India UK USA Total (%) Orthopaedic Surgeon 1 1 1 1 2 3 9 (39) Nurse Practitioner 0 1 0 0 0 0 1 (4) Physiotherapist 1 2 0 0 1 0 4 (17) Occupational Therapist 0 2 0 0 0 0 2 (9) Psychologist 0 0 0 0 1 0 1 (4) Prosthetist 0 1 0 0 1 0 2 (9) Parent 0 4 0 0 0 0 4 (17) Total 2 (9) 13 (57) 1 (4) 1 (4) 5 (22) 3 (13) N= 23    131 Table 9: Sequence of cognitive debriefing interviews and expert feedback Round 1  Country N Patients  Canada 5 Experts  Canada 3 Consultation with an orthopaedic surgeon and experts in PROM development and HRQL Round 2 Patients  Australia 2 Canada 5 US 1 Experts  Australia 1 UK 2 US 2 Parents  Canada 1 Consultation with an orthopaedic surgeon and experts in PROM development and HRQL Round 3 Patients  Australia 2 Experts  Australia 1 Canada 3 Ethiopia 1 India 1 UK 2 US 1 Consultation with an orthopaedic surgeon and experts in PROM development and HRQL Round 4 Patients  Canada 3 Experts  Canada 3 UK 2 US 2 Parents  Canada 3 Consultation with an orthopaedic surgeon and experts in PROM development and HRQL Round 5 Patients  Australia 1 Consultation with an orthopaedic surgeon and experts in PROM development and HRQL     132 Table 10: Number of items retained, revised, dropped, and added during each round  Round 1: Version 1 Patients =5 Experts =3 Round 2: Version 2 Patients = 8 Experts = 7 Parents= 1 Round 3: Version 3 Patients = 2 Experts = 9 Round 4: Version 4 Patients = 3 Experts = 7 Parents =3 Round 5: Version 5 Patients = 1 Physical Function Scale  Number of Items Retained 27 23 30 32 32 Number of Items Revised 1 5 9 0 0 Number of Items Dropped 0 0 0 0 0 Number of Items Added 0 2 2 0 0 Total Items at The End of Round 28 30 32 32 32 Leg Symptoms Scale  Number of Items Retained 6 4 9 11 14 Number of Items Revised 1 3 0 0 0 Number of Items Dropped 0 0 0 0 0 Number of Items Added 0 2 2 3 0 Total Items at The End of Round 7 9 11 14 14 Hip Symptoms Scale Number of Items Retained 2 5 7 10 10 Number of Items Revised 3 2 0 0 0 Number of Items Dropped 0 1 2 0 0 Number of Items Added 3 2 3 0 0 Total Items at The End of Round 8 9 10 10 10 Knee Symptoms Scale  Number of Items Retained 2 2 9 13 13  133  Round 1: Version 1 Patients =5 Experts =3 Round 2: Version 2 Patients = 8 Experts = 7 Parents= 1 Round 3: Version 3 Patients = 2 Experts = 9 Round 4: Version 4 Patients = 3 Experts = 7 Parents =3 Round 5: Version 5 Patients = 1 Number of Items Revised 3 4 0 0 0 Number of Items Dropped 0 1 0 0 0 Number of Items Added 2 3 4 0 0 Total Items at The End of Round 7 9 13 13 13 Ankle Symptoms Scale  Number of Items Retained This scale was added after round 2 8 10 10 Number of Items Revised 0 0 0 Number of Items Dropped 0 0 0 Number of Items Added 2 0 0 Total Items at The End of Round 10 10 10 Foot Symptoms Scale  Number of Items Retained 4 4 5 9 9 Number of Items Revised 2 2 0 0 0 Number of Items Dropped 0 0 3 0 0 Number of Items Added 0 2 4 0 0 Total Items at The End of Round 6 8 9 9 9 Leg-Related Distress Scale  Number of Items Retained 14 13 13 13 14 Number of Items Revised 1 2 5 0 0 Number of Items Dropped 0 0 1 0 0  134  Round 1: Version 1 Patients =5 Experts =3 Round 2: Version 2 Patients = 8 Experts = 7 Parents= 1 Round 3: Version 3 Patients = 2 Experts = 9 Round 4: Version 4 Patients = 3 Experts = 7 Parents =3 Round 5: Version 5 Patients = 1 Number of Items Added 0 0 0 0 0 Total Items at The End of Round 15 15 14 14 14 Appearance Scale  Number of Items Retained 18 14 17 17 17 Number of Items Revised 0 4 0 0 0 Number of Items Dropped 0 0 1 0 0 Number of Items Added 0 0 0 0 0 Total Items at The End of Round 18 18 17 17 17 School Function Scale  Number of Items Retained 17 15 16 17 17 Number of Items Revised 0 1 1 0 0 Number of Items Dropped 0 1 0 0 0 Number of Items Added 0 1 0 0 0 Total Items at The End of Round 17 17 17 17 17 Social Function Scale  Number of Items Retained 13 12 12 12 12 Number of Items Revised 0 0 0 0 0 Number of Items Dropped 0 1 0 0 0 Number of Items Added 0 0 0 0 0 Total Items at The End of Round 13 12 12 12 12  135  Round 1: Version 1 Patients =5 Experts =3 Round 2: Version 2 Patients = 8 Experts = 7 Parents= 1 Round 3: Version 3 Patients = 2 Experts = 9 Round 4: Version 4 Patients = 3 Experts = 7 Parents =3 Round 5: Version 5 Patients = 1 Psychological Function Scale  Number of Items Retained 10 10 10 10 10 Number of Items Revised 0 0 0 0 0 Number of Items Dropped 0 0 0 0 0 Number of Items Added 0 0 0 0 0 Total Items at The End of Round 10 10 10 10 10  *Some experts from previous rounds were interviewed again in round 4 to review the Leg Symptoms Scale. These experts were only counted once in the total number of experts.     136 Table 11: Evidence used to retain, revise, add, and drop items for the Physical Function Scale Physical Function Scale  Final Version of Items  Supporting Data 1. …get out of bed? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 2. …sit in a chair? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 3. …get up from a chair? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 4. …sit on a toilet? Item was added after round 3 of interviews as suggested by experts to capture the physical function required for using the toilet. This item was easily understood by 1 patient interviewed after round 3. 5. …sit cross-legged on the floor? Easy to understand: 15/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited during round 2 from 'sit cross-legged' to 'sit cross-legged on the floor' as clarification was sought by one participant. This item was edited once and retained. 6. …get up from the floor? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 7. …lift one leg while you stand on the other? Easy to understand: 13/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited from ‘lift your leg (e.g. to kick a ball) to ‘lift your leg while standing' after round 1. This item was further revised from ‘lift your leg while standing’ to ‘lift one leg while standing on the other’ (F-K grade level 2.2) during round 2. It was edited slightly after round 3 of interviews, to its current form, to simplify to a lower F-K grade level (1). 8. …bend your knees? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 9. …pick up a book from the floor? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited from 'pick something up from the floor' (F-K grade level 2.4) to 'pick up a book from the floor' (F-K grade level 0) after the third round of interviews to simplify to a lower F-K grade level. 10. …walk without a limp? Easy to understand: 13/16 patients, 17/18 experts; Relevant: 16/16 patients, 18/18 experts. 3/15 patients found word limping difficult to understand but on further probing they were able to explain it. 1/18 experts said that it might be a difficult word for some young children. Item was retained and modified after round 3 to simplify to a lower F-K grade level. It was edited from ‘walk without limping' (F-K grade level 5.2) to 'walk without a limp' (F-K grade level 0.7). 11. …walk and not fall? Easy to understand: 17/17 patients, 18/18 experts; Relevant: 17/17 patients, 18/18 experts. This item was edited slightly after  137 Physical Function Scale  Final Version of Items  Supporting Data round 3 to simplify to a lower F-K grade level. It was edited from ‘walk without falling' (F-K grade level 5.2) to 'walk and not fall' (F-K grade level 0). 12. …walk on a bumpy road? Easy to understand: 3/3 patients, 9/9 experts; Relevant: 3/3patients, 9/9 experts. This item was added after round 2. It was retained as such for round 3. It was modified after round 3 to lower the F-K grade level. It was edited from ‘walk on uneven (bumpy) surfaces’ (F-K grade level 7.6) to 'walk on a bumpy road' (F-K grade level 0.5). 13. …walk as fast as you like? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 14. …walk as far as you like? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 15. …walk up the stairs? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/17 patients, 18/18 experts. It was modified after round 3 from' walk up stairs' to ‘walk up the stairs'. 16. …walk down the stairs? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts It was edited after round 3 from' walk down stairs' to ‘walk down the stairs'. 17. …walk while you carry a book? Easy to understand: 14/16 patients; 18/18 experts; Relevant; 16/16 Patients, 18/18 Experts. 2 patients had questions about the item in round 1. No edits made after round 1 but it was decided to probe for this item in round 2. One patient asked for clarification in round 2, content in the brackets was removed to avoid any confusion. Item was found easy to understand in round 3. Item was revised after round 3 of interviews to simplify it further to a lower F-K grade level. It was edited from ‘walk while you carry something' (F-K grade level 5.2) to 'walk while you carry a book' (F-K grade level 0). 18. …walk up a hill? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 19. …walk down a hill? Easy to understand: 17/17 patients, 18/18 experts; Relevant: 17/17 patients, 18/18 experts 20. …stand for a long time? Easy to understand: 16/17 patients, 18/18 experts; Relevant: 17/17 patients, 18/18 experts, 1/17 patients asked for clarification on how long to stand for. 21. …climb (such as up a ladder)? Easy to understand: 15/15 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. Item was edited after round 2 of interviews from 'climb (e.g. up a ladder)' to 'climb (such as up a ladder)'. 1/16 patients (8 year old) didn’t know what ‘e.g.’ means. 22. …run? Easy to understand: 17/17 patients, 18/18 experts; Relevant: 17/17 patients, 18/18 experts 23. …run and not fall Easy to understand: 16/16 patients, 18/18 experts; Relevant:  138 Physical Function Scale  Final Version of Items  Supporting Data down? 16/16 patients, 18/18 experts. Item was revised after round 3 to reduce the grade reading level. It was edited from ‘run without falling' (F-K grade level 5.2) to 'run and not fall down' (F-K grade level 0). 24. …run as fast as you want? Easy to understand: 16/16 patients, 18/18 experts ; Relevant: 16/16 patients, 18/18 experts 25. …run as far as you want? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 26. …ride a bike? Easy to understand: 16/16 patients, 15/18 experts; Relevant: 16/16 patients, 16/18 experts. 3 experts (2 were from India and Ethiopia, one was practising in the UK while trained in India) suggested to replace it with word bicycle for children from India or Ethiopia since it’s a more commonly used term. Interviewer suggested that these words will be replaced as needed during the translation and cultural adaptation phase later.  27. …play sports you like? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. 28. …jump? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. 29. …put on or take off shoes? Easy to understand: 16/16patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. 30. …put on or take off clothes? Easy to understand: 17/17 patients, 18/18 experts; Relevant: 15/15 patients, 18/18 experts. 31. …jump up and down on one leg? Easy to understand: 3/3 patients, 9/9 experts; Relevant: 3/3 patients, 9/9 experts. This item was added after round 2 based on expert input. It was tested in 3 children and 9 experts. They all found it easy to understand and relevant. 32. …stand on one leg? This item was added after round 3 based on expert input. This item was considered a harder item for the concept balance. F-K grade level is 0 for this item. Items 7, 31 and 32 are all testing balance. One patient interviewed after round 3 found the item easy to understand.  *This scale was reviewed by 16/17 patients, 1 patient reviewed the Leg Symptom Scale only; 18/23 experts reviewed this scale (including parents).    139 Table 12: Evidence used to retain, revise, add, and drop items for the Leg Symptoms Scale Leg Symptoms Scale  Final Version of Items Supporting Data 1. My leg hurts when I rest or relax. Easy to understand: 17/17 patients, 23/23 experts; Relevant: 17/17 patients, 23/23 experts. After round 2 of interviews, it was edited from 'My leg hurts when I am resting or relaxing' to 'My leg hurts when I rest or relax'. 2. My leg hurts when I sleep. Easy to understand: 2/2 patients, 5/5 experts; Relevant: 2/2 patients, 5/5 experts. This item was added after round 3 of interviews as suggested by experts.  3. My leg hurts when I stand for a long time. Easy to understand: 2/2 patients, 5/5 experts; Relevant: 2/2 patients, 5/5 experts. This item was added after round 3 of interviews as suggested by experts.  4. My leg hurts when I walk. Easy to understand: 17/17 patients, 23/23 experts; Relevant: 17/17 patients, 23/23 experts. 5. My leg hurts when I run. Easy to understand: 17/17 patients, 23/23 experts; Relevant: 17/17 patients, 23/23 experts.  6. My leg hurts when I touch it. Easy to understand: 2/2 patients, 5/5 experts; Relevant: 2/2 patients, 5/5 experts. This item was added after round 3 after additional feedback from patients with amputations, parents and experts. 7. My leg gets tired when I walk. Easy to understand: 17/17 patients, 23/23 experts; Relevant: 17/17 patients, 23/23 experts. 8. My leg gets tired when I run. Easy to understand: 17/17 patients, 18/18 experts; Relevant: 17/17 patients, 23/23 experts. 9. My leg feels weak (shaky).  Easy to understand: 17/17 patients, 23/23 experts; Relevant: 17/17 patients, 23/23 experts. After round 2, this item was edited from 'My leg feels weak' to ‘My leg feels weak (shaky)’. All interviewed children understood the word ‘weak’ but they all described it differently. As suggested by experts, word ‘shaky’ was added in brackets to provide an alternate word.  10. My leg feels numb (cannot feel it). Easy to understand: 4/4 patients, 14/14 experts; Relevant: 4/4 patients, 14/14 experts. This item was added after round 2 of interviews as suggested by experts. It was tested in 4 children and 14 experts. 11. My leg feels tingly (pins and needles feeling). Easy to understand: 4/4 patients, 14/14 experts; Relevant: 4/4 patients, 14/14 experts. This item was added after round 2 of interviews as suggested by the experts. It was tested in 4children and 14 experts. 12. My leg is swollen or puffy. Easy to understand: 15/17 patients, 23/23 experts; Relevant: 17/17 patients, 23/23 experts. After round 1, this item was edited from 'My leg is puffy or swollen (bigger than normal)’ to 'My leg is swollen' as 1 patient asked for clarification on whether its comparison to their other leg or another person's leg. Word 'puffy' was removed to test whether one word 'swollen' would be sufficient for patients to understand the item. After round 2, word 'puffy' was added again as suggested by experts.  13. My leg feels itchy. Easy to understand: 2/2 patients, 5/5 experts; Relevant: 2/2 patients, 5/5  140 Leg Symptoms Scale  Final Version of Items Supporting Data experts. This item was added after round 3 after additional feedback from patients with amputations, parents and experts.  14. My leg has a rash (looks red).  Easy to understand: 4/4 patients, 5/5 experts; Relevant: 4/4 patients, 5/5 experts. This item was added after round 3 after additional feedback from patients with amputations, parents and experts.  *This scale was reviewed by additional patients with amputations and experts (including parents) after round 3. Seventeen patients and 23 experts reviewed this scale.    141 Table 13: Evidence used to retain, revise, add, and drop items for the Hip Symptoms Scale Hip Symptoms Scale  Final Version of Items  Supporting Data 1. My hip hurts when I rest or relax. Easy to understand: 16/16patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. After round 1 of interviews, this item was edited from' My hip hurts' to 'My hip hurts when I am resting or relaxing' as suggested by experts to separate the hip symptoms while resting, walking and running. This was further edited after round 2 of interviews to ' My hip hurts when I rest or relax'. 2. My hip hurts when I sit for a long time. Easy to understand: 3/3 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was added after round 2 of interviews as suggested by experts and tested in 3 patients and 9 experts. 3. My hip hurts when I stand for a long time. This item was added after round 3 of interviews as suggested by the experts and tested in 1 patient. 4. My hip hurts when I walk. Easy to understand: 11/11 patients, 15/15 experts; Relevant: 11/11 patients, 15/15 experts. This item was added after round 1 of interviews as suggested by experts. 5. My hip hurts when I walk up the stairs. This item was added after round 3 of interviews as suggested by experts and tested in 1 patient. 6. My hip hurts when I walk down the stairs. This item was added after round 3 of interviews as suggested by experts and tested in 1 patient. 7. My hip hurts when I run. Easy to understand: 11/11 patients, 15/15 experts; Relevant: 11/11 patients, 18/18 experts. This item was added after round 1 of interviews as suggested by experts. 8. My hip hurts when it makes a sound (clicks or pops). Easy to understand: 14/16 patients, 18/18 experts; Relevant: 5/16 patients, 18/18 experts. After round 2 of interviews, two items 'My hip clicks when I move' and 'My hip makes a popping sound when I move' were consolidated into one item 'My hip hurts when it makes a sound (clicks or pops)’ to be tested during round 3 of interviews. Clicking and popping was described as similar symptoms by some participants. Experts suggested consolidating the items as the symptoms are very similar. Clicking or popping with pain was suggested to be a clinically relevant symptom by experts. The item in its current form was understood by 2 patients and approved by 9 experts in round 3. 9. My hip gets stuck (not able to move it). Easy to understand: 13/15 patients, 18/18 experts; Relevant: 15/15 patients, 18/18 experts. After round 1 of interviews, this item was edited from' My hip locks (gets stuck)' to 'My hip gets stuck'. 3/5 patients during round 1 of interviews described locking as being stuck. This item was further edited after round 2 of interviews to ' My hip gets stuck (not able to move it)' as described by a patient. The item in its current form was understood by 2 patients and approved by 9 experts in round 3. 10. My hip feels weak Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2  142 Hip Symptoms Scale  Final Version of Items  Supporting Data (shaky). patients, 18/18 experts. This item was added after round 2 of interviews as suggested by experts and tested in 2 patients and 9 experts.  *This scale was reviewed by 16/17 patients, 1 patient reviewed the Leg Symptom Scale only; 18/23 experts reviewed this scale (including parents).    143 Table 14: Evidence used to retain, revise, add, and drop items for the Knee Symptoms Scale Knee Symptoms Scale  Final Version of Items  Supporting Data 1. My knee hurts when I rest or relax. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. After round 1 of interviews, this item was edited from 'My knee hurts' to 'My knee hurts when I am resting or relaxing'. After round 2 of interviews, it was edited from 'My knee hurts when I am resting or relaxing' to 'My knee hurts when I rest or relax'. 2. My knee hurts when I stand for a long time. This item was added after round 3 of interviews as suggested by experts. One patient found it easy to understand. 3. My knee hurts when I walk. Easy to understand: 11/11 patients, 15/15 experts; Relevant: 11/11 patients, 15/15 experts. This item was added after round 1of interviews. 4. My knee hurts when I walk up the stairs. This item was added after round 3 of interviews as suggested by experts and tested in 1 patient. 5. My knee hurts when I walk down the stairs. This item was added after round 3 of interviews as suggested by experts and tested in 1 patient. 6. My knee hurts when I run. Easy to understand: 11/11 patients, 15/15 experts; Relevant: 11/11 patients, 15/15 experts. This item was added after round 1 of interviews. 7. My knee hurts when I bend it. Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was added after round 2 of interviews as suggested by experts. This item was tested in with 2 patients and 9 experts. 8. My knee hurts when I straighten it. This item was added after round 3 of interviews as suggested by experts and tested in 1 patient. 9. My knee hurts when it makes a sound (clicks or pops). Easy to understand: 14/16 patients, 18/18 experts; Relevant: 16/16 patients. After round 2 of interviews, two items 'My knee clicks when I move' and 'My knee makes a popping sound when I move' were consolidated into one item 'My knee hurts when it makes a sound (clicks or pops) to be tested during round 3 of interviews. Clicking and popping was described as similar symptoms by some participants. Experts suggested consolidating the items as the symptoms are very similar. Clicking or popping with pain was suggested to be a clinically relevant symptom by experts. The item in its current form was understood by 3 patients and approved by 9 experts.  10. My knee gets stuck (not able to move it). Easy to understand: 14/16 patients, 18/18 experts; Relevant: 16/16 patients. After round 1 of interviews, this item was edited from' My knee locks (gets stuck)' to 'My knee gets stuck'. 3/5 patients during round 1 of interviews described locking as being stuck. This item was further edited after  144 Knee Symptoms Scale  Final Version of Items  Supporting Data round 2 of interviews to ' My knee gets stuck (not able to move it)' as described by a patient. The item in its current form was understood by 3 patients and approved by 9 experts.  11. My knee feels wobbly (like it is going to give out). Easy to understand: 14/16 patients, 18/18 experts; Relevant: 16/16 patients. After round 1 of interviews, this item was edited from' My knee gives way when I move (feels wobbly) to 'My knee feels wobbly'. Patients related more to word 'wobbly' than to 'gives way'. This item was further edited after round 2 of interviews from 'My knee feels wobbly’ to 'My knee feels wobbly (like it is going to give out)' as suggested by experts. The item in its current form was understood by 3 patients and approved by 9 experts.  12. My knee feels weak (shaky). Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was added after round 2 of interviews as suggested by experts. This item was tested in 2 patients and 9 experts. 13. My knee is swollen or puffy. Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts; This item was added after round 2 of interviews as suggested by experts. This item was tested in round 3 of interviews with 2 patients and 9 experts.  *This scale was reviewed by 16/17 patients, 1 patient reviewed the Leg Symptom Scale only; 18/23 experts reviewed this scale (including parents).     145 Table 15: Evidence used to retain, revise, add, and drop items for the Ankle Symptoms Scale Ankle Symptoms Scale  Final Version of Items Supporting Data 1. My ankle hurts when I rest or relax. Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was tested in 2 patients and 9 experts in round 3. 2. My ankle hurts when I stand for a long time. This item was added after round 3 of interviews as suggested by the experts.  3. My ankle hurts when I walk. Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts.  This item was tested in 2 patients and 9 experts in round 3. 4. My ankle hurts when I walk on a bumpy road. This item was added after round 3 of interviews as suggested by the experts. This item was similar in wording to item 12 in physical function scale. 5. My ankle hurts when I run. Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was tested in 2 patients and 9 experts in round 3. 6. My ankle hurts when it makes a sound (clicks or pops) Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts.  This item was tested in 2 patients and 9 experts in round 3. 7. My ankle gets stuck (not able to move it). Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was tested in 2 patients and 9 experts in round 3. 8. My ankle feels wobbly (like it is going to give out). Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts; This item was tested in 2 patients and 9 experts in round 3. 9. My ankle feels weak (shaky). Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was tested in 2 patients and 9 experts in round 3. 10. My ankle is swollen or puffy. Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts;  This item was tested in 2 patients and 9 experts in round 3.  *Ankle symptoms were added after round 2 of interviews and expert feedback. The items were similar in wording to other symptom scales. This scale was reviewed by 2 patients and 9 experts.    146 Table 16: Evidence used to retain, revise, add, and drop items for the Foot Symptoms Scale Foot Symptoms Scale  Final Version of Items  Supporting Data 1. My foot hurts when I rest or relax. Easy to understand: 13/13 patients, 18/18 experts; Relevant: 13/13 patients, 18/18 experts. After round 1 of interviews, this item was edited from 'My foot hurts' to 'My foot hurts when I am resting or relaxing'. After round 2 of interviews, it was edited from 'My foot hurts when I am resting or relaxing' to 'My foot hurts when I rest or relax'. 2. My foot hurts when I stand for a long time. This item was added after round 3 of interviews as suggested by the experts. This item was similar in wording to items in other symptom scales. 3. My foot hurts when I walk on a bumpy road. This item was added after round 3 of interviews as suggested by the experts. This item was similar in wording to items in other symptom scales. 4. My foot hurts when I run. Easy to understand: 13/13 patients, 18/18 experts; Relevant: 13/13 patients, 18/18 experts. No edit was made to this item. 5. My foot hurts when I walk with shoes on. Easy to understand: 13/13patients, 18/18 experts; Relevant: 13/13 patients, 18/18 experts; 'My foot hurts when I walk' was tested in round 1 and was later edited to the item in its current form. This item was edited after round 2 of interviews as suggested by the experts. It was tested in 2 patients and 9 experts. 6. My foot hurts when I walk barefoot (no shoes on my feet). Easy to understand: 2/2 patients, 9/9 experts; Relevant: 2/2 patients, 9/9 experts. This item was added after round 2 of interviews as suggested by experts. It was tested in 2 patients and 9 experts during round 3. 7. My foot gets stuck (not able to move it). This item was added after round 3 of interviews as suggested by experts. This item was similar in wording to items in other symptom scales. 8. My foot feels weak (shaky). This item was added after round 3 of interviews as suggested by experts. This item was similar in wording to items in other symptom scales. 9. My foot is swollen or puffy. Easy to understand: 12/13 patients, 18/18 experts; Relevant: 13/13 patients; After round 1, this item was edited from 'My foot is puffy or swollen (bigger than normal) to 'My foot is swollen’. Word 'puffy' was removed to test whether one word 'swollen' would be sufficient for patients to understand the item. After round 2 of interviews, word 'puffy' was added again as suggested by experts. One patient was not familiar with the word ‘swollen’ but understood word 'puffy' when probed.  *This scale was reviewed by 13/17 patients, 1 patient reviewed the Leg Symptom Scale only, 3 Patients with amputation did not review the foot symptoms scale; 18/23 experts reviewed this scale (including parents).  147 Table 17: Evidence used to retain, revise, add, and drop items for the Leg-Related Distress Scale Leg-Related Distress Scale  Final Version of Items  Supporting Data 1. I hide my leg when I go out. Easy to understand: 14/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. After round 1, this item was edited from 'I cover up or hide my leg when I go out' to 'I hide my leg when I go out'. 2 patients in round 1 did not understand that the item is asking about covering up the leg to hide it. 2. I avoid wearing shorts or skirts that show my leg. Easy to understand: 15/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. After round 2 of interviews, this item was edited from 'I avoid wearing shorts or skirts' to 'I avoid wearing shorts or skirts that show my leg'. This was edited as suggested by experts to make it clearer. 1 patient did not understand why someone would not wear shorts or skirts. 3. I dislike how my leg looks. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 4. I feel unhappy about my leg. Easy to understand: 15/15 patients, 18/18 experts; Relevant: 15/15 patients, 18/18 experts 5. I worry about my leg. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 6. I get upset when people look at my leg. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 7. I get upset when people ask about my leg. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 8. I dislike photos that show my leg. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited from 'I avoid having photos taken that show my leg' to 'I dislike photos that show my leg' to get rid of the word 'avoid' and to reduce the F-K grade reading level. The F-K reading level was reduced from 4.9 to 2.3. 9. I avoid going out because of my leg (like to a party). Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 10. I dislike how I look when I walk. Easy to understand: 15/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. After round 3, this item was edited from 'I dislike how I look when I walk (e.g. limp)' to 'I dislike how I look when I walk'. While the item was found relevant but different patients described how they walk differently. One patient described looking 'funky' not necessarily 'limping' when walking. 11. My leg makes me feel different from other people. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 3 from 'I feel different from other people because of my leg' to 'My leg makes me feel different from other people' to reduce the F-K grade level. F-K grade level was reduced from 6 to 4.9. 12. I get teased about my leg. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts  148 Leg-Related Distress Scale  Final Version of Items  Supporting Data 13. I get upset when my leg stops me from having fun. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 2 of interviews from 'I get upset when I can’t join activities and games' to 'I get upset when my leg stops me from joining games and activities'. After round 3, this item was further edited to 'I get upset when my leg stops me from having fun' to reduce the F-K grade level from 5.8 to 2.6. 14. I get upset when I am not able to wear the type of shoes I like. Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 3 of interviews from' I get upset when I can't wear different types of shoes' to ' I get upset when I am not able to wear the type of shoes I like' to remove the word 'can't'.  *This scale was reviewed by 16/17 patients, 1 patient reviewed the Leg Symptom Scale only; 18/23 experts reviewed this scale (including parents).     149 Table 18: Evidence used to retain, revise, add, and drop items for the Appearance Scale Appearance  Final Version of Items  Supporting Data 1. …how your leg looks when you walk? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 2. …how your leg looks when you run? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 3. …the length of your leg? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 4. …the size of your leg? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 2 from 'the size of your leg (not too skinny)' to 'the size of your leg'. 1 parent objected on using word 'skinny'. 5. …how your leg looks when you wear shorts or a skirt? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 2 from 'how your leg looks in shorts or a skirt' to ' how your leg looks when you wear shorts or a skirt'. 6. …how your leg looks when you wear pants or jeans? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 7. …how your leg looks in photos? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 8. …how your leg looks when you stand? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 9. …how your leg looks when you sit on the floor? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 10. …how your leg looks when you sit in a chair? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 11. …how your leg looks compared with other people's’ legs? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 12. …how well your legs match each other (look the same)? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 13. …how straight your leg looks? Easy to understand: 14/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 2 from 'how straight your leg looks (not crooked or lopsided)' to 'how straight your leg looks'. Words 'crooked' or 'lopsided' were removed as some patients were not familiar with these words. These words were initially added as words used by patients to describe their leg during the qualitative interviews. 14. …how your foot looks? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts  150 Appearance  Final Version of Items  Supporting Data 15. …how your knee looks? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts 16. …how well your hips match each other (straight or uneven)? Easy to understand: 15/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts. This item was edited after round 2 from 'how well your hips match each other (straight or tilted) to 'how well your hips match each other (straight or uneven)’. One patient did not understand word 'tilted'. 17. …how your leg looks overall? Easy to understand: 16/16 patients, 18/18 experts; Relevant: 16/16 patients, 18/18 experts  *This scale was reviewed by 16/17 patients, 1 patient reviewed the Leg Symptom Scale only; 18/23 experts reviewed this scale (including parents).     151 Table 19: Flesch-Kincaid grade reading level of final version of items included in LIMB-Q Kids   Flesch-Kincaid (F-K) Reading Level Scale  Number of Items  Average  Range  Physical Function 32 0.15 0 to 0.9 Leg Symptoms 14 0.63 0 to 3.7 Hip Symptoms 10 0.26 0 to 0.8 Knee Symptoms 13 0.57 0 to 2.4 Ankle Symptoms 10 2.08 0 to 4.4 Foot Symptoms 9 0.71 0 to 2.4 Leg-Related Distress 14 2.29 0 to 4.9 Appearance 17 0.52 0 to 3.6 School Function 17 1.74 0 to 3.7 Social Function 12 1.81 0 to 3.7 Psychological Function 10 2.23 0 to 5.2 Scar Item 1 0.00 0 Total Items 159 1.04 0 to 5.2  *The F-K readability level indicates the grade-reading level of an item     152 Figure 1: Multiphase mixed methods protocol for development of PRO instruments  Flow diagram showing the multiphase mixed methods protocol for developing the CLEFT-Q. It is important to note that the process can be iterative and interactive as opposed to strictly linear. QUAN, quantitative study component; QUAL, qualitative study component. Figure reproduced with permission from the journal       153 Figure 2: PRISMA flow diagram     154 Figure 3: Preliminary conceptual framework developed as part of the systematic review     155 Figure 4: Final conceptual framework developed as part of the qualitative study    156 Bibliography 1.  Wilson GN. Heritable limb deficiencies. In: JA H, Birch J, editors. . Rosemont, IL: American Academy of Orthopaedic Surgeons; 1998. page 39–51. 2.  Lynn Staheli. Practice of Pediatric Orthopaedics. 2nd ed. Philadelphia: Lippincott Williams and Wilkins; 2006.  3.  Setoguchi Y, Rosenfelder R. The limb deficient child. Springfield, IL: 1982.  4.  Ephraim PL, Dillingham TR, Sector M, Pezzin LE, MacKenzie EJ. Epidemiology of limb loss and congenital limb deficiency: A review of the literature. 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Orthop Traumatol Surg Res 2014;100(1 S):S113-23.     180 Appendix A: Supplementary material for Chapter 2  Search Strategy for systematic review from Ovid Medline  Database: Ovid MEDLINE(R) In-Process & Other Non-Indexed Citations and Ovid MEDLINE(R) <1946 to Present> Search Strategy: -------------------------------------------------------------------------------- 1     Lower Extremity/ or Foot/ or Ankle/ or Hip/ or Knee/ or Leg/ or Thigh/ or lower limb?.mp. or lower extremit*.mp. or leg?.mp. or foot.mp. or feet.mp. or hip?.mp. or knee?.mp. or thigh?.mp. or shin?.mp. or ankle?.mp. (538494) 2     "Bones of Lower Extremity"/ or Foot Bones/ or Leg Bones/ or Femur/ or Femur Head/ or Femur Neck/ or Fibula/ or Tibia/ or foot bone?.mp. or leg bone?.mp. or femur?.mp. or femur head.mp. or femur neck.mp. or femoral.mp. or fibula?.mp. or tibia?.mp. or tibial.mp. or hip bone.mp. or coxal bone.mp. (236165) 3     1 or 2 [Lower Limbs] (669288) 4     arthritis, infectious/ (9514) 5     (Infectious arthritis or septic arthritis or postinfectious arthritis or post-infectious arthritis).mp. (4831) 6     4 or 5 [Arthritis] (11118) 7     bone diseases, infectious/ or osteitis/ or osteomyelitis/ or tuberculosis, osteoarticular/ (27255)  181 8     (infectious bone disease? or osteitis or bone inflammation or osteomyelitis or Osteoarticular Tuberculos#s or joint tuberculos#s or bone tuberculos#s).mp. (36631) 9     7 or 8 [Infectious Bone Disease] (40419) 10     6 or 9 [BACTERIAL INFECTIONS AND MYCOSES] (49585) 11     Neoplasms/ (353468) 12     (Neoplasm? or tumo?r? or cancer).mp. (3101002) 13     11 or 12 [Neoplasms] (3101002) 14     13 and 3 [Neoplasms AND Lower Limbs] (51911) 15     cysts/ or bone cysts/ or bone cysts, aneurysmal/ or synovial cyst/ (41698) 16     (Cyst? or bone cyst? or subchondral cyst? or intra-osseous gangli* or intraosseous gangli* or solitary cyst or aneurysmal bone cyst? or synovial cyst?).mp. (128929) 17     15 or 16 [Cysts] (128929) 18     17 and 3 [Cysts AND Lower Limbs] (5937) 19     Neoplasms, Bone Tissue/ or Fibroma, Ossifying/ or Osteoblastoma/ or Osteosarcoma/ (20040) 20     (bon# tissue neoplasm? or ossifying fibroma? or Osteoblastoma? or giant osteoid osteoma? or Osteosarcoma? or osteogenic sarcoma?).mp. (27043) 21     19 or 20 [Bone tissue neoplasms] (27300) 22     21 and 3 [Bone tissue neoplasms and lower limbs] (4101) 23     neoplasms by site/ or bone neoplasms/ or adamantinoma/ or femoral neoplasms/ (56761) 24     (bone neoplasm? or bone cancer or adamantinoma? or femoral neoplasm?).mp. (57586) 25     23 or 24 [Bone Neoplasms] (57586) 26     25 and 3 [Bone Neoplasms AND Lower Limbs] (12283)  182 27     exostoses, multiple hereditary/ or neurofibromatoses/ or neurofibromatosis 1/ or neurofibromatosis 2/ (11873) 28     (multiple hereditary exostos#s or diaphyseal aclasis or multiple cartilaginous exostos#s or multiple exostos#s or bessel-hagen disease or neurofibromatos#s or multiple neurofibroma?).mp. (15677) 29     27 or 28 [MHE neurofibroma] (16253) 30     14 or 18 or 22 or 26 or 29 [NEOPLASMS] (72474) 31     Musculoskeletal Diseases/ (10037) 32     (musculoskeletal disease? or musculoskeletal disorder?).mp. (13795) 33     31 or 32 [Musculoskeletal diseases] (13795) 34     Bone Diseases/ (20730) 35     (Bone disease? or bone disorder?).mp. (42094) 36     34 or 35 [Bone Diseases] (42094) 37     Bone Diseases, Developmental/ (5055) 38     (bone dysplasia? or developmental bone disease?).mp. (868) 39     37 or 38 [Developmental Bone Diseases] (5651) 40     acro-osteolysis/ or hajdu-cheney syndrome/ (174) 41     (Acro-Osteolysis or Acroosteolysis or hajdu-cheney syndrome or multicentric osteolysis).mp. (572) 42     40 or 41 [Acro-Osteolysis] (572) 43     dwarfism/ or achondroplasia/ or thanatophoric dysplasia/ or cockayne syndrome/ or congenital hypothyroidism/ or dwarfism, pituitary/ or laron syndrome/ or mulibrey nanism/ or weill-marchesani syndrome/ (14444)  183 44     (Dwarfism or short stature or nanism or Achondroplasia? or saddan? or Thanatophoric Dysplasia? or thanatophoric dwarfism? or Cockayne Syndrome or progeria-like syndrome? or Congenital Hypothyroidism or cretinism or pituitary dwarfism or Laron Syndrome or laron dwarfism or Mulibrey Nanism or perheentupa syndrome or Weill-Marchesani Syndrome or spherophakia brachymorphia syndrome? or GEMSS).mp. (25480) 45     43 or 44 [Dwarfism] (25480) 46     dysostoses/ or synostosis/ or antley-bixler syndrome phenotype/ or syndactyly/ (3877) 47     (Dysostos#s or Synostos#s or Antley-Bixler Syndrome Phenotype or antley-bixler syndrome or Syndactyl* or polysyndactyly).mp. (11533) 48     46 or 47 [Dysostoses] (11533) 49     Gigantism/ (1278) 50     gigantism.mp. (1780) 51     49 or 50 [Gigantism] (1780) 52     Leg Length Inequality/ (2714) 53     (Leg Length Inequalit* or leg length discrepanc*).mp. (3450) 54     52 or 53 [Leg Length Inequality] (3450) 55     Marfan Syndrome/ (5326) 56     Marfan? syndrome.mp. (6738) 57     55 or 56 [Marfan syndrome] (6738) 58     osteochondrodysplasias/ or camurati-engelmann syndrome/ or chondrodysplasia punctata/ or chondrodysplasia punctata, rhizomelic/ or ellis-van creveld syndrome/ or enchondromatosis/ or "fibrous dysplasia of bone"/ or fibrous dysplasia, monostotic/ or fibrous dysplasia, polyostotic/ or hyperostosis, cortical, congenital/ or kashin-beck disease/ or langer-giedion syndrome/ or  184 osteochondroma/ or osteochondromatosis/ or osteogenesis imperfecta/ or osteosclerosis/ or melorheostosis/ or osteopetrosis/ or osteopoikilosis/ or pycnodysostosis/ or slipped capital femoral epiphyses/ (22156) 59     (Osteochondrodysplasia? or skeletal dysplasia? or melnick needles syndrome or multiple epiphyseal dysplasia? or Camurati-Engelmann Syndrome or progressive diaphyseal dysplasia or engelmann disease or Chondrodysplasia Punctata or happle syndrome or Rhizomelic Chondrodysplasia Punctata? or Ellis-Van Creveld Syndrome or chondroectodermal dysplasia or Enchondromatos#s or maffucci syndrome or ollier? disease or Fibrous Dysplasia of Bone or bone fibrous dysplasia? or Monostotic Fibrous Dysplasia? or Polyostotic Fibrous Dysplasia? or Albright? syndrome or Hyperostosis, Cortical, Congenital or infantile cortical hyperostos#s or caffey? Disease or Kashin-Beck disease or Langer-Giedion Syndrome or Osteochondroma? or cartilaginous exostos#s or osteocartilaginous exostos#s or Osteochondromatos#s or Osteogenesis imperfecta or brittle bone disease or lobstein? Disease or Osteoscleros#s or Melorheostos#s or Osteopetrosi#s or marble bone disease or albers-schonberg disease or Osteopoikilos#s or Py?nodysostos#s or Slipped Capital Femoral Epiphys#s or slipped femoral capital epiphys#s).mp. (26231) 60     58 or 59 [Osteochondrodysplasias] (28650) 61     Proteus Syndrome/ (337) 62     proteus syndrome.mp. (497) 63     61 or 62 [Proteus syndrome] (497) 64     63 and 3 [Proteus syndrome AND Lower Limbs] (119) 65     Bone Diseases, Endocrine/ (40) 66     endocrine bone disease?.mp. (9)  185 67     65 or 66 [Endocrine bone diseases] (47) 68     Osteitis Fibrosa Cystica/ (1565) 69     (Osteitis Fibrosa Cystica or (recklinghausen? disease adj2 bone)).mp. (1678) 70     68 or 69 [Osteitis Fibrosa Cystica] (1678) 71     bone diseases, metabolic/ or bone demineralization, pathologic/ or decalcification, pathologic/ or mucolipidoses/ or osteoporosis/ or pseudohypoparathyroidism/ or pseudopseudohypoparathyroidism/ or renal osteodystrophy/ or rickets/ or osteomalacia/ or rickets, hypophosphatemic/ or familial hypophosphatemic rickets/ (56746) 72     (metabolic bone disease? or osteopenia? or pathologic bone demineralization or pathologic* decalcification or Mucolipidos#s or Osteoporos#s or Pseudohypoparathyroidism? or pseudopseudohypoparathyroidism? or renal osteodystroph* or renal rickets or rickets or rachitis or osteomalacia or Hypophosphatemic Rickets).mp. (90709) 73     71 or 72 [Metabolic bone disease] (93148) 74     bone malalignment/ or bone anteversion/ or coxa vara/ or bone retroversion/ or coxa valga/ (1558) 75     (bone malalignment? or bone misalignment? or bone malposition? or bone anteversion? or bone antetorsion? or coxa var#? or bone retroversion? or bone retrotorsion? or coxa valg#?).mp. (2252) 76     74 or 75 [Bone Malalignment] (2252) 77     Epiphyses, Slipped/ (1867) 78     (slipped epiphyses or epiphysiolys#s).mp. (570) 79     77 or 78 [Slipped Epiphyses] (2006) 80     genu valgum/ or genu varum/ (228)  186 81     (Genu valg#* or knock knee? or Genu varu? or bow leg? or tibia? torsion).mp. (1284) 82     80 or 81 [Genu Valgum/Genu Varum] (1284) 83     osteitis deformans/ or osteoarthropathy, primary hypertrophic/ or osteoarthropathy, secondary hypertrophic/ (7238) 84     (Osteitis Deformans or (paget? disease adj2 bone) or osseous paget? disease or Primary Hypertrophic Osteoarthropath* or hereditary acropach*or touraine solente gole syndrome or idiopathic hypertrophic osteoarthropath* or pachydermoperiostosis or Secondary Hypertrophic Osteoarthropath* or marie-bamberger disease).mp. (5818) 85     83 or 84 [Osteitis] (7712) 86     osteochondritis/ or osteochondritis dissecans/ or osteochondrosis/ (7061) 87     (Osteochondriti* or Osteochondritis Dissecans or Osteochondrosis or Koehler? disease or osgood-schlatter disease).mp. (8027) 88     86 or 87 [Osteochondritis] (8027) 89     osteonecrosis/ or femur head necrosis/ or legg-calve-perthes disease/ (12548) 90     (Osteonecros#s or kienbo?ck? disease or (bone adj2 necrosis) or (Femur Head adj2 Necros#s) or (necrosis adj2 femoral head) or Legg-Calve-Perthes Disease or coxa plana or perthes disease).mp. (17789) 91     89 or 90 [Osteonecrosis] (17789) 92     36 or 39 or 42 or 45 or 48 or 51 or 54 or 57 or 60 or 64 or 67 or 70 or 73 or 76 or 79 or 82 or 85 or 88 or 91 [Bone Diseases] (222011) 93     Foot Deformities/ or Foot Deformities, Acquired/ or Equinus Deformity/ (4152) 94     (Foot Deformit* or Acquired Foot Deformit* or Equinus Deformit* or equinus contracture?).mp. (7885)  187 95     93 or 94 [Foot Deformities] (7885) 96     Foot Diseases/ (11269) 97     Foot disease?.mp. (11622) 98     96 or 97 [Foot diseases] (11622) 99     joint diseases/ or ankylosis/ or arthralgia/ (32030) 100     (Joint disease? or Ankyloses or Arthralgia? or joint pain).mp. (44194) 101     99 or 100 [Joint pains] (47513) 102     osteoarthritis/ or osteoarthritis, hip/ or osteoarthritis, knee/ (48464) 103     (osteoarthritis* or (hip? adj2 osteoarthritis) or (knee? adj2 osteoarthritis)).mp. (63654) 104     102 or 103 [Arthritis] (63654) 105     arthrogryposis/ (1656) 106     Arthrogrypos#s.mp. (2201) 107     105 or 106 [Arthrogryposis] (2201) 108     contracture/ or hip contracture/ (7508) 109     (Contracture? or Hip Contracture?).mp. (22128) 110     108 or 109 [Contracture] (22128) 111     joint deformities, acquired/ or joint instability/ or joint loose bodies/ (17762) 112     (Acquired Joint Deformit* or Joint Instabilit* or joint laxit* or Joint Loose Bod*).mp. (17905) 113     111 or 112 [Joint deformities] (18894) 114     nail-patella syndrome/ (473) 115     (Nail-Patella Syndrome or osteo-onychodysplasias).mp. (531) 116     114 or 115 [Patella syndromes] (531)  188 117     101 or 104 or 107 or 110 or 113 or 116 [Joint diseases] (144382) 118     musculoskeletal abnormalities/ or campomelic dysplasia/ or hip dislocation, congenital/ (8321) 119     (Musculoskeletal abnormalit* or musculoskeletal disorder? or Camp?omelic Dysplasia? or camp?omelic syndrome? or Congenital Hip Dislocation?).mp. (7628) 120     118 or 119 [Musculoskeletal abn] (13797) 121     limb deformities, congenital/ or arachnodactyly/ or brachydactyly/ or ectromelia/ or lower extremity deformities, congenital/ or foot deformities, congenital/ or clubfoot/ or polydactyly/ (12713) 122     (Congenital Limb Deformit* or arachnodactyl* or Brachydactyl* or Ectromelia or hemimelia or Fibular hemimelia or Tibial hemimelia or Amelia or sirenomelia or phocomelia or Congenital Lower Extremit* Deformit* or Congenital Lower limb* Deformit* or Congenital Foot Deformit* or clubfoot or Club foot or congenital talipes equinovarus or talipes equinovarus or Polydactyl*).mp. (11669) 123     121 or 122 [Congenital Limb Deformities] (17163) 124     120 or 123 [Musculoskeletal Abnormalities] (30581) 125     33 or 92 or 95 or 98 or 117 or 124 [MUSCULOSKELETAL DISEASES] (397191) 126     "Wounds and Injuries"/ (67026) 127     (injur* or trauma?).mp. (961356) 128     126 or 127 [Wounds and Injuries] (961356) 129     128 and 3 [Wounds and Injuries AND Lower Limbs] (93302) 130     femoral fractures/ or hip fractures/ or femoral neck fractures/ or tibial fractures/ (43608)  189 131     (Femoral Fracture? or Hip Fracture? or trochanteric fracture? or Fem#r* Neck Fracture? or ((intertrochanteric or trochanteric or subtrochanteric) and fracture?) or Tibial Fracture?).mp. (50302) 132     130 or 131 [Lower Limb Fractures] (50302) 133     fractures, bone/ or fractures, closed/ or fractures, comminuted/ or fractures, malunited/ or fractures, open/ or fractures, ununited/ or pseudarthrosis/ (72916) 134     (bone fracture? or broken bone? or closed fracture? or comminuted fracture? or Malunited Fracture? or crossunited fracture? or open fracture? or compound fracture? or ununited fracture? or Pseud?arthros#s).mp. (21496) 135     133 or 134 [Bone Fractures] (82700) 136     135 and 3 [Bone Fractures AND Lower Limbs] (29088) 137     hip injuries/ (1818) 138     hip injur*.mp. (1940) 139     137 or 138 [Hip Injuries] (1940) 140     leg injuries/ (8544) 141     Leg injur*.mp. (8673) 142     140 or 141 [Leg Injuries] (8673) 143     abnormalities, radiation-induced/ (811) 144     radiation-induced abnormalit*.mp. (19) 145     143 or 144 [Radiation Injuries] (827) 146     129 or 132 or 136 or 139 or 142 or 145 [WOUNDS AND INJURIES] (139428) 147     Lower Extremity/gd, in, su [Growth & Development, Injuries, Surgery] (1693) 148     Foot/gd, su [Growth & Development, Surgery] (2910)  190 149     Hip/ab, gd, su [Abnormalities, Growth & Development, Surgery] (3056) 150     Knee/ab, gd, su [Abnormalities, Growth & Development, Surgery] (2658) 151     Leg/ab, gd, su [Abnormalities, Growth & Development, Surgery] (6120) 152     Thigh/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (1623) 153     ((lower limb? or lower extremit* or leg? or foot or feet or hip? or coxa? or knee? or thigh? or shin?) adj2 (malform* or defect* or abnormalit* or deformit* or injur* or disorder? or disease?)).mp. (60808) 154     147 or 148 or 149 or 150 or 151 or 152 or 153 [Lower Limb Regions Abnormalities] (75436) 155     "Bones of Lower Extremity"/ab, in, su [Abnormalities, Injuries, Surgery] (37) 156     Foot Bones/ab, gd [Abnormalities, Growth & Development] (48) 157     Leg Bones/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (205) 158     Femur/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (10853) 159     Femur Head/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (2647) 160     Femur Neck/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (952) 161     Fibula/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (3578) 162     Tibia/ab, gd, in, su [Abnormalities, Growth & Development, Injuries, Surgery] (10707) 163     ((foot bone? or leg bone? or femur? or femoral or fibula? or tibia? or tibial or hip bone or coxal bone) adj2 (malform* or defect* or abnormalit* or deformit* or injur* or disorder? or disease?)).mp. (4713)  191 164     155 or 156 or 157 or 158 or 159 or 160 or 161 or 162 or 163 [Bones of Lower Extremity Abnormalities] (28837) 165     154 or 164 [LOWER EXTREMITY ABNORMALITIES] (101016) 166     Orthopedic Procedures/ or Reconstructive Surgical Procedures/ or Bone Transplantation/ or Osteotomy/ or Osseointegration/ or Fracture Fixation/ or Fracture Fixation, Internal/ or Arthrodesis/ or Bone Screws/ (163247) 167     (orthop?edic procedure? or orthop?edic surgical procedure? or orthop?edic surger* or Reconstructive Surgical Procedure? or reconstructive surger* or Bone Transplant* or bone graft* or Osteotom* or Osseointegration or Fracture Fixation? or skeletal fixation? or internal fracture fixation? or fracture? osteosynthes#s or Arthrodes#s or allograft fusion or bone bridging or Bone Screw?).mp. (206558) 168     166 or 167 [General Orthopedic procedures] (206558) 169     168 and 3 [Orthopedic procedures AND lower limb] (69554) 170     Ilizarov Technique/ or Bone Lengthening/ or Artificial limbs/ or Prosthesis Fitting/ or Limb Salvage/ or Osteogenesis, Distraction/ (17413) 171     (Ilizarov Technique or ilizarov method or Bone Lengthening or Artificial limb? or artificial leg? or prosthes#s limb? or leg prosthes#s or artificial extremit* or Prosthesis Fitting? or Limb Salvage or distraction osteogenes#s or callotas#s).mp. (21115) 172     (Van Nes rotationplasty or Van Ness-Borggreve rotationplasty or rotationplasty or Ponseti treatment or limb reconstruction surgery or limb-sparing surgery or Limb preservation surgery or external fixator treatment or transtibial amputation or taylor spatial frame).mp. (1102) 173     exp Amputation/ or Amputation Stumps/ or Amputation, Traumatic/ or Amputees/ or (amputee* or amputat*).mp. (44060)  192 174     170 or 171 or 172 or 173 [Limb-related Orthopedic procedures] (59070) 175     169 or 174 [ORTHOPAEDIC PROCEDURES] (124073) 176     (Gait disorder? or gait abnormalit*).mp. (6030) 177     (Proximal femoral focal deficienc* or Proximal femoral deficienc* or Congenital Femoral Deficienc* or short femur or Congenital Pseudarthrosis of Tibia or Blount? disease).mp. (531) 178     (hemi-hypertrophy or hemihypertrophy or hemi hypertrophy or hemihyperplasia or hemi-hyperplasia or hemi hyperplasia or Beckwith-Wiedemann syndrome or Hemi-atrophy or hemiatrophy or hemi atrophy).mp. (3297) 179     177 or 178 [Misc 1] (3828) 180     arteriovenous malformations/ or arteriovenous fistula/ (19171) 181     (Arteriovenous malformation? or malformation? Arteriovenous or Arteriovenous Fistula?).mp. (35470) 182     180 or 181 [Arteriovenous malformation] (35470) 183     (Angular deformit* or Rotation* deformit*).mp. (1331) 184     ((limb? or extremit*) adj2 (defect* or malform* or deformit* or disorder? or disease? or abnormalit*)).mp. (9555) 185     183 or 184 [Misc 2] (10787) 186     Amniotic Band Syndrome/ (559) 187     (Amniotic Band Syndrome or amputation intrauterine or groove annular or amniotic band?).mp. (773) 188     186 or 187 [Amniotic Band Syndrome] (773) 189     176 or 179 or 182 or 185 or 188 [MISCELLANEOUS] (56515) 190     10 or 30 or 125 or 146 or 165 or 175 or 189 [DEFORMITIES] (719249)  193 191     Questionnaires/ (336930) 192     "Outcome Assessment (Health Care)"/ (54136) 193     Patient Outcome Assessment/ (1931) 194     (Assess* or Questionnaire? or Instrument? or outcome? or Measur* or Form? or Survey* or Evaluat* or Scale? or Score? or Report? or tool? or test?).mp. (11353849) 195     191 or 192 or 193 or 194 [ALL Questionnaires] (11353849) 196     (((self or patient* or parent*) adj1 (report* or percept* or perceiv* or perspect*)) or parent-prox* or self-evaluation or self-concept).mp. (227047) 197     195 and 196 [Questionnaires and Self-Report] (199875) 198     Diagnostic Self Evaluation/ (1482) 199     Self Report/ (13531) 200     (((((self or patient* or parent*) adj1 (report* or percept* or perceiv* or perspect*)) or parent-prox* or self-evaluation) adj3 (Assessment? or Questionnaire? or Instrument? or outcome? or Measure? or Form? or Survey? or Evaluation? or Scale? or Score? or Report? or tool? or test?)) or PROM or PROMs).mp. (88764) 201     198 or 199 or 200 [PROMs] (88764) 202     197 or 201 [All PROMs] (201364) 203     "Quality of Life"/ (134206) 204     (Quality of life or life quality* or QOL or Health related quality of life or HRQ?L or well-being or well being or life satisfaction).mp. (269433) 205     203 or 204 [Quality of Life] (269433) 206     190 and 202 and 205 [Deformities and PROMs and QOL] (1460)  194 207     Pediatrics/ or adolescent/ or child/ or child, preschool/ or infant/ or p?ediatric?.mp. or adolescen*.mp. (2881857) 208     206 and 207 [Deformities and PROMs and QOL and child] (288) 209     limit 208 to ed=20150827-20160131 [new papers from Aug 27 to Jan 31] (15)    195 List of PROMs Identified in the Systematic Review  # All PRO instruments used by 10 final papers  1 AAOS Lower Limb Outcomes  2 Child Behaviour Checklist CBCL/4-18 3 Child Health Questionnaire (CHQ PF-28) 4 Children's Assessment of Participation and Enjoyment (CAPE) 5 Children's Depression Inventory CDI 6 Children's Depression Inventory - Short CDI-S 7 Culture-Free Self-Esteem Inventory-2, Form B (CFSEI) 8 Dutch Self-Report Inventory for Children (Depression questionnaire for children) 9 Family Environment Scale -- Form R (FES) 10 Fast Performance Test on Intelligence I and II (SPIQ) 11 I think I am 12 Interview (not validated) 13 Kidcope 14 KIDSCREEN-52  15 Offer Self-Image Scale (OSIQ) 16 Pediatric Quality of Life (PedsQL) version 4.0 core scale 17 PedsQL NMM 3.0 18 Personal Adjustment and Role Skills Scale III (PARS III) 19 Pediatric Outcomes Data Collection Instrument (PODCI) 20 Post-Hospitalization Behavior Questionnaire (PHBQ) 21 Questionnaire (not validated) 22 Revised Children's Manifest Anxiety Scale (RCMAS) 23 Rosenberg Self-Esteem 24 Satisfaction (unvalidated) 25 SF-36 26 Specific LLD questionnaire (valid) 27 State Trait Anxiety Inventory for Children (STAIC) 28 State Trait Anxiety Inventory (STAI) - Form Y 29 Youth Self-Report 30 Zung Depression Scale for Adolescents    196 Appendix B: Supplementary material for Chapter 3  Child Interview Script This script will serve as a guide for interviewing children ages 8-18 years. Some questions might not be relevant for age group 8-13 years such as question 8 and 13.  Introduction Introduce self, Explain study; Read information sheet together Read through consent form together and sign if not done already. "As we go along, please ask questions at any time. Do you have any questions before we begin?" Questions/probes 1. Please tell me a little bit about yourself and your family 2. Tell me about your problems with your leg.  Probe: What are your biggest challenges with your leg? 3. Can you tell me about the treatment you received for your leg problem? 4. Was your treatment an overall positive or negative experience? 5. What were the best things about the treatment? 6. What were the biggest challenges with the treatment for you? 7. What were your expectations from the treatment? 8. What were you hoping to get out of your treatment? 9. How did the type of treatments offered meet or differ from your expectations? Probe: what do you expect/hope your leg to look like after treatment? 10. How do you think your leg problems have affected you?  11. How do you think your challenges with your leg have affected you?  197 Probe: Is there anything about your leg that bothers you? 12. How do you think has your leg problems affected how you look?  13. What do you currently like/dislike about the appearance of your leg? 14. What are your experiences of finding clothes appropriate for your leg problems? 15. What are your experiences of finding shoes/footwear appropriate for your leg problems? 16. Have you ever had problems finding appropriate clothes? Probe: wearing shorts, skirts or dresses? 17. Have you ever had problems finding appropriate footwear/shoes? 18. Is there anything that you would like to change about how your leg looks? 19. How important is your leg's appearance to you? 20. Do you worry about the future impact of the appearance of your leg? If so, how?  Probe for descriptive detail. 21. How does the appearance of your leg make you feel?  Probe: happy, sad, anxious, worried, frustrated, and self-conscious. 22. How do you feel about scars due to the frames/surgeries for your leg problems? 23. Did you ever have to wear shoe lifts/splints? 24. How do you feel about wearing them? Different footwear/shoe modifications? Probe: self-conscious about shoe raise? 25. How does the function of your leg make you feel?  Probe: happy, sad, anxious, worried, frustrated, and self-conscious. 26. How does your leg problem affect your function in day-to-day activities? 27. Do you worry about limping? 28. Do you worry about abnormal walking?  198 29. Do you worry about running? 30. What happens when someone new meets you for the first time? 31. How do you accomplish tasks compared to other kids of your age? 32. How do you feel your leg problems will affect your future function in day-to-day activities? 33. How has your leg problems affected how you feel? 34. Do you feel that someone with a lower limb problem has a better, worse, or same life as other people? 35. How has your leg problems affected your behavior? 36. How has your leg problems affected your self-esteem? 37. How has your leg problems affected your confidence? 38. How has your leg problems affected your school? Probes: Have you missed a lot of school due to surgeries, appointments? Ask about any issues with like concentrating or your attention or memory at school?  39. How do your friends react towards your leg problems?  Probes: For adolescents above 15 years only? Having a leg problem and being able to have intimate relationships? Have you had any particular negative experiences with guys/girls saying things to you? 40. How does your family react towards your leg problems? Probe: Supportive? How did they support? 42. How would you describe the attitudes of your friends and community? Probes: Supportive or not; how did they support you? Anything in particular they did to accommodate you  199 What support services help - and why?  If you were to kinda go out and just like be walking about, meeting people, um, have you felt like people have treated you the same, like strangers or have you felt quite judged and treated differently?  Have you used any other support services or anything or found comfort in any other, like engaging with people with leg problems? 43. Are there any activities/situations you avoid because of your leg problems? 44. What are your most challenging day to day activities with your leg problems? 45. Do you feel like having a leg problem has made your life better or worse or about the same as other people? 46. Is there anything else you would like to talk about your leg problems that we haven't covered so far? 47. Can you please tell me anything that bothers you about your leg, even if it’s minor and unimportant? Are you interested in talking to us again for the next step in this study?        200 Parent Interview Script This interview script is supposed to serve as a guide for interviewing families of children with lower limb deformities. Some of the question might not need to be asked as they would have been discussed already by the family during the discussion. Introduction Introduce self, explain study and read the information sheet together Read through consent form together and sign if not done already. "As we go along, please ask questions at any time. Do you have any questions before we begin?" Background  1. Please tell me a little bit about yourself and your family Lower Limb Specific Questions 2. Tell me about your experience of having a child with lower limb problems. 3. How would you describe your experience? Positive or negative Probe: why positive or negative? What made it positive or negative? How can we make it positive? 4. How do you perceive your child's physical appearance due to lower limb problems? 5. How does your child perceive his/her physical appearance due to lower limb problems? 6. What do you think your child currently like/dislike about the appearance of his/her leg? 7. What do you think your child currently like/dislike about the function of his/her leg? 8. Is there anything that you would like to change about how your child's leg looks? 9. How important is your child's leg's appearance to you? 10. Do you worry about the future impact of the appearance of your child's leg? If so, how? Probe for descriptive detail.  201 11. How does the appearance of your child's leg make you feel?  Probe: happy, sad, anxious, worried, frustrated, and self-conscious. 12. Do you worry about the future impact of the function of your child's leg? If so, how? Probe for descriptive detail. 13. How do you feel about scars due to the frames/surgeries for your child's leg problems? 14. Did your child ever have to wear shoe lifts/splints? 15. How do you think he/she feels about wearing them?  Probe: Different footwear/shoe modifications? Probe: self-conscious about shoe raise? 16. How does your child's leg problem affect his/her function in day-to-day activities? 17. Do you think your child worries about limping/abnormal walking/running? 18. How do you think your child accomplishes tasks compared to other kids of your age? 19. How do you feel your child's leg problems will affect his/her future function in day-to-day activities? Experience of care  20. Is there anything you would like to comment about with regard to their hospital treatment? 21. Is there anything that could have been done better? 22. Is there anything that you wished health professionals knew or understood better about having a child with a lower limb problems? 23. Is there anything else that comes to your mind that you would like to talk about your experience of care? Quality of life Specific Questions  202 24. What does Quality of Life means to you? 25. How would you describe your child's quality of life? 26. How do you think having a lower limb problem has affected your child's quality of life in general? 27. How has the lower limb problem affected your child's school? 28. How has the lower limb problem affected your child's Social life? 29. How has the lower limb problem affected your child's emotions? 30. How has the lower limb problem affected your child's behavior? 31. How has the lower limb problem affected your child's mental health? 32. How has the lower limb problem affected your child's self-esteem? 33. How has the lower limb problem affected your child's confidence? 34. How do you think we can improve the quality of life of your child? 35. How do you think your child perceives him/herself? 36. How do his/her friends react towards his/her lower limb problems? 37. How would you describe the attitudes of your extended family? 38. How would you describe the attitudes of your friends and community? 39. Are there any activities/situations you avoid because of your child's lower limb problems? 40. Is there anything else you would like to talk about your child's quality of life that we haven't covered above? 41. Can you please tell me anything that bothers you about your child's leg, even if it’s minor and unimportant? Parental QOL  203 42. How do you think having a child with limb problem has affected your life? 43. Family time 44. Emotionally 45. Social life 46. Friends 47. Financially 48. Do you feel the need for more support? Are you interested in us contacting you again for cognitive interviews once we have the preliminary scale developed?     204 Table: Saturation table showing the number of interviews endorsing each theme and sub-theme  Interviews (N=79)     Canada Site  1  USA  Canada Site  2  Total (%) India  Ethiopia  Total (%) Themes Sub-themes 1-5 6-10  11-16 17-21 22-26 27-31 32-37 38-42 43-47   48-52 53-57 58-62 63-67 68-72 73-77 78-79   Appearance Appearance of body parts and scars 4 4 3 1 3 2 3 4 3 27 (47) 4 3 5 3 3 1 0 19 (59) Appearance Clothes 1 1 2 2 0 0 1 0 2 9 (17) 3 1 4 3 3 1 1 16 (50) Appearance Shoes and other devices 1 2 2 1 0 0 3 0 0 9 (17) 0 1 3 1 1 0 0 6 (19) Physical Health Physical Function 4 2 3 1 2 2 4 3 2 23 (40) 2 3 2 3 3 4 2 19 (59) Physical Health Symptoms 1 3 2 1 1 1 1 2 3 15 (30) 1 3 2 2 2 2 2 14(44) School Function 1 1 3 0 0 0 2 0 0 7 (13) 1 1 1 3 3 4 1 14 (44) School Participation 1 3 2 1 0 0 2 1 1 11 (21) 2 2 2 2 2 1 1 12(38) School Isolation 3 2 1 0 0 0 1 0 0 7 (9) 2 0 2 0 0 0 0 4(13) School Environmental Barriers 0 0 0 0 0 0 0 0 0 0 0 0 0 1 1 0 0 2(6)  205 Social Social Function 2 1 1 1 0 1 1 3 2 12 (21) 3 2 2 2 2 3 2 16(50) Interviews (N=79)     Canada Site  1  USA  Canada  Site  2  Total (%) India  Ethiopia  Total (%) Social Isolation 2 2 3 1 2 1 2 1 1 15(28) 3 4 3 4 4 3 1 22(69) Psychological Health Body Image 2 4 3 1 1 1 2 3 0 17(32) 2 2 3 3 3 2 1 16(50) Psychological Health Distress 2 2 4 2 0 2 2 3 2 19 (36) 3 2 2 3 3 3 1 17(53) Psychological Health Confidence and self-esteem 3 0 2 1 0 0 1 3 0 10(15) 1 0 0 3 3 1 1 9(28) Mediating Factors Coping  0 1 3 0 0 0 0 1 0 5 (11) 1 0 0 0 0 0 0 1(3) Emotional Support  2 2 2 0 0 0 0 2 0 8 (17) 2 0 2 1 3 1 0 9(28) Instrumental Support  2 1 2 0 0 0 1 2 1 9 (19) 1 1 2 1 1 1 1 8(25)   206 Figure A proposed model showing the relationship of concepts with each other and HRQL     207 Appendix C: Supplementary material for Chapter 4  LIMB-Q Kids Version 1 for cognitive debriefing interviews and expert feedback.  Physical Function Scale  How easy is it to use your leg? Answer each question by circling one number.  Please answer thinking of the past week. Note: Do you have problems with both legs? If yes, please answer thinking of the leg that is worse. Are you able to: I CANNOT do this I have A LOT of trouble doing this I have A LITTLE trouble doing this I CAN do this 1. …get out of bed? 1 2 3 4 2. …sit in a chair? 1 2 3 4 3. …get up from a chair? 1 2 3 4 4. …sit cross-legged? 1 2 3 4 5. …get up from the floor? 1 2 3 4 6. …lift your leg (e.g. to kick a ball)? 1 2 3 4 7. …bend your knee? 1 2 3 4 8. …pick something up from the floor? 1 2 3 4 9. …walk without limping? 1 2 3 4 10.  …walk without falling? 1 2 3 4 11.  …walk as fast as you like? 1 2 3 4 12.  …walk as far as you like? 1 2 3 4  208 13.  …walk up stairs? 1 2 3 4 14.  …walk down stairs? 1 2 3 4 15.  …walk while you carry something (e.g. backpack, books)? 1 2 3 4 16.  …walk up a hill? 1 2 3 4 17.  …walk down a hill? 1 2 3 4 18.  …stand for a long time? 1 2 3 4 19.  …climb (e.g. up a ladder)? 1 2 3 4 20.  …run? 1 2 3 4 21.  …run without falling? 1 2 3 4 22.  …run as fast as you want? 1 2 3 4 23.  …run as far as you want? 1 2 3 4 24.  …ride a bike? 1 2 3 4 25.  …play sports you like? 1 2 3 4 26.  …jump? 1 2 3 4 27.  …put on or take off shoes? 1 2 3 4 28.  …put on or take off clothes? 1 2 3 4   209 Leg Symptoms Scale  How does your leg (s) feel? Answer each question by circling one number. Please answer thinking of the past week.  Note: Do you have problems with both legs? If yes, please answer thinking of the leg that is worse.   Never Sometimes Often Always 1. My leg hurts when I am resting or relaxing.  1 2 3 4 2. My leg is puffy or swollen (bigger than normal). 1 2 3 4 3. My leg hurts when I walk.  1 2 3 4 4. My leg hurts when I run. 1 2 3 4 5. My leg gets tired when I walk. 1 2 3 4 6. My leg gets tired when I run. 1 2 3 4 7. My leg feels weak.  1 2 3 4   210 Foot Symptoms Scale  How does your foot feel? Answer each question by circling one number. Please answer thinking of the past week.  Note: Do you have problems with both feet? If yes, please answer thinking of the foot that is worse.   Never Sometimes Often Always 1. My foot hurts. 1 2 3 4 2. My foot is puffy or swollen (bigger than normal). 1 2 3 4 3. My foot hurts when I walk.  1 2 3 4 4. My foot hurts when I run.  1 2 3 4 5. My foot gets tired when I walk. 1 2 3 4 6. My foot gets tired when I run. 1 2 3 4   211 Hip Symptoms Scale  How does your hip feel? Answer each question by circling one number. Please answer thinking of the past week. Note: Do you have problems with both hips? If yes, please answer thinking of the hip that is worse.  Never Sometimes Often Always 1. My hip hurts. 1 2 3 4 2. My hip clicks when I move.  1 2 3 4 3. My hip locks (gets stuck).  1 2 3 4 4. My hip gets tired. 1 2 3 4 5. My hip makes a popping sound when I move. 1 2 3 4    212 Knee Symptoms Scale  How does your knee feel? Answer each question by circling one number. Please answer thinking of the past week.  Note: Do you have problems with both knees? If yes, please answer thinking of the knee that is worse.  Never Sometimes Often Always 1. My knee hurts. 1 2 3 4 2. My knee clicks when I move. 1 2 3 4 3. My knee locks (gets stuck).  1 2 3 4 4. My knee gives way when I move (feels wobbly).  1 2 3 4 5. My knee makes a popping sound when I move. 1 2 3 4  213 Leg-Related Distress Scale  How do you feel about your leg (s)? Answer each question by circling one number. Please answer thinking of the past week.   Never Sometimes Often Always 1. I cover up or hide my leg when I go out.  1 2 3 4 2. I avoid wearing shorts or skirts.  1 2 3 4 3. I dislike how my leg looks. 1 2 3 4 4. I feel unhappy about my leg. 1 2 3 4 5. I worry about my leg.  1 2 3 4 6. I get upset when people look at my leg. 1 2 3 4 7. I get upset when people ask about my leg. 1 2 3 4 8. I avoid having photos taken that show my leg. 1 2 3 4 9. I avoid going out because of my leg (like to a party). 1 2 3 4 10.  I dislike how I look when I walk (e.g. limp). 1 2 3 4 11.  I feel different from other people because of my leg.  1 2 3 4 12.  I get teased about my leg.  1 2 3 4 13.  I get upset when I can’t join activities and games. 1 2 3 4 14.  I get upset when I can’t wear different types of shoes. 1 2 3 4 15.   I feel unhappy about the scars on my leg.  1 2 3 4     214 Appearance Scale  How does your leg (s) look? Answer each question by circling one number. Please answer thinking of how your leg looks now.  Note: Do your legs look different from each other? If yes, please answer thinking of the leg that you like the least. How much do you like… Not at all A little bit Quite a bit Very much 1. …the length of your leg? 1 2 3 4 2. …the size of your leg (not too skinny)? 1 2 3 4 3. …how your leg looks when you walk? 1 2 3 4 4. …how your leg looks when you run? 1 2 3 4 5. …how your leg looks in shorts or a skirt? 1 2 3 4 6. …how your leg looks when you wear pants or jeans? 1 2 3 4 7. …how your leg looks in photos? 1 2 3 4 8. …how your leg looks when you stand?  1 2 3 4 9. …how your leg looks when you sit on the floor?  1 2 3 4 10.  …how your leg looks when you sit in a chair? 1 2 3 4 11.  …how your leg looks compared with other people’s legs? 1 2 3 4 12.  …how well your legs match each other (look the same)? 1 2 3 4 13.  …how straight your leg looks (not crooked or lopsided)? 1 2 3 4 14.  …how your foot looks? 1 2 3 4 15.  …how your knee looks? 1 2 3 4  215 16.  …how well your hips match each other (straight or tilted)? 1 2 3 4 17.   …how your leg looks overall? 1 2 3 4 18.  …how the scars on your leg look? 1 2 3 4     216 School Function Scale How is your school life? Answer each question by circling one number. Please answer thinking of the past week.  Note: if you were not in school this week, think about when you were last in school.  Never Sometimes Often Always 1. I like seeing my friends at school. 1 2 3 4 2. My teachers help me at school. 1 2 3 4 3. I feel accepted at school.  1 2 3 4 4. Other students like me.  1 2 3 4 5. I am happy at school.  1 2 3 4 6. Other students are nice to me. 1 2 3 4 7. Other students listen to me when I talk. 1 2 3 4 8. Other students help me at school. 1 2 3 4 9. I feel safe at school (not bullied because of my leg).  1 2 3 4 10.  It’s easy for me to make friends.  1 2 3 4 11.   I get asked to join activities and games at school.  1 2 3 4 12.  I fit in at school.  1 2 3 4 13.  I like going to school. 1 2 3 4 14.  It’s easy for me to get to my classes at school. 1 2 3 4 15.  I am able to attend school. 1 2 3 4 16.  I forget about my leg problem when I am at school. 1 2 3 4 17.  I am ok if students ask about my leg. 1 2 3 4  217 Social Function Scale   How is your social life? Answer each question by circling one number. Please answer thinking of the past week.   Never Sometimes Often Always 1. My friends accept me.  1 2 3 4 2. I have fun with friends.  1 2 3 4 3. People listen to what I have to say.  1 2 3 4 4. People treat me the same as everyone else.  1 2 3 4 5. I like being with other people.  1 2 3 4 6. I feel confident when I go out (like to a party). 1 2 3 4 7. I get asked to go out with friends (like to a party). 1 2 3 4 8. I feel like I fit in. 1 2 3 4 9. It’s easy for me to make new friends.  1 2 3 4 10.  I feel the same as other people my age. 1 2 3 4 11.  It’s okay when people look at my leg.  1 2 3 4 12.  It’s okay if people ask me about my leg.  1 2 3 4 13.  My friends treat me the same as others. 1 2 3 4    218 Psychological Function Scale  How do you feel? Answer each question by circling one number. Please answer thinking of the past week.   Never Sometimes Often Always 1. I am happy with my life. 1 2 3 4 2. I enjoy life.  1 2 3 4 3. I feel happy. 1 2 3 4 4. I feel okay about myself.  1 2 3 4 5. I believe in myself. 1 2 3 4 6. I am proud of myself. 1 2 3 4 7. I like myself. 1 2 3 4 8. I feel confident.  1 2 3 4 9. I feel great about myself. 1 2 3 4 10.  I feel good about how I look. 1 2 3 4      219 Appendix D: Supplementary material for Chapter 5  Cognitive Interview Guide: This was used as a guide for the cognitive debriefing interviews. Questions were modified and probes were used as needed depending upon the age of the patient.  About the study: We are working to develop a set of questions called a patient-reported outcome questionnaire for patients with lower limb problems. We have interviewed children with lower limb problems and their parents from different sites in Canada, India, Ethiopia and the USA. Based on these interviews, we have come up with a list of questions that will form the new questionnaire. These questions represent (are about) what is important to children with lower limb problems. Right now, we are taking these questions back to the children to get their feedback and know if they are able to understand the questions and if there is anything that we have missed in the questions but is important to the children. Your surgeon wants a questionnaire (set of questions) to be able to know how you are doing with your leg problems. We want to know if the questions we have written work for you. We need your help with these questions. We need you to look at the questions and response options (answers) and tell us what is missing. Tell us what questions work for you and what questions are difficult to understand Draft of cognitive debriefing interview script Part 1: Instructions 1. What are the instructions at the top asking you to do (please explain to me in your own words)? There are a few lines before the actual questions. These lines are telling you about what to do and how to answer these questions. 2. What days do you include when you think about the past week?  220 3. Are there any words we should change to make the instructions easier? 4. At the end of each scale:  Which items resonate more? Or which items do you think are related to your own experience with your leg problems? Or which items are more important to you?  Which items resonate less? Or which items do you think are related to your own experience with your leg problems? Or which items are more important to you?  Items difficult to answer in general.  Items easy to answer? Clarifications – Physical Function Scale   1. Ask about past week. Is this easy to understand? Would you like us to use a different word instead such as last week? 2. Which leg are you thinking about right now? 3. Are you using any special device at the moment? 4. What do you understand by cross-legged? Symptoms Can you think of an alternate word for ‘hurt’? Is ‘sore’ a better word? Are they the same or different words to you? Symptoms –Hip Probe for hip ‘click’   What do you understand by the word ‘click’? What does ‘click’ mean to you? Has this ever happened to you? Do you think we should use a different word instead of ‘click’? Probe for hip gets stuck   221  What do you understand by the word ‘stuck’? What does ‘stuck’ mean to you? Has this ever happened to you? Do you think we should use a different word instead of stuck? Probe for hip makes a ‘popping’ sound when I move   What do you understand by the word ‘popping’? What does ‘pop’ mean to you? Has this ever happened to you? Do you think we should use a different word instead of ‘pop’? Appearance Scale Ask which leg they are thinking about when looking at the instructions at the top. School Function scale Ask at the end if they were thinking about their leg while answering these questions about school? Social Function Scale Ask at the end if they were thinking about their leg while answering these questions about social life? Psychological Function Scale Ask at the end if they were thinking about their leg while answering these questions? Part 2: Items  (Say the questions to the participant and ask)What do you think the question is asking?  Can you please say the question in your own words?  What do you think of when answering this question?  Is (are) any word(s) difficult to understand?  What other word should we use?  Is (are) any question(s) hard to answer?   If so, why?  222  How can we change the question so that it is easier for you to understand?  Would you say that the question itself is difficult to understand?  Do you think any of the questions need clarification?  Does this question talk about something that is important for you?  What was your answer for this question?  Why did you choose that answer? Part 3: General questions to ask at the end of each group of questions  1. Can you describe in your own words what this group of questions is asking about? 2. Does this group of questions talk about something that is important for you? 3. Are there any questions that do not ‘belong’ with the rest? 4. What items/questions don’t work for you? 5. Thinking about this group of questions, do you think we are missing something? 6. What is missing from this set of questions/scale to capture your experience? 7. What do you think about the response choices/answer options for these questions? 8. Can you tell me which items resonate more with your experience? Which items are more important to you? (alternate wording) Part 4: General questions to ask at the end of the interview 1. What do you think about these questions? 2. Is there anything we forgot to ask that is important to you with leg problems? 3. Is there anything we should change about this questionnaire/survey? 4. Is there anything else that you would like to add or comment upon?  223 Guide for interviews with the experts   These questions served as a guide to conduct one-on-one interviews with the experts. Probes will be used as needed during the conversation.  Are children with lower limb deformities the main focus if your practice?  Are adults with lower limb deformities the main focus if your practice?  These nine questions will be asked at the end of each of the scales.   1. Do you have any feedback about the instructions for this scale?  2. Are there any items we should add?  3. Are there any items we should remove?  4. Do you have any feedback for the response options?  5. Do you have any feedback for the recall period for this scale?  6. Do you think that the items in this scale are relevant to the patients with lower limb deformities?  7. Any items you think are not relevant and should be removed?  8. Do you find any words or items that you think will be difficult for your patients to understand?  9. Do you have any suggestions for rewording any items?  10. Do you have any other feedback for this scale that is not covered in the questions above?  11. Will these items measure change in HRQL after treatment?  12. Which items do you think will be best at measuring change?  13. Which items will not measure change?  14. Do you think you will use this new PROM in your practice?  224 LIMB-Q Kids Final version Physical Function Scale  How easy is it to use your leg (s)? Answer each question by circling one number. Please answer thinking of the past week. These questions ask about the leg you are seeing the doctor or nurse about.  If you wear a special shoe, a shoe-lift or a brace or have an artificial leg (prosthetic leg), please answer the questions thinking of when you have them on.  Are you able to:  I CANNOT do this I have A LOT of trouble doing this I have A LITTLE trouble doing this I CAN do this 1. …get out of bed? 1 2 3 4 2. …sit in a chair? 1 2 3 4 3. …get up from a chair? 1 2 3 4 4. …sit on a toilet?  1 2 3 4 5. …sit cross-legged on the floor? 1 2 3 4 6. …get up from the floor? 1 2 3 4 7. …lift one leg while you stand on the other? 1 2 3 4 8. …bend your knees? 1 2 3 4 9. …pick a book up from the floor? 1 2 3 4 10.  …walk without a limp? 1 2 3 4 11.  …walk and not fall down? 1 2 3 4 12.   …walk on a bumpy road? 1 2 3 4 13.  …walk as fast as you like? 1 2 3 4 14.  …walk as far as you like? 1 2 3 4 15.  …walk up the stairs? 1 2 3 4 16.  …walk down the stairs? 1 2 3 4 17.  …walk while you carry a book? 1 2 3 4 18.  …walk up a hill? 1 2 3 4 19.  …walk down a hill? 1 2 3 4 20.  …stand for a long time? 1 2 3 4 21.  …climb (such as up a ladder)? 1 2 3 4 22.  …run? 1 2 3 4 23.  …run and not fall down? 1 2 3 4 24.  …run as fast as you want? 1 2 3 4 25.  …run as far as you want? 1 2 3 4  225 26.  …ride a bike? 1 2 3 4 27.  …play sports you like? 1 2 3 4 28.  …jump? 1 2 3 4 29.   …put on or take off shoes? 1 2 3 4 30.  …put on or take off clothes? 1 2 3 4 31.  …jump up and down on one leg?  1 2 3 4 32.  …stand on one leg? 1 2 3 4     226 Leg Symptoms Scale  How does your leg feel? Answer each question by circling one number. Please answer thinking of the past week. These questions ask about the leg you are seeing the doctor or nurse about.    Never Sometimes Often Always 1. My leg hurts when I rest or relax. 1 2 3 4 2. My leg hurts when I sleep. 1 2 3 4 3. My leg hurts when I stand for a long time. 1 2 3 4 4. My leg hurts when I walk.  1 2 3 4 5. My leg hurts when I run. 1 2 3 4 6. My leg hurts when I touch it. 1 2 3 4 7. My leg gets tired when I walk. 1 2 3 4 8. My leg gets tired when I run. 1 2 3 4 9. My leg feels weak (shaky).  1 2 3 4 10.  My leg feels numb (cannot feel it). 1 2 3 4 11.  My leg feels tingly (pins and needles feeling). 1 2 3 4 12.  My leg is swollen or puffy. 1 2 3 4 13.  My leg feels itchy. 1 2 3 4 14.  My leg has a rash (looks red). 1 2 3 4     227 Hip Symptoms Scale  How does your hip feel? Answer each question by circling one number. Please answer thinking of the past week. These questions ask about the hip that is part of the leg you are seeing the doctor or nurse about.    Never Sometimes Often Always 1. My hip hurts when I rest or relax. 1 2 3 4 2. My hip hurts when I sit for a long time. 1 2 3 4 3. My hip hurts when I stand for a long time. 1 2 3 4 4. My hip hurts when I walk. 1 2 3 4 5. My hip hurts when I walk up the stairs. 1 2 3 4 6. My hip hurts when I walk down the stairs. 1 2 3 4 7. My hip hurts when I run.  1 2 3 4 8. My hip hurts when it makes a sound (clicks or pops). 1 2 3 4 9. My hip gets stuck (not able to move it).  1 2 3 4 10.  My hip feels weak (shaky). 1 2 3 4    228 Knee Symptoms Scale  How does your knee feel? Answer each question by circling one number. Please answer thinking of the past week. These questions ask about the knee on the leg you are seeing the doctor or nurse about.   Never Sometimes Often Always 1. My knee hurts when I rest or relax. 1 2 3 4 2. My knee hurts when I stand for a long time. 1 2 3 4 3. My knee hurts when I walk.  1 2 3 4 4. My knee hurts when I walk up the stairs. 1 2 3 4 5. My knee hurts when I walk down the stairs. 1 2 3 4 6. My knee hurts when I run.  1 2 3 4 7. My knee hurts when I bend it. 1 2 3 4 8. My knee hurts when I straighten it. 1 2 3 4 9. My knee hurts when it makes a sound (clicks or pops). 1 2 3 4 10. My knee gets stuck (not able to move it).  1 2 3 4 11. My knee feels wobbly (like it is going to give out).  1 2 3 4 12. My knee feels weak (shaky). 1 2 3 4 13. My knee is swollen or puffy. 1 2 3 4    229 Ankle Symptoms Scale  How does your ankle feel? Answer each question by circling one number. Please answer thinking of the past week.  These questions ask about the ankle on the leg you are seeing the doctor or nurse about.      Never Sometimes Often Always 1. My ankle hurts when I rest or relax. 1 2 3 4 2. My ankle hurts when I stand for a long time. 1 2 3 4 3. My ankle hurts when I walk.  1 2 3 4 4. My ankle hurts when I walk on a bumpy road.  1 2 3 4 5. My ankle hurts when I run.  1 2 3 4 6. My ankle hurts when it makes a sound (clicks or pops). 1 2 3 4 7. My ankle gets stuck (not able to move it).  1 2 3 4 8. My ankle feels wobbly (like it is going to give out). 1 2 3 4 9. My ankle feels weak (shaky). 1 2 3 4 10.   My ankle is swollen or puffy. 1 2 3 4  230 Foot Symptoms Scale  How does your foot feel? Answer each question by circling one number. Please answer thinking of the past week.  These questions ask about the foot on the leg you are seeing the doctor or nurse about.   Never Sometimes Often Always 1. My foot hurts when I rest or relax. 1 2 3 4 2. My foot hurts when I stand for a long time. 1 2 3 4 3. My foot hurts when I walk on a bumpy road. 1 2 3 4 4. My foot hurts when I run.  1 2 3 4 5. My foot hurts when I walk with shoes on. 1 2 3 4 6. My foot hurts when I walk bare foot (no shoes on my feet). 1 2 3 4 7. My foot gets stuck (not able to move it). 1 2 3 4 8. My foot feels weak (shaky). 1 2 3 4 9. My foot is swollen or puffy. 1 2 3 4    231 Leg-Related Distress Scale  How do you feel about your leg? Answer each question by circling one number. Please answer thinking of the past week.  These questions ask about the leg you are seeing the doctor or nurse about. If you wear a special shoe, a shoe-lift, a brace or have an artificial leg (prosthetic leg), please answer the questions thinking of when you have them on.   Never Sometimes Often Always 1. I hide my leg when I go out.  1 2 3 4 2. I avoid wearing shorts or skirts that show my leg.  1 2 3 4 3. I dislike how my leg looks. 1 2 3 4 4. I feel unhappy about my leg. 1 2 3 4 5. I worry about my leg.  1 2 3 4 6. I get upset when people look at my leg. 1 2 3 4 7. I get upset when people ask about my leg. 1 2 3 4 8. I dislike photos that show my leg. 1 2 3 4 9. I avoid going out because of my leg (like to a party). 1 2 3 4 10.  I dislike how I look when I walk. 1 2 3 4 11.  My leg makes me feel different from other people.  1 2 3 4 12.  I get teased about my leg.  1 2 3 4 13.  I get upset when my leg stops me from having fun. 1 2 3 4 14.  I get upset when I am not able to wear the type of shoes I like. 1 2 3 4     232 Appearance Scale  How does your leg look? Answer each question by circling one number. Please answer thinking of how your leg looks now.  These questions ask about the leg you are seeing the doctor or nurse about. If you wear a special shoe, a shoe-lift, a brace or have an artificial leg (prosthetic leg), please answer the questions thinking of when you have them on.  How much do you like… Not at all A little bit Quite a bit Very much 1. …how your leg looks when you walk? 1 2 3 4 2. …how your leg looks when you run? 1 2 3 4 3. …the length of your leg? 1 2 3 4 4. …the size of your leg? 1 2 3 4 5. …how your leg looks when you wear shorts or a skirt? 1 2 3 4 6. …how your leg looks when you wear pants or jeans? 1 2 3 4 7. …how your leg looks in photos? 1 2 3 4 8. …how your leg looks when you stand?  1 2 3 4 9. …how your leg looks when you sit on the floor?  1 2 3 4 10.  …how your leg looks when you sit in a chair? 1 2 3 4 11.  …how your leg looks compared with other peoples’ legs? 1 2 3 4 12.  …how well your legs match each other (look the same)? 1 2 3 4 13.  …how straight your leg looks? 1 2 3 4 14.  …how your foot looks? 1 2 3 4 15.  …how your knee looks? 1 2 3 4 16.  …how well your hips match each other (straight or uneven)? 1 2 3 4 17.   …how your leg looks overall? 1 2 3 4     233 Do you have scars from your leg surgery? If yes, please answer each question by circling one number.  Please answer thinking of how your scars look now.  This question asks about the leg you are seeing the doctor or nurse about.    How much do you like… Not at all A little bit Quite a bit Very much 1. …how the scars on your leg look? 1 2 3 4  234 School Function Scale  How is your school life? Answer each question by circling one number. Please answer thinking of the past week.  If you were not in school this week, think about when you were last in school.  Never Sometimes Often Always 1. I like seeing my friends at school. 1 2 3 4 2. My teachers help me at school. 1 2 3 4 3. I feel accepted at school.  1 2 3 4 4. Other students like me.  1 2 3 4 5. I am happy at school.  1 2 3 4 6. Other students are nice to me. 1 2 3 4 7. Other students listen to me when I talk. 1 2 3 4 8. Other students help me at school. 1 2 3 4 9. I feel safe at school (not bullied). 1 2 3 4 10.  It is easy for me to make friends.  1 2 3 4 11.  I get asked to join activities and games at school.  1 2 3 4 12.  I fit in at school.  1 2 3 4 13.  I like going to school. 1 2 3 4 14.  It is easy for me to get to my classes in school. 1 2 3 4 15.  I forget about my leg when I am at school. 1 2 3 4 16.  I am ok if students ask about my leg. 1 2 3 4 17.  My teachers treat me well. 1 2 3 4     235 Social Function Scale  How is your social life? Answer each question by circling one number. Please answer thinking of the past week.    Never Sometimes Often Always 1. My friends accept me.  1 2 3 4 2. I have fun with friends.  1 2 3 4 3. People listen to what I have to say.  1 2 3 4 4. People treat me the same as everyone else. 1 2 3 4 5. I like being with other people.  1 2 3 4 6. I feel confident when I go out (like to a party). 1 2 3 4 7. I get asked to go out with friends. 1 2 3 4 8. I feel like I fit in. 1 2 3 4 9. It is easy for me to make friends.  1 2 3 4 10.  I feel the same as other people my age. 1 2 3 4 11.  It is okay when people look at my leg.  1 2 3 4 12.  It is okay if people ask me about my leg.  1 2 3 4    236 Psychological Function Scale  How do you feel? Answer each question by circling one number. Please answer thinking of the past week.   Never Sometimes Often Always 1. I am happy with my life. 1 2 3 4 2. I enjoy life.  1 2 3 4 3. I feel happy. 1 2 3 4 4. I feel okay about myself.  1 2 3 4 5. I believe in myself. 1 2 3 4 6. I am proud of myself. 1 2 3 4 7. I like myself. 1 2 3 4 8. I feel confident.  1 2 3 4 9. I feel great about myself. 1 2 3 4 10.  I feel good about how I look. 1 2 3 4     237 Appendix E: A link to the protocol manuscript https://bmjopen.bmj.com/content/9/5/e027079 

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