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Pediatric Nephrology
Published in: Pediatric Nephrology 11/2019

01-11-2019 | Educational Review

Biologics for childhood systemic vasculitis

Authors: Keiji Akamine, Marilynn Punaro

Published in: Pediatric Nephrology | Issue 11/2019

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Abstract

Recent advances have allowed better understanding of vasculitis pathogenesis and led to more targeted therapies. Two pivotal randomized controlled trials, RITUXVAS and rituximab in ANCA-associated vasculitis (RAVE), provide high-quality evidence demonstrating rituximab (RTX) is efficacious in inducing remission in adult ANCA-associated vasculitis (AAV) patients compared with cyclophosphamide (CYC). RAVE also demonstrated superiority of RTX to oral CYC for induction of remission in relapsing disease. Disappointingly, the RTX regimen was not associated with reduction in early serious adverse events. At least nine randomized trials are in progress, aiming to further delineate optimal dosing and duration of RTX therapy in AAV. In particular, the 6-month interim results of the PEPRS trial provide encouraging data specific to children. Due to special concerns related to growth, preservation of fertility, and potential for high cumulative medication doses, children with AAV should be considered as candidates for RTX even as a first-line remission induction therapy. Two randomized clinical trials have defined the role of infliximab in Kawasaki disease (KD), which appears to be as an alternative to a second infusion of intravenous immunoglobulin (IVIG) for treatment-resistant disease. Support for other biologics in the treatment of AAV or for biologics in the treatment of other vasculidities is largely lacking due to either unimpressive trial results or lack of trials. Except for the KD trials and the PEPRS, trials enrolling children remain scant. This review touches on the key trials and case series with biologics in the treatment of vasculitis that have influenced practice and shaped current thinking.
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Metadata
Title
Biologics for childhood systemic vasculitis
Authors
Keiji Akamine
Marilynn Punaro
Publication date
01-11-2019
Publisher
Springer Berlin Heidelberg
Published in
Pediatric Nephrology / Issue 11/2019
Print ISSN: 0931-041X
Electronic ISSN: 1432-198X
DOI
https://doi.org/10.1007/s00467-018-4076-2

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