title: 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.
authors.affiliation: Renal and Vascular Inflammation Section, Department of Medicine, Hammersmith Hospital Campus, Imperial College London, Du Cane Road, London W12 0NN, UK.
authors.forename: Ruth M
authors.initials: RM
authors.lastname: Tarzi
authors.affiliation: Renal and Vascular Inflammation Section, Department of Medicine, Hammersmith Hospital Campus, Imperial College London, Du Cane Road, London W12 0NN, UK.
authors.forename: Justin C
authors.initials: JC
authors.lastname: Mason
authors.affiliation: Vascular Sciences Unit, National Heart and Lung Institute, Hammersmith Hospital Campus, Imperial College London, Du Cane Road, London W12 0NN, UK.
authors.forename: Charles D
authors.initials: CD
authors.lastname: Pusey
mesh_terms: D056648:Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis / Q000097:blood / Q000175:diagnosis* / Q000188:drug therapy*; D019268:Antibodies, Antineutrophil Cytoplasmic / Q000097:blood*; D015415:Biomarkers / Q000097:blood; D019317:Evidence-Based Medicine; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D016032:Randomized Controlled Trials as Topic; D012107:Research Design / Q000639:trends*; D016896:Treatment Outcome; D014657:Vasculitis / Q000175:diagnosis / Q000188:drug therapy
chemical_list: D019268:Antibodies, Antineutrophil Cytoplasmic; D015415:Biomarkers; D007166:Immunosuppressive Agents