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Issues in trial design for ANCA-associated and large-vessel vasculitis

Abstract

Randomized clinical trials (RCTs) have informed the management of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis, although challenges still exist. The evidence base for treating large-vessel vasculitis (LVV) is weaker, but initiatives to standardize diagnostic criteria and outcome measures, and to validate biomarkers in LVV, together with newly initiated RCTs should start to address this need. In this Perspectives, we discuss the prerequisites for RCTs in vasculitis, existing trial evidence, continuing unmet needs, potential therapeutic avenues to explore and considerations in the design of future trials.

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Figure 1: Potential therapeutic targets in AAV and LVV.

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Acknowledgements

R.M.T. gratefully acknowledges salary support from an Arthritis Research UK Clinician Scientist Fellowship. The authors acknowledge support from the Imperial NIHR Biomedical Research Centre.

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R.M.T. and J.C.M. researched data for and wrote the article. All authors made equal contributions to discussion of content and reviewing/editing the manuscript before submission.

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Correspondence to Ruth M. Tarzi.

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C.D.P. has received a research grant from GlaxoSmithKline. R.M.T. and J.C.M. declare no competing interests.

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Tarzi, R., Mason, J. & Pusey, C. Issues in trial design for ANCA-associated and large-vessel vasculitis. Nat Rev Rheumatol 10, 502–510 (2014). https://doi.org/10.1038/nrrheum.2014.67

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