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Abstract

Objective: To characterize early disease course in childhood onset antineutrophil cytoplasmic antibody (ANCA) associated vasculitis (AAV) and 12-month outcomes. Methods: Eligible subjects were children diagnosed with GPA, MPA, EGPA, and ANCA-positive pauci-immune glomerulonephritis before their eighteenth birthday and entered into The Pediatric Vasculitis Initiative (PedVas) study. The primary outcome was remission (Pediatric Vasculitis Activity Score (PVAS) = 0 with corticosteroid dose (CS) <0.2mg/kg/day) at 12-months. Secondary outcomes included rates of inactive disease (PVAS 0, any CS dose) and improvement at post-induction (4-6 months after diagnosis) and at 12-months, damage at 12-months, and relapse rates. Results: 105 patients were included. Median age at diagnosis was 13.8 years (IQR 10.9 – 15.8 years); 42% achieved remission at 12-months, 49% had inactive disease at post-induction (4-6 months), and 61% had inactive disease at 12-months. The majority of patients improved even if they did not achieve inactive disease. An improvement in PVAS score of 50% from time-of-diagnosis to post-induction was seen in 92% of patients. Minor relapses occurred in 12 of 51 patients (24%) after achieving inactive disease at post-induction. The median damage score (measured by a modified pediatric vasculitis damage index (pVDI)) at 12-months was 1 (range 0-6). 63% of patients had ≥ 1 damage item scored at 12-months. Conclusion: This is the largest study to date reporting outcomes in pediatric AAV. Although a significant proportion of patients do not achieve remission, the majority of patients respond to treatment. Unfortunately, more than half of patients have damage early in their disease course.