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Charting the Territory : Symptoms and Functional Assessment in Children With Progressive, Non-Curable… Steele, Rose; Siden, Harold; Cadell, Susan; Davies, Betty; Andrews, Gail; Feichtinger, Leanne; Singh, Mina 2014

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1 Charting the Territory: Symptoms and Functional Assessment in Children With Progressive, Non-Curable Conditions  Rose Steelea, RN, PhD; Harold Sidenb,c, MD, MHSc, FRCP; Susan Cadelld, RSW, PhD; Betty Daviese, RN, PhD, FAAN, Gail Andrewsc, MEd; Leanne Feichtingerc, BSc; Mina Singha, RN, PhD  Affiliations: aSchool of Nursing, Faculty of Health, York University, Toronto, Ontario, Canada; bDepartment of Paediatrics, University of British Columbia; Canuck Place Children’s Hospice, Vancouver, British Columbia, Canada; c Developmental Neuroscience and Child Health, Child & Family Research Institute, Vancouver, British Columbia, Canada; dSchool of Social Work, Renison University College – University of Waterloo, Waterloo, Ontario, Canada; eSchool of Nursing, University of Victoria, Victoria, British Columbia, Canada  Corresponding Author: Rose Steele, Professor, School of Nursing, Faculty of Health, York University, Room 342 HNES Building, 4700 Keele Street, Toronto, ON M3J 1P3 Canada. Email: rsteele@yorku.ca; telephone: (416) 736-2100 ext. 40556; fax: (416) 736-5714.  Key Words: Palliative Care; Genetics; Metabolic; Neurodisability                                                       This article has been accepted for publication in Archives of Disease in Childhood following peer review. The definitive copyedited, typeset version is available online at: http://adc.bmj.com/content/99/8/754.  Steele R, Siden H, Cadell S, et al. Charting the territory: Symptoms and functional assessment in children with progressive, non-curable conditions. Arch Dis Child. 2014;99:754–62. doi: 10.1136/archdischild-2013-305246.  2  Abstract  Background: Children with progressive, non-curable genetic, metabolic, or neurological conditions require specialized care to enhance their quality of life. Prevention and relief of physical symptoms for these children needs to begin at diagnosis, yet, little is known about their patterns of symptoms and functional abilities.  Aim: To describe these children’s symptoms, as well as how the children’s condition affects them physically.  Design: Cross-sectional, baseline results from an observational, longitudinal study, Charting the Territory, that followed 275 children and their families.  Setting/participants: Seven tertiary care children’s hospitals in Canada, 2 in the USA. Families were eligible based on the child’s condition. A total of 275 children from 258 families participated.  Results: The 3 most common symptoms in these children were pain, sleep problems, and feeding difficulties; on average, they had 3.2 symptoms of concern. There was a pattern of underreporting of children’s symptoms for clinicians compared with parents. Regardless of use of associated medications, pain, feeding, and constipation symptoms were often frequent and distressing. Children with a G/J tube had a higher total number of symptoms, and respiratory problems, pain, feeding difficulties, and constipation were more likely to occur. They also tended to have frequent and distressing symptoms and to need extensive mobility modifications, which in turn was associated with higher numbers of symptoms.  Conclusions: These children experience multiple symptoms that have been previously documented individually, but not collectively. Effective interventions are needed to reduce their symptom burden. Future longitudinal analyses will examine which disease-modifying interventions improve, or not, symptom burden.   3  Children with progressive genetic, metabolic, or neurological conditions for which there is no cure (PNCs) and their families require specialized care to enhance their quality of life. In Canada, about half of the annual 2500 childhood non-traumatic deaths occur because of PNCs.[1,2] These diagnoses account for more than 50% of children who receive pediatric palliative care in North America.[1,3,4] Though individual conditions are rare, they have been broadly categorized within a single group because of their unifying problem: they involve impairment/injury of the central nervous system and are progressive.[5,6] These children typically display problems seen with such impairment, e.g., pain, seizures, and loss of mobility,[7] therefore prevention and relief of physical symptoms needs to begin at diagnosis. Yet, little is known about the patterns of symptoms or possible correlations with functional abilities.  Research in pain and symptom management is a priority in pediatric palliative care,[8] and families of children with PNCs report symptoms and issues around medications as two important milestones in a child’s illness trajectory.[6] Common symptoms reported by clinicians include: pain, breathing problems, feeding difficulties, alertness/interaction changes, sleep problems, seizures, and constipation.[3,7,9-22] There is a need to research children’s symptoms, but information about symptoms is not always documented even in patient records[4] and the co-occurrence of symptoms is rarely explored. Children may be prescribed multiple medications,[3,20-24] but little is published about the specific types of medications commonly used by clinicians and whether or not the prescribed medications alleviate symptoms. These children usually experience developmental delays and functional losses that increase over time.[6,9,25] There is some evidence that pain behaviour may be different for children with neurodegenerative conditions,[25] but the literature is inconsistent regarding 4 whether functional levels affect pain expression.[26,27] The relationships between functional levels and other symptoms remain unclear, yet loss of mobility and needing modifications/equipment are significant milestones in this population.[6] Many children with PNCs receive enteral feeds, e.g., via gastrostomy or jejunal (G/J) tubes.[6,24] Improved nutrition can lead to improved quality of life for the child and family,[28] but G/J feeding can result in complications such as aspiration pneumonia, site infection, and reflux.[22,28] Consequently, the children may have related symptoms, e.g., pain, feeding or respiratory problems, but little is known about correlations between G/J feeds and symptoms. Further, clinical experience suggests that children with G/J tubes often have restricted mobility, which may contribute to increased symptoms. Exploration of this anecdotal evidence is warranted.  Overall, there is a paucity of research on which to base best practices for care of children with PNCs.[3,29] Much of the fairly limited literature focuses on specific conditions.[9-15,30,31] Discussion is often about the treatment of one symptom such as seizures.[9,16] A majority of the research is retrospective[13,14,30] and often the sample sizes are small.[9,10,15] Though some information has been published, there is little to describe the natural progression of this group as a whole, particularly from a prospective approach. Little information exists about differences in symptoms by physical functioning, e.g., by the need for mobility modifications or presence/absence of a G/J tube. This paper reports baseline results about 275 child participants (from 258 families) in a multisite, longitudinal study, Charting the Territory, that followed children with these conditions, and their families. The focus is on symptoms reported, and differences in selected symptoms by use of medications, G/J tube presence, and/or level of mobility modifications. 5 METHODS The larger study was developed to determine the child’s clinical trajectory including symptoms, and the associated emotional, social, physical, and spiritual experiences of the family over time. It used quantitative methods, with established instruments and record reviews. Children (0-19 years) and their families were followed for 18-48 months, depending on when they entered the study. Data collection began at baseline and continued through bereavement or until the end of study.[1] Recruitment  Families were recruited from July 2009 till October 2012, mostly through referrals from hospital clinics (49%) and hospice/palliative teams/services (43%). Eligibility was based on the child’s condition (Appendix A, Web only). Of 385 families evaluated for eligibility, 93 did not meet criteria or declined further contact, 34 could not be contacted, leaving 258 families. Data Collection To facilitate continuity and reduce the likelihood of inter-rater discrepancies, parents identified a ‘designated’ parent in their family to answer questions about the child. Designated parents completed baseline questionnaires in-person, by mail, or a combination thereof and, subsequently, reported on their child’s symptoms monthly via the Internet or, for a few, by telephone. Parents who chose the online method needed to have an existing email account and access to the Internet. The software included an email component every month to prompt parents to visit the secure website and complete the symptom questionnaire. A Research Assistant (RA) called telephone responders to obtain answers. Record review 6 After enrollment, the site RA collected baseline information regarding the child’s diagnosis, clinical condition, medications, and procedures undergone. Disease diagnoses were characterized according to a scheme developed in an iterative process by one of the authors (HS) and 2 study collaborators from Biochemical Diseases/Genetics and Pediatric Neurology. Clinical symptoms Information about 7 common symptoms in this population was collected at baseline and then monthly. Parents were given definitions for each symptom - pain, respiratory/breathing problems, feeding difficulties, alertness/interaction changes, sleep problems, seizures, and constipation - prior to completing the baseline assessment (Appendix B, Web only).  The instrument takes 6 to 10 minutes to complete and was designed by the research team in consultation with other experienced clinicians, families linked to a pediatric palliative care hospice program, and a psychometrician. It is a modification of the PediQUEST symptom recording tool and the revised Memorial Symptom Assessment Scale (7-12 years of age) developed for symptoms in children with cancer.[32] Symptoms are tracked for timing of onset (symptom latency[15]), frequency in last week, change since first appeared (or since previous month), and extent of distress. Face and content validity were evaluated prior to the start of the study with the assistance of parents of children who received palliative care, and the instrument was pilot tested in clinical practice (HS). Annual functional assessment: Pediatric Evaluation of Disability Inventory (PEDI©) The PEDI©[33,34] was used to assess the children’s function at baseline, then annually. It yields information under Parts I (Functional Skills), II (Caregiver Assistance), and III (Modifications) in the domains of self-care, mobility, and social function. Scores are summarized into composite scores. Raw scores from Parts I and II can be converted to normative standard 7 scores as well as scaled scores for comparison against norm-age values. Raw scores are reported for this study because children with PNCs tend to have significant disabilities; therefore, it was deemed unreasonable to compare them to ‘norms’. Appendix C (Web only) provides details about the reliability and validity of the PEDI©, procedures for administering it, and steps taken to ensure RAs were competent in using the instrument. Data Analysis Data were entered into a database for preprocessing, data cleaning, and determination of scale composites according to the appropriate procedures for the standardized measures. Missing data for outcome measures were linearly imputed, if possible, according to standard procedures for the specific tool. Analyses were performed using the Statistical Package for the Social Sciences (SPSS®), Version 20, with statistical significance at p<.05.  Demographics and outcome measures were summarized using descriptive statistics: frequencies and % for categorical variables, means and SD for continuous variables. Two-tailed comparison tests included: χ², two-sample t-test, Spearman’s Rho and Pearson r correlations, 1-way and 2-way ANOVA. Ethics approval for this bi-national (Canada/USA) study was obtained from 9 study settings plus the 4 universities where the researchers were affiliated. The University of British Columbia and Children’s & Women’s Health Centre of BC (Certification #H08-00124) approved the central site in Vancouver. On behalf of themselves and their child(ren), parent participants signed a consent form; the ill children were unable to sign consent/assent forms due to their health conditions. RESULTS Sample Characteristics 8 Few meaningful differences were detected by study site across all analyses, therefore, results are reported for the whole sample. On average, the 258 families had 2.5 (SD 1.4) living children, range 1-10; 283/574 of the total number of children had a PNC, with two such children enrolled from 17 families. Prior to baseline, a total of 20 children in 16 families had died, 50% from a PNC; of the remainder, 5 were miscarriages. Most families (n=169, 65.5%) received some form of palliative care. Families had been engaged with the medical system for over 6 years on average (table 1). They typically waited almost a year between bringing the child to a physician and receiving a diagnosis; though many children still did not have a diagnosis (n=48, 17.5%). About 30% of parents learned prenatally (n=33) or perinatally (n=51) that the child had a problem. Others initiated investigation because of concerns such as the child not meeting developmental milestones (n=54, 19.6%). The child’s average age at which parents began the diagnostic process was 12.1 months (SD 25.5). Clinicians typically employed multiple methods for confirming diagnoses. Records indicated that all children underwent at least one surgery or interventional radiology procedure (table 2). Most had multiple assessments and many took several types of medications (mean 3.5, SD 2.3). Sixty-one percent (n=168) received nourishment other than orally and 13.1% (n=36) used a ventilator. Many children (n=125, 45.5%) had an abnormal EEG. As detailed in table 3, symptoms were common, with about half of the children experiencing any of the symptoms except for difficulties with alertness. Parents reported an average of 3.2 (SD 1.9) symptoms at baseline, whereas clinicians documented fewer (mean 2, SD 1.7). Correlations between parental and clinician reports of symptoms were positive, and weak to moderately strong; all were statistically significant (p=<.001). 9  Parents reported frequency and change for symptoms, as well as the distress they ascribed to the child (table 4). Only two symptoms showed statistically significant differences by conditions: change in seizures (χ2=35.79, p=.02), and distress from feeding difficulties (χ2=38.26, p=.03). The only statistically significant difference between 4 selected symptoms and their associated medication use (table 5) was frequency for seizures (χ2=11.71, p=<.01), though distress was present for the majority who were receiving seizure medications. Regardless of whether medications were used, parents also reported children experiencing and being distressed by pain, feeding difficulties, and constipation. Children with G/J tubes had more symptoms (mean 3.67, SD 1.74) than children without (mean 2.68, SD 2; p=<.001). Differences were seen in breathing, pain, feeding, and constipation symptoms depending on G/J tube presence (table 6). Despite mostly statistically insignificant results, G/J tube presence was generally consistent with higher levels of symptom frequency and distress (table 7). Table 8 shows that average functional levels were all on the low end of the respective subscales, indicating that children could provide little self-care (mean 14.7, SD 19.6), had difficulty in getting around (mean 14.6, SD 19.2), and were limited in social function (mean 16.9, SD 17.8). All Functional and Caregiver Assistance subscales showed statistically significant differences by conditions (p=<.001). As shown in table 9, children who scored lower on functional skills had a higher total number of symptoms; children with G/J tubes were more likely to require extensive mobility modifications; and both G/J tube presence and level of mobility modification were associated with higher numbers of symptoms, though their interaction was not statistically significant. 10 DISCUSSION Results from this study are supported by the literature in a number of ways, including the plethora of medications given to these children.[3,16,17] One of the most striking findings is the children’s large symptom experience. The children had, on average, just over 3 symptoms of concern and almost every symptom appeared in at least half of the children. This finding in children with metabolic, chromosomal, or neurological conditions parallels findings about symptom burden in children with cancer.[35,36]  Symptom management is a hallmark of pediatric palliative care and it is not surprising that many of the children were being treated for multiple symptoms. However, symptoms were often not well-controlled and children frequently experienced and were distressed by symptoms, whether or not they were receiving associated medications. Where children were receiving an indicated medication, the treatment was often not effective. These findings highlight the difficulties in managing symptoms in this population, especially seizures, and suggest that further research is needed to identify optimal management.  Though there were significant correlations between parent and clinician reports of children’s symptoms, there was a pattern of underreporting for clinicians compared with parents, a finding that is consistent with some pediatric palliative care literature.[7,21,37,38] There are many potential reasons for this finding, including missing documentation.[4] One reason may be that neurological symptoms especially are difficult to deal with in this population.[23] Another is that clinicians sometimes report only those symptoms that they feel confident in treating[7] or they do not assess for a symptom because of the child’s impaired cognition,[21] and, finally, sometimes clinicians disbelieve parents, especially if a child is non-verbal.[38] Clinicians whose approach is to question why they should not believe a parent rather than why they should and 11 who view symptoms as multidimensional experiences that have meaning for patients and their families rather than simply as side effects may be less likely to underreport.[7,38] Also, many anxieties/difficulties accompany parents dealing with PNCs.[6]. If a symptom causes parental concern then parents may report it more frequently.[39] Therefore, clinicians should ask parents why a symptom is of concern so they can address the parents’ needs. From a clinical perspective, it is equally important to be cognizant of both the frequency and the severity of symptoms as each may lead to a different understanding of the nature of a symptom, and, therefore, may contribute to identifying the most appropriate intervention.  The increased pattern of symptom burden when a G/J tube is present and/or when there are extensive mobility modifications indicates that clinicians need to be alert to assessing and managing symptoms in these children. We do not know whether feeding devices or mobility tools increase symptoms directly, but both interventions are indicative of children with more severe conditions and, consequently, more symptom burden. Therefore, extra attention to symptoms in children with feeding devices and/or mobility modifications is warranted. Limitations A main limitation of this report is its cross-sectional, primarily descriptive nature. But these are only the first results from a longitudinal study. Future analyses of data, for example examining correlations among variables and over time, will provide a better understanding of changing outcomes as the child’s illness trajectory unfolds. Another limitation is that parents reported on behalf of their child. However, this population of children with PNCs is typically non-communicative and parents usually speak for their child in everyday situations. Therefore, it may be reasonable to expect parents rather than these children to provide information. 12 A limitation of the PEDI© in a population with a high level of neurological impairment is that it does not indicate other functional limitations that can impact health, such as respiratory decline and earlier death correlating with a child’s inability to hold his head up when prone. However, an important point to note is that a functional assessment can highlight a child’s deterioration over time or it can point out when an intervention may be warranted. Routine evaluation of functioning over time would not only document a condition’s trajectory, but would also identify places for clinical intervention, as well as provide comparative data about an individual child over time. CONCLUSIONS  Pediatric palliative care is an expanding field, yet the paucity of research means that clinicians have little evidence on which to base their practice. This baseline report from a unique longitudinal study offers detailed information about symptoms in children with progressive, non-curable genetic, metabolic, or neurological conditions. The large sample size lends credence to findings and provides a solid foundation for understanding trajectories over time. Future longitudinal analyses will examine which disease-modifying interventions improve, or not, symptom burden.  Acknowledgments: The primary study was supported by the Canadian Institutes of Health Research Grant MOP-89984.  The authors would like to thank all the children and families who participated in the study. We also thank the research assistants in each study setting for their contribution to data collection.  We appreciate that the terminology is not settled regarding the most appropriate term to use when reporting on children with the types of conditions in this study. Various terms, such as life-threatening, life-limiting, life-shortening, and complex medical problems, have all been proposed and often one is preferred over another depending on one’s country. For the purposes of this paper, we have used the term ‘progressive, non-curable conditions’ because it reduces ambiguity and is acceptable to this journal.  13 Financial disclosure: The authors have no financial relationships relevant to this article to disclose.  Conflicts of interest: The authors have no conflicts of interest to disclose.  Funding source: External funding was secured from the Canadian Institutes of Health Research, Canada; MOP 89984.  Writing Assistance: The authors attest that no writing assistance was provided for this article. 14  What is known about this topic:   Progressive, non-curable genetic, metabolic, or neurological conditions involve impairment/injury of the central nervous system.  Children often have multiple symptoms associated with such impairment, e.g., pain, seizures, and loss of mobility, and need appropriate interventions.  These children often rely on enteral nutrition, e.g., via G/J tube, and require mobility modifications.  What this study adds:   The 3 most common symptoms in these children are pain, sleep problems, and feeding difficulties; on average, they have 3 symptoms of concern.  Regardless of medication use, seizure, pain, feeding, and constipation issues may be frequent and distressing to these children.  Children with G/J tubes, lower levels of function, or extensive mobility modification requirements have a higher total number of symptoms.  15  REFERENCES 1. 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The natural history of juvenile or subacute GM2 gangliosidosis: 21 new cases and literature review of 134 previously reported. Pediatrics 2006;118:e1550-62. 16. Wusthoff CJ, Shellhaas RA, Licht DJ. Management of common neurologic symptoms in pediatric palliative care: seizures, agitation, and spasticity. Pediatr Clin North Am 2007;54:709-733. 17 17. Ho C, Straatman L. A review of pediatric palliative care service utilization in children with a progressive neuromuscular disease who died on a palliative care program. J Child Neurol 2013;28:40-44. 18. Malcolm C, Forbat L, Anderson G, et al. Challenging symptom profiles of life-limiting conditions in children: a survey of care professionals and families. Palliat Med 2011;25:357-364. 19. Malcolm C, Hain R, Gibson F, et al. Challenging symptoms in children with rare life-limiting conditions: findings from a prospective diary and interview study with families. Acta Pediatr 2012;101:985-992. 20. Herbert A, Seton C, Gamble A. 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Mitochondrial disease: needs and problems of children, their parents and family. a systematic review and pilot study into the need for information of parents during the diagnostic phase. J Inherit Metab Dis 2007;30:333-340. 31. Wasserstein MP, Desnick RJ, Schuchman EH, et al. The natural history of Type B Niemann-Pick disease: results from a 10-year longitudinal study. Pediatrics 2004;114:E672-e677. 32. Dussel V, Aldridge SE, Kang T, et al. Feasibility of Conducting a Randomized Controlled Trial (RCT) in Children With Advanced Cancer: Lessons From the Pediatric Quality of Life and Evaluation of Symptoms Technology (PediQuest) Study. Salt Lake City, UT: American Academy of Hospice and Palliative Medicine 2007. 33. Haley M, Coster W, Ludlow L, et al. Pediatric Evaluation of Disability Inventory (PEDI). Boston, MA: Trustees of Boston University 1998. 19 34. Berg M, Jahnsen R, Frǿslie KF, et al. Reliability of the Pediatric Evaluation of Disability Inventory (PEDI). Phys Occup Health Pediatr 2004;24:61-77. 35. Heath JA, Clarke NE, Donath SM, et al. Symptoms and suffering at the end of life in children with cancer: an Australian perspective. Med J Aust 2010;192:71-75. 36. Theunissen JMJ, Hoogerbrugge PM, van Achterberg T, et al. Symptoms in the palliative phase of children with cancer. Pediatr Blood Cancer 2007;49:160-165. 37. Hunt A. Pain assessment. In: Goldman A, Hain R, Liben S, eds. Oxford Textbook of Palliative Care for Children. 2nd ed. Oxford, UK: Oxford University Press; 2012:204-217. 38. Hauer JM, Faulkner, KW. Neurological and neuromuscular conditions and symptoms. In: Goldman A, Hain R, Liben S, eds. Oxford Textbook of Palliative Care for Children. 2nd ed. Oxford, UK: Oxford University Press; 2012:295-308. 39. Pritchard M, Burghen EA, Gattuso JS, et al. factors that distinguish symptoms of most concern to parents from other symptoms of dying children. J Pain & Symptom Manage 2010;39:627-636. 20  Licence for Publication Statement The Corresponding Author has the right to grant on behalf of all authors and does grant on behalf of all authors, an exclusive licence (or non-exclusive for government employees) on a worldwide basis to the BMJ Group and co-owners or contracting owning societies (where published by the BMJ Group on their behalf), and its Licensees to permit this article (if accepted) to be published in Archives of Disease in Childhood and any other BMJ Group products and to exploit all subsidiary rights, as set out in our licence. Contributor Statement  Gail Andrews: Ms. Andrews conceptualized and designed the study, analysed and interpreted the data, revised the resubmitted manuscript, and approved the resubmitted manuscript.  Susan Cadell: Dr. Cadell conceptualized and designed the study, revised the resubmitted manuscript, and approved the resubmitted manuscript.  Betty Davies: Dr. Davies conceptualized and designed the study, revised the resubmitted manuscript, and approved the resubmitted manuscript.  Leanne Feichtinger: Ms. Feichtinger analysed and interpreted the data, revised the resubmitted manuscript, and approved the resubmitted manuscript.  Hal Siden: Dr. Siden conceptualized and designed the study, analysed and interpreted the data, revised the resubmitted manuscript, and approved the resubmitted manuscript.  Mina Singh: Dr. Singh analysed and interpreted the data, revised the resubmitted manuscript, and approved the resubmitted manuscript.  Rose Steele: Dr. Steele conceptualized and designed the study, analysed and interpreted the data, drafted and revised the resubmitted manuscript, and approved the resubmitted manuscript. 21  Table 1: Selected Demographic Information at Baseline About 275 Children With Progressive, Non-Curable Conditions (PNCs)  Demographic Ill Children Gender    Female    Male  139 (50.5) 136 (49.5) Age (years) on entry to study     Age (months) at initiation of diagnostic evaluation (n = 261)     Age (years) at diagnosis (n = 203) 6.6 + 5.2a; 0 to 19 12.1+ 25.5; -5.8 to 183.5a,b 2.2+ 3; -0.4 to 15.7a,b New vs. Pre-existing condition (n = 258)    New (<6 weeks)    Pre-existing (>6 weeks)  3 (1.2) 255 (98.8) Top concerns that brought parent to MDc    Not meeting developmental milestones    Perinatal: diagnosis or problem noted    Seizures    Change in muscle tone    Prenatal: diagnosis or problem noted    Feeding difficulties    Breathing difficulties    Losing developmental milestones  54 (19.6) 51 (18.5) 47 (17.1) 34 (12.4) 33 (12) 30 (10.9) 21 (7.6) 21 (7.6) Weeks since parent first sought medical treatment (n = 261) 315.2+ 248.9; 7.9 to 980a Weeks since child’s diagnosis (n = 203) 269.6 + 231.3; 0 to 903.7a Months from initial diagnostic evaluation to confirmation of diagnosis (n = 197) 11.9+ 23.6; 0 to 177.9a Diagnostic methodsd    Molecular    Clinical    Biochemistry    Anatomic pathology  192 (69.8) 178 (64.7) 142 (51.6) 61 (22.2) Primary condition    Multi-organ congenital abnormalities    Severe neurological impairment - not yet diagnosed    Epileptic encephalopathy/neurodegenerative disease    Lysosomal/peroxisomal leukodystrophy    Mitochondrial encephalo-/myopathy    Structural CNS abnormalities    Small molecules diseases    Neuromuscular diseases    Other inborn errors of metabolism    Congenital disorders of glycosylation     Other conditions not otherwise specified (NOS)  57 (20.7) 48 (17.5) 44 (16) 43 (15.6) 29 (10.5) 18 (6.5) 13 (4.7) 10 (3.6) 6 (2.2) 4 (1.5) 3 (1.1) 22  Values are number (%) unless otherwise indicated.  a Plus-minus values are means + SD; followed by minimum to maximum. b Negative values reflect prenatal evaluations and diagnoses. c Some responses not reported in table; overall, numbers totaled more than sample size because some parents provided multiple responses. d Numbers totaled more than sample size because multiple methods could be documented.  23  Table 2: Record Review: Assessments, Interventions, and Medications Prior to Baseline for 275 Children With Progressive, Non-Curable Conditions  Assessments, Interventions, and Medications Ill Children Assessments    PT/OT (n = 274)    Dietician (n = 248)    Home nurse (n = 270)  249 (90.5) 246 (89.5) 198 (72.8) Selected interventions    Gastric or jejunal tube    Oxygen by nasal cannula or mask    Limits of care/Do not attempt resuscitation orders    Routine suctioning    Non-invasive ventilation    Clinical trial    Total parenteral nutrition    Nasogastric tube    Invasive ventilator    Complementary/alternative modalities    Complementary/alternative medications  148 (53.8) 29 (10.6) 42 (15.3) 50 (18.4) 23 (8.4) 20 (7.3) 4 (1.5) 16 (5.8) 13 (4.7) 7 (2.5) 6 (2.2) Surgery or interventional radiology procedures    Total number prior to baseline 275 (100) 11.4+ 5.5; 6-41a Used with surgery or interventional radiology procedures    General anesthesia       Total number of times anesthesia used    Sedation       Total number of times sedation used  170 (61.8) 3.23+ 4.8; 0-36a 61 (22.2) 0.6+ 1.6; 0-11a Electroencephalography (EEG) prior to baseline    Total number of EEGs 149 (54.2) 2.6 + 4.1; 0-30a Abnormal EEG prior to baseline 125 (45.5) Medications    Total number of different drug categories    Anticonvulsants    Antacids    Metabolic    Anxiolytic    Other    Laxatives    Acetaminophen/NSAIDs    Melatonin/hypnotic     Pro-kinetics    Cardiac    Antispasticity    Neuroleptic    Opiates    Antiemetics    Anesthetics  3.5 + 2.3; 0-12a 152 (55.5) 125 (45.6) 118 (43.1) 111 (40.7) 108 (39.4) 97 (35.4) 52 (19) 48 (17.5) 43 (15.8) 27 (9.9) 27 (9.9) 21 (7.7) 21 (7.7) 12 (4.4) 3 (1.1)  24 Values are number (%) unless otherwise indicated.  a Plus-minus values are means + SD; followed by minimum to maximum.  25  Table 3: Symptoms Present at Baseline for 275 Children With Progressive, Non-Curable Conditions  Symptom Reported by Parentsa  pb: conditions Reported by Clinicians in Children’s Recordsc pd: conditions Months Since Birth for Symptom to Appeare pf: conditions Pain 149 (55.2) rs = .34, <.001 .78   67 (26)  .13 45.7+59.3g  <.001 Sleep problems 136 (50.2) rs = .33, <.001 .28  75 (29.1)  .<01 31.4 +50.4g   .02 Feeding difficulties 130 (48) rs = .38, <.001 .24  108 (41.7)   .42 29.9 +49.3g <.001 Constipation 127 (47) rs = .28, <.001 .33  72 (27.9)   .30 30.7 +43.1g  <.001 Respiratory or breathing problems 126 (46.5) rs = .28, <.001 .37  66 (25.6)  .57 46.7 +60.3g  <.001 Seizures 109 (40.2) rs = .65, <.001 <.001  110 (42.5)   <.01 34 +49g  .001 Alertness and interaction changes 94 (34.7) rs = .24, <.001 .03  42 (16.3)  .61 55.4 + 61.5g  <.001 Total number of symptoms 3.2+1.9g rs = .46, <.001 <.01  2 +1.7g   .02    Values are number (%) unless otherwise indicated.  a % for parents calculated based on number of parents who responded to the question; rs is the Spearman’s Rho result for bivariate correlations between parental and clinician reports, followed by p-value. bχ2 test was used for categorical variables and 1-way ANOVA was used for the continuous variable to test by children’s conditions. c% for clinicians calculated based on number of records reviewed by RA. dχ2 test was used for categorical variables and 1-way ANOVA was used for the continuous variable to test by children’s conditions. e Results predicated on symptom being present. f 1-way ANOVA was used to test continuous variables by children’s conditions. g Plus-minus values are means + SD.  26  Table 4: Parental Report of Frequency, Change, & Distress for Symptoms Present at Baseline in 275 Children With Progressive, Non-Curable Conditions  Symptoma Frequencyb pc: conditions Changed pc: conditions Distresse pc: conditions Pain (n = 149) 4 (2.7); 70 (47.3); 60 (40.5); 14 (9.5) .31 41 (27.5); 33 (22.1); 52 (34.9); 23 (15.4) .31 5 (3.4); 64 (43.2); 69 (46.6); 10 (6.8) .05   Sleep problems (n = 136) 6 (4.5); 39 (29.1); 88 (65.7); 1 (0.7) .59 30 (22.4); 63 (47); 36 (26.9); 5 (3.7) .52 34 (25.4); 38 (28.4); 47 (35.1); 15 (11.2) .14 Feeding difficulties (n = 130) 11 (8.5); 35 (27.1); 81 (62.8); 2 (1.6) .48 41 (31.5); 43 (33.1); 43 (33.1); 3 (2.3) .25 26 (20.2); 39 (30.2); 51 (39.5); 13 (10.1) .03 Constipation (n = 127) 7 (5.5); 48 (37.8); 72 (56.7); 0 .93 34 (27); 63 (50); 29 (23); 0 .37 11 (8.8); 36 (28.8); 66 (52.8); 12 (9.6) .44 Respiratory or breathing Problems (n = 126) 5 (4); 59 (47.2); 60 (48); 1 (0.8) .83 40 (32.8); 37 (30.3); 43 (35.2); 2 (1.6) .81 16 (13); 41 (33.3); 56 (45.5); 10 (8.1) .20 Seizures (n = 109) 9 (8.3); 45 (41.3); 49 (45); 6 (5.5) .53 40 (36.7); 27 (24.8); 38 (34.9); 4 (3.7) .02 22 (20.4); 28 (25.9); 42 (38.9); 16 (14.8) .39 Alertness and interaction changes (n = 94) 4 (4.3); 57 (62); 27 (29.3); 4 (4.3) .66 41 (44.1); 22 (23.7); 26 (28); 4 (4.3) .08 28 (30.1); 22 (23.7); 25 (26.9); 18 (19.4) .17  Numbers reported as frequency (%).  a Sample size for each symptom is the number of times parents reported a symptom was present for their child at baseline. b Frequency of symptom in last week reported as Almost never; Sometimes; Most of the time; Unsure/Can’t tell. % calculated based on number of parents who responded to the question. c χ2 tests used for all variables by children’s conditions; for all comparisons. d Change in symptom since symptom first appeared reported as Better; About the same; Worse; Unsure/Can’t tell. % calculated based on number of parents who responded to the question. e Extent of child’s distress from symptom in last week reported as None or a little bit; Somewhat; A lot; Unsure/Can’t tell. % calculated based on number of parents who responded to the question. 27  Table 5: Relationships Between Use of Associated Medications and Frequency & Distress for 4 Selected Symptoms Present at Baseline in 275 Children With Progressive, Non-Curable Conditions  Symptoma Frequency Medications                No      Yes Pb  Distress Medications                 No     Yes Pb  Seizuresc (n = 109) Almost never:               3         6 Sometimes:                   8       37 Most of the time:          1       48 Unsure/Can’t tell:         2         4  <.01 None or a little bit:        5       17 Somewhat:                    4       24 A lot:                             3       39 Unsure/Can’t tell:          2       14  .37 Paind (n = 147) Almost never:               3         1 Sometimes:                 50       19 Most of the time:        38       22 Unsure/Can’t tell:       13         1  .17 None or a little bit:        3        2 Somewhat:                   52       12 A lot:                            42       26 Unsure/Can’t tell:          7         3  .1 Feeding difficultiese (n = 129) Almost never:               3         8 Sometimes:                 10       25 Most of the time:        35       46 Unsure/Can’t tell:         0          2  .26 None or a little bit:        9       17 Somewhat:                   18       21 A lot:                            14       37 Unsure/Can’t tell:          6         7  .27 Constipationf (n = 126) Almost never:              4         3 Sometimes:                 29       18 Most of the time:        34       38 Unsure/Can’t tell:       0        0  .3 None or a little bit:        8         3 Somewhat:                   23       13 A lot:                            29       36 Unsure/Can’t tell:          6         6  .15  Numbers reported as frequency.  a Sample size for each symptom is the number of times parents reported on frequency or distress of a symptom for their child at baseline. b χ2 tests used for all comparisons. c Medications considered to be associated with management of seizures: anticonvulsants. d Medications considered to be associated with management of pain: NSAIDS, Acetaminophen, anesthetics; counted as ‘yes’ if any of these medications used. e Medications considered to be associated with management of feeding difficulties: antacids, prokinetics, antiemetics; counted as ‘yes’ if any of these medications used. f Medications considered to be associated with management of constipation: laxatives.  28  Table 6: Relationships Between Presence of G/J Tube and Presence of Symptoms at Baseline for 275 Children With Progressive, Non-Curable Conditions  Symptoma G/J Tube Present          No        Yes Pb  Respiratory or breathing problems (n = 270)                                                                         No                                                                        Yes           84        61          41        85  <.001 Pain (n = 270)                                                                         No                                                                        Yes           67        54          58        91  <.01 Feeding difficulties (n = 271)                                                                         No                                                                        Yes           76        65          49        81  <.01 Constipation (n = 270)                                                                         No                                                                        Yes           76        67          48        79  <.05 Seizures (n = 271)                                                                         No                                                                        Yes           82        80          43        66  .07 Sleep problems (n = 271)                                                                        No                                                                        Yes           69        66          56        80  .1 Alertness and interaction changes (n = 271)                                                                         No                                                                        Yes           85        92          40        54  .39 Total # symptoms (n = 271)         125     146 <.001  Numbers reported as frequency.  a Sample size for each symptom is the number of times parents reported on presence of a symptom for their child at baseline. b χ2 tests were used for the categorical variables; two-sample t-test was used for the continuous variable.  29  Table 7: Relationships Between Presence of G/J Tube and Frequency & Distress for Symptoms Present at Baseline in 275 Children With Progressive, Non-Curable Conditions  Symptoma Frequency G/J tube Present         No      Yes Pb  Distress G/J tube Present          No      Yes Pb  Respiratory or breathing problems (n = 125) Almost never:               2         3 Sometimes:                 20       39 Most of the time:        18       42 Unsure/Can’t tell:         0         1  .84 None or a little bit:        7          9 Somewhat:                   16        25 A lot:                            10        46 Unsure/Can’t tell:          6          4  <.05 Pain (n = 148) Almost never:               1         3 Sometimes:                 31       39 Most of the time:        19       41 Unsure/Can’t tell:         7         7  .36 None or a little bit:        4          1 Somewhat:                   27        37 A lot:                            20        49 Unsure/Can’t tell:          6          4  <.05 Feeding difficulties (n = 129) Almost never:               3         8 Sometimes:                 13       22 Most of the time:        32       49 Unsure/Can’t tell:         1         1  .86 None or a little bit:       13        13 Somewhat:                   19         20 A lot:                            12        39 Unsure/Can’t tell:          5           8  <.05 Constipation (n = 127) Almost never:               3         4 Sometimes:                 17       31 Most of the time:        28       44 Unsure/Can’t tell:       0        0  .89 None or a little bit:         6          5 Somewhat:                    17        19 A lot:                             23        43 Unsure/Can’t tell:           2        10  .16 Alertness and interaction changes (n = 93) Almost never:              3          1 Sometimes:                27        30 Most of the time:         8        19 Unsure/Can’t tell:        2          2  .25 None or a little bit:        12        16 Somewhat:                     13         9 A lot:                             10        15 Unsure/Can’t tell:           5         13  .25 Sleep (n = 134) Almost never:              3          3 Sometimes:                19        20 Most of the time:        33       55 Unsure/Can’t tell:        0          1  .51 None or a little bit:        13        21 Somewhat:                      3         25 A lot:                             20        27 Unsure/Can’t tell:           9          6  .37 Seizures (n = 109) Almost never:              4          5 Sometimes:                20        25 Most of the time:       16        33 Unsure/Can’t tell:        3          3  .62 None or a little bit:         7         15 Somewhat:                    13        15 A lot:                             18        24 Unsure/Can’t tell:           4         12  .44  Numbers reported as frequency.  a Sample size for each symptom is the number of times parents reported on frequency or distress of a symptom for their child at baseline. b χ2 tests used for all comparisons.  30  Table 8: Outcome Measure Results for 275 Children With Progressive, Non-Curable Conditions: Pediatric Evaluation of Disability Inventory  PEDI©: Parts I, II, & III and domains  Ill Childrena pb: conditions pc: gender Functional skill: Self-care (n = 270) 14.7 + 19.6 <.001 .23 Functional skill: Mobility (n = 270) 14.6 +19.2 <.001 .65 Functional skill: Social function (n = 270) 16.9 +18 <.001 .44 Caregiver assistance: Self-care (n = 270) 4.7 +9.7 <.001 .11 Caregiver assistance: Mobility (n = 269) 6.5 +11.1 <.001 .63 Caregiver assistance: Social function (n = 270) 4.1 +6.8 <.001 .62 Modifications: Self-care (n = 270)    No modifications    Child-oriented (non-specialized)    modifications    Rehabilitation equipment    Extensive modifications  3.9 +2.2 3.0 +2  0.4 +0.8 0.7 +1.1  <.01 <.01  .22 .05  .55 .49  .05 .14 Modifications: Mobility (n = 270)    No modifications    Child-oriented (non-specialized)    modifications    Rehabilitation equipment    Extensive modifications  4 +2.3 1 +1.6  0.6 +1.2 1.4 +2.2  .001 .01  .30 .001  .26 .51  .68 .67 Modifications: Social function (n = 270)    No modifications    Child-oriented (non-specialized])    modifications    Rehabilitation equipment    Extensive modifications  4.5 +1.2 0.1 +0.4  0.2 +0.8 0.2 +0.7  <.01 .33  .04 .42  .65 .62  .25 .89  a Plus-minus values are means + SD. b 1-way ANOVA was used for all continuous variables to test by children’s conditions. c Two-sample t-test was used for all continuous variables to test by gender.  31  Table 9: Relationships Between Functional Skills, Presence of G/J Tube, Highest Level of Mobility Modification Required, and Total Number of Symptoms Present at Baseline in 275 Children With Progressive, Non-Curable Conditions  PEDI©-G/J Tube Variables Test Statistics P  Functional skills – Self-care (Raw score)a (n = 271) Total # symptoms; r = -.287 <.001 Functional skills – Mobility (Raw score)a (n = 271) Total # symptoms; r = -.297 <.001 Functional skills – Social function (Raw score)a (n = 271) Total # symptoms; r = -.21 .001    PEDI©: Highest level of mobility modificationsb,c (n = 270) G/J tube present          No       Yes None:                           30        31 Child:                           39        28 Rehab:                          27        13 Extensive:                    27        75 χ2 = 27.39; df = 3  <.001    Interaction of Highest level of mobility modifications & G/J tube present or notd Total # symptoms; F = 1.686; df = 3,258 .17 PEDI©: Highest level of mobility modificationsd Total # symptoms; F = 13.654; df = 1,258 <.001 G/J tube present or notd Total # symptoms; F = 4.866; df = 3,258 <.01  Numbers reported as frequency.  a Pearson correlation between functional skills and total number of symptoms at baseline. b Highest level determined by presence of a positive response on at least one of the PEDI modification questions, where None is lowest level possible and Extensive is highest level possible. c χ2 test used for comparison between highest level of mobility modifications and presence of G/J tube at baseline. d 2-way ANOVA with highest level of mobility modifications and presence of G/J tube as the 2 factors, and total number of symptoms as the response variable.  

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