Abstract
Objective. Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of linked multisystem life- and organ-threatening diseases. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group has been at the forefront of outcome development in the field and has achieved OMERACT endorsement of a core set of outcomes for AAV. Patients with AAV report as important some manifestations of disease not routinely collected through physician-completed outcome tools; and they rate common manifestations differently from investigators. The core set includes the domain of patient-reported outcomes (PRO). However, PRO currently used in clinical trials of AAV do not fully characterize patients’ perspectives on their burden of disease. The OMERACT vasculitis working group is addressing the unmet needs for PRO in AAV.
Methods. Current activities of the working group include (1) evaluating the feasibility and construct validity of instruments within the PROMIS (Patient-Reported Outcome Measurement Information System) to record components of the disease experience among patients with AAV; (2) creating a disease-specific PRO measure for AAV; and (3) applying The International Classification of Functioning, Disability and Health to examine the scope of outcome measures used in AAV.
Results. The working group has developed a comprehensive research strategy, organized an investigative team, included patient research partners, obtained peer-reviewed funding, and is using a considerable research infrastructure to complete these interrelated projects to develop evidence-based validated outcome instruments that meet the OMERACT filter of truth, discrimination, and feasibility.
Conclusion. The OMERACT vasculitis working group is on schedule to achieve its goals of developing validated PRO for use in clinical trials of AAV.
Footnotes
Sponsored by the Vasculitis Clinical Research Consortium, with support from the US National Institute of Arthritis and Musculoskeletal and Skin Diseases (U54 AR057319 and U01 AR51874), the National Center for Research Resources (U54 RR019497); and the Office of Rare Diseases Research and the National Center for Advancing Translational Science. Additional support for the work of the OMERACT vasculitis working group was received by a Patient-Centered Outcomes Research Institute Pilot Project Grant, the Medical Research Fund, Oxford, and the Oxfordshire Health Services Research Committee. Dr. Robson and Professor Luqmani are supported in part by the National Institute for Health Research Musculoskeletal Biomedical Research Unit, Oxford, UK. Dr. Milman was supported by a UCB/Canadian Rheumatology Association/Arthritis Society postgraduate rheumatology fellowship award and a research fellowship from the Department of Medicine at the Ottawa Hospital.
