There is no standardized approach to the treatment of pediatric antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (ped-AAV). Because of the rarity of ped-AAV, randomized trials have not been feasible. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed consensus treatment plans (CTPs) for severe ped-AAV to enable the future study of comparative effectiveness and safety.
A workgroup of CARRA members (rheumatologists and nephrologists) formed the AAV working group. This group performed a literature review on existing evidence-based treatments and guidelines for the management of AAV. They determined that the target population for CTP development was patients <18 years with new-onset granulomatosis with polyangiitis (GPA), microscopic polyangiitis, or renal-limited AAV (eosinophilic-GPA was excluded) with presentation confined to those with severe disease i.e. organ- or life-threatening. Face-to-face consensus conferences employed nominal group techniques to identify treatment strategies for remission-induction and remission-maintenance, data elements to be systematically collected, and outcomes to be measured over time.
The ped-AAV workgroup developed two CTPs for each of the remission-induction and remission-maintenance of severe AAV. A corticosteroid-weaning regimen for induction and maintenance, a core dataset, and outcome measures were also defined. A random sample of CARRA membership voted acceptance of the CTPs for remission-induction and remission-maintenance with a 94% (75/80) and 98% (78/80) approval rate respectively.
Consensus methodology established standardized CTPs for treating severe ped-AAV. These CTPs were in principle accepted by CARRA-wide membership for pragmatic comparative effectiveness evaluation in a long-term registry.