Therapeutic advances in the treatment of vasculitis.

Despina Eleftheriou,Paul A Brogan

doi:10.1186/s12969-016-0082-8

Abstract

Considerable therapeutic advances for the treatment of vasculitis of the young have been made in the past 10 years, including the development of outcome measures that facilitate clinical trial design. Notably, these include: a recognition that some patients with Kawasaki Disease require corticosteroids as primary treatment combined with IVIG; implementation of rare disease trial design for polyarteritis nodosa to deliver the first randomised controlled trial for children; first clinical trials involving children for anti-neutrophil cytoplasmic antibody (ANCA) vasculitis; and identification of monogenic forms of vasculitis that provide an understanding of pathogenesis, thus facilitating more targeted treatment. Robust randomised controlled trials for Henoch Schönlein Purpura nephritis and Takayasu arteritis are needed; there is also an over-arching need for trials examining new agents that facilitate corticosteroid sparing, of particular importance in the paediatric population since glucocorticoid toxicity is a major concern.

Highlights

Primary systemic vasculitides of the young are relatively rare diseases, but are associated with significant morbidity and mortality, if there is diagnostic delay [1, 2]
This review summarises therapeutic advances in current management strategies for systemic vasculitides, and describes important unmet needs
Treatment for paediatric associated vasculitis (AAV) is broadly similar to the approach in adults based on evidence derived from a number of clinical trials conducted by the European Vasculitis Study group (EUVAS) [19]

Summary

Introduction

Primary systemic vasculitides of the young are relatively rare diseases, but are associated with significant morbidity and mortality, if there is diagnostic delay [1, 2]. There have been a number of notable advances recently in the field of paediatric vasculitis research, including the development of classification criteria; and tools to assess disease outcome for use in clinical trials and other research involving children with vasculitis [3, 4].

Results

Conclusion