Outcome measures for antineutrophil cytoplasmic antibody-associated vasculitis.

Abstract

To describe the design and validation of current outcome measures for antineutrophil cytoplasmic antibody-associated vasculitis, and provide insight into ongoing outcome measure development for future randomized controlled trials (RCTs). Substantial evidence now supports that patient-reported outcomes (PROs) capture unique disease components not represented in current physician-based measures. Further incorporation of formal disease assessment in the setting of RCT will both improve disease assessment in terms of adding new important disease domains and could improve psychometric properties of outcome measures and, therefore, increase trial efficiency. Optimal outcome measures for AAV promote identification of therapeutic agents that treat all important disease manifestations.