Session Information
Session Type: Abstract Submissions (ACR)
Background/Purpose: Childhood AAV is rare and outcome studies are limited. The PedVas Study is an international initiative collecting clinical data (to A Registry of Childhood Vasculitis -ARChiVe) and biological samples from children with primary systemic vasculitis. This is the largest study to date reporting early outcomes in pediatric AAV.
Methods: Patients diagnosed with granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA), eosinophilic granulomatosis with polyangiitis (EGPA) and ANCA-associated pauci-immune glomerulonephritis before 18 years of age and with 12-month follow up data were included. Diagnoses were verified by applying a pediatric modification of the European Medicines Agency algorithm for classifying AAV (incorporating EULAR/PRINTO/PRES classification criteria). Descriptive statistics were used for baseline characteristics, remission-induction medications, and rates of remission at 4-6 months (post-induction) and at 12-months. Remission (on or off medications) was defined by a Pediatric Vasculitis Activity Score (PVAS) of 0. Relapse was defined as recurrence or new onset of disease activity (an increase in PVAS ≥1, from 0) attributable to active inflammation. Major relapse was defined as the recurrence, or new onset of potentially organ-or life-threatening disease activity requiring increased treatment, additional to corticosteroids (CS). Other relapses were considered minor.
Results: Among 40 patients eligible for inclusion in June 2014, 39 had verified AAV (35 GPA, 3 MPA, 1 EGPA), one patient became “unclassified” following verification. Median age at diagnosis was 12.4 years. 64% of patients were female. Median PVAS score at the time of diagnosis was 14 (IQR 11,19). 20 patients (51%) achieved remission 4-6 months after diagnosis and 25 patients (64%) were in remission at 12-months. 6 relapses occurred in 20 (30%) patients following remission, all were minor. At 12-months two patients were dialysis dependent and two patients had received renal transplants. No deaths occurred.
Primary treatments used for remission induction were: cyclophosphamide (CYC) in 29 (74%), methotrexate in 8 (21%), and rituximab (RTX) in 2 (5%). Plasmapheresis was used in conjunction with CYC or RTX in 8 patients. The most common maintenance treatments were azathioprine (38%), methotrexate (36%), and mycophenolate mofetil (18%). 37 (95%) of patients received CS in addition to their primary treatments. At 12-months, 22 patients (56%) were off (n=14) or on a low dose ≤ 0.1mg/kg/day (n=8) CS.
Following time-of-diagnosis, 20 patients required 31 hospitalizations: 16 in relation to underlying disease; 7 due to infection; 7 due to health problems unrelated to vasculitis; 1 due to medication effects.
The median score of a pediatric modification of the vasculitis damage index (pVDI) score completed for 21 patients at 12-months was 1 (range 0-5).
Conclusion: A significant proportion of children with AAV do not achieve remission following remission induction treatment, or at 12-months despite aggressive therapy. Compared to adults with AAV, remission rates may be lower in children and about half of children remain on corticosteroids at 12 months.
Disclosure:
K. Morishita,
None;
S. Benseler,
None;
R. S. M. Yeung,
Novartis Pharmaceutical Corporation,
2;
T. Mason II,
None;
D. Wahezi,
None;
K. N. Schikler,
Roche Genentech,
2;
E. F. Lawson,
None;
S. Nielsen,
None;
S. Charuvanij,
None;
P. Dancey,
None;
S. Shenoi,
None;
L. Wagner-Weiner,
None;
A. Sarmiento,
None;
D. A. Cabral,
None;
F. T. P. Initiative,
None.
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ACR Meeting Abstracts - https://acrabstracts.org/abstract/early-outcomes-in-pediatric-antineutrophil-cytoplasmic-antibody-anca-associated-vasculitis-aav/