OMERACT Endorsement of Patient-reported Outcome Instruments in Antineutrophil Cytoplasmic Antibody–associated Vasculitis

Joanna C. Robson; Gunnar Tomasson; Nataliya Milman; Sue Ashdown; Annelies Boonen; George C. Casey; Peter F. Cronholm; David Cuthbertson; Jill Dawson; Haner Direskeneli; Ebony Easley; Tanaz A. Kermani; John T. Farrar; Don Gebhart; Georgia Lanier; Raashid A. Luqmani; Alfred Mahr; Carol A. McAlear; Jacqueline Peck; Beverley Shea; Judy A. Shea; Antoine G. Sreih; Peter S. Tugwell; Peter A. Merkel

doi:10.3899/jrheum.161139

Abstract

Objective. The antineutrophil cytoplasmic antibody–associated vasculitides (AAV) are multiorgan diseases. Patients with AAV report impairment in their health-related quality of life (HRQOL) and have different priorities regarding disease assessment compared with physicians. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group previously received endorsement for a core set of domains in AAV. Two approaches to measure patient-reported outcomes (PRO) were presented at OMERACT 2016.

Methods. A novel 5-step tool was used to facilitate assessment of the instruments by delegates: the OMERACT Filter 2.0 Instrument Selection Algorithm, with a red-amber-green checklist of questions, including (1) good match with domain (face and content validity), (2) feasibility, (3) do numeric scores make sense (construct validity)?, (4) overall ratings of discrimination, and (5) can individual thresholds of meaning be defined? Delegates gave an overall endorsement. Three generic Patient-Reported Outcomes Measurement Information System (PROMIS) instruments (fatigue, physical functioning, and pain interference) and a disease-specific PRO, the AAV-PRO (6 domains related to symptoms and HRQOL), were presented.

Results. OMERACT delegates endorsed the use of the PROMIS instruments for fatigue, physical functioning, and pain interference (87.6% overall endorsement) and the disease-specific AAV-PRO instrument (89.4% overall endorsement).

Conclusion. The OMERACT Vasculitis Working Group gained endorsement by OMERACT for use of the PROMIS and the AAV-PRO in clinical trials of vasculitis. These instruments are complementary to each other. The PROMIS and the AAV-PRO need further work to assess their utility in longitudinal settings, including their ability to discriminate between treatments of varying efficacy in the setting of a randomized controlled trial.

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Footnotes

Supported through 2 Patient Centered Outcomes Research Institute Awards: Contract Number IP2-PI000603 for Pilot Award and Contract Number PPRN-1306-04758 for the Vasculitis Patient-Powered Research Network (part of the Patient-Centered Outcomes Research Network). The Vasculitis Clinical Research Consortium (VCRC; U54 AR057319, U01 AR5187404, and R01 AR064153) is part of the Rare Diseases Clinical Research Network, an initiative of the Office of Rare Diseases Research, and the US National Center for Advancing Translational Science (NCATS). The VCRC is funded through collaboration between NCATS and the US National Institute of Arthritis and Musculoskeletal and Skin Diseases, and has received funding from the National Center for Research Resources (U54 RR019497). UK funding is from the Medical Research Fund, Oxford, and the Oxfordshire Health Services Research Committee, Oxford. Patient survey support is from Vasculitis UK. Dr. Robson was supported by a UK National Institute for Health Research Academic Clinical Lectureship. 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.