Abstract
BackgroundBecause pediatric antineutrophil cytoplasmic antibody-associated vasculitis is rare, management generally relies on adult data. We assessed treatment practices, uptake of existing clinical assessment tools, and interest in pediatric treatment protocols among rheumatologists caring for children with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).MethodsA needs-assessment survey developed by an international working group of pediatric rheumatologists and two nephrologists was circulated internationally. Data were summarized with descriptive statistics. Pearson’s chi-square tests were used in inferential univariate analyses.ResultsThe 209 respondents from 36 countries had collectively seen ~1600 children with GPA/MPA; 144 had seen more than two in the preceding 5 years. Standardized and validated clinical assessment tools to score disease severity, activity, and damage were used by 59, 63, and 36%, respectively; barriers to use included lack of knowledge and limited perceived utility. Therapy varied significantly: use of rituximab rather than cyclophosphamide was more common among respondents from the USA (OR = 2.7 [1.3-5.5], p = 0.0190, n = 139), those with >5 years of independent practice experience (OR = 3.8 [1.3-12.5], p = 0.0279, n = 137), and those who had seen >10 children with GPA/MPA in their careers (OR = 4.39 [2.1-9.1], p = 0.0011, n = 133). Respondents who had treated >10 patients were also more likely to continue maintenance therapy for at least 24 months (OR = 3.0 [1.4-6.4], p = 0.0161, n = 127). Ninety six percent of respondents believed in a need for pediatric-specific treatment guidelines; 46% supported adaptation of adult guidelines while 69% favoured guidelines providing a limited range of treatment options to allow comparison of effectiveness through a registry.ConclusionsThese data provide a rationale for developing pediatric-specific consensus treatment guidelines for GPA/MPA. While pediatric rheumatologist uptake of existing clinical tools has been limited, guideline uptake may be enhanced if outcomes of consensus-derived treatment options are evaluated within the framework of an international registry.
Highlights
Because pediatric antineutrophil cytoplasmic antibody-associated vasculitis is rare, management generally relies on adult data
This balance has been incrementally fine-tuned through clinical trials that rely on accurate disease sub-classification and scoring tools to stage disease severity, activity, and damage (Table 1)
A survey draft developed by the Pediatric Rheumatology group and a nephrologist at British Columbia Children’s Hospital was finalized with input from the Vasculitis Working Groups of Childhood Arthritis and Rheumatology Research Alliance (CARRA) and Pediatric Rheumatology European Society (PRES)
Summary
Because pediatric antineutrophil cytoplasmic antibody-associated vasculitis is rare, management generally relies on adult data. Uptake of existing clinical assessment tools, and interest in pediatric treatment protocols among rheumatologists caring for children with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA). Antineutrophil cytoplasmic antibody- (ANCA-) associated vasculitis (AAV) describes the subset of vasculitides primarily involving small vessels: granulomatosis with polyangiitis (GPA, formerly Wegener’s granulomatosis), microscopic polyangiitis (MPA), eosinophilic granulomatosis with polyangiitis (EGPA, formerly Churg-Strauss syndrome), and renal-limited pauci-immune glomerulonephritis [1]. Balancing the risks associated with existing therapies against the damage associated with under- or overtreatment remains a challenge In adult populations, this balance has been incrementally fine-tuned through clinical trials that rely on accurate disease sub-classification and scoring tools to stage disease severity, activity, and damage (Table 1). The rarity of pediatric AAV has limited opportunities for pediatric clinical trials such that management decisions are largely informed by adult data [2], with potential for significant practice variation
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