Exploration, Development, and Validation of Patient-reported Outcomes in Antineutrophil Cytoplasmic Antibody-associated Vasculitis Using the OMERACT Process

Joanna C Robson; Nataliya Milman; Gunnar Tomasson; Jill Dawson; Peter F Cronholm; Katherine Kellom; Judy Shea; Susan Ashdown; Maarten Boers; Annelies Boonen; George C Casey; John T Farrar; Don Gebhart; Jeffrey Krischer; Georgia Lanier; Carol A McAlear; Jacqueline Peck; Antoine G Sreih; Peter S Tugwell; Raashid A Luqmani; Peter A Merkel

doi:10.3899/jrheum.141143

Exploration, Development, and Validation of Patient-reported Outcomes in Antineutrophil Cytoplasmic Antibody-associated Vasculitis Using the OMERACT Process

J Rheumatol. 2015 Nov;42(11):2204-9. doi: 10.3899/jrheum.141143. Epub 2015 Sep 1.

Authors

Joanna C Robson¹, Nataliya Milman¹, Gunnar Tomasson¹, Jill Dawson¹, Peter F Cronholm¹, Katherine Kellom¹, Judy Shea¹, Susan Ashdown¹, Maarten Boers¹, Annelies Boonen¹, George C Casey¹, John T Farrar¹, Don Gebhart¹, Jeffrey Krischer¹, Georgia Lanier¹, Carol A McAlear¹, Jacqueline Peck¹, Antoine G Sreih¹, Peter S Tugwell¹, Raashid A Luqmani¹, Peter A Merkel¹

Affiliation

¹ From the Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Science, University of Oxford, Oxford, UK; Division of Rheumatology, University of Ottawa, Ottawa, Ontario, Canada; Department of Public Health Sciences, University of Iceland, Reykjavik, Iceland; Nuffield Department of Population Health (HSRU), University of Oxford, Oxford, UK; Department of Family Medicine and Community Health, and Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania, and Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA; Departments of Epidemiology and Biostatistics; and Rheumatology, VU University Medical Center, Amsterdam, The Netherlands; Division of Rheumatology, Maastricht University Medical Center and Caphri Research Institute, University Maastricht, Maastricht, The Netherlands; Vasculitis Foundation, Denver, Colorado, USA; Division of Biostatistics and Informatics, Department of Pediatrics, College of Medicine, University of South Florida, Tampa, Florida, USA; Vasculitis Clinical Research Consortium, and Division of Rheumatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA.J.C. Robson, MBBS, PhD, Clinical Lecturer in Rheumatology, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Science, University of Oxford; N. Milman, MD, FRCPC, Clinical Lecturer in Rheumatology, Division of Rheumatology, University of Ottawa; G. Tomasson, MD, Department of Public Health Sciences, University of Iceland; J. Dawson, MA, MSc, DPhil, University Research Lecturer and Senior Research Scientist, Nuffield Department of Population Health (HSRU), University of Oxford; P.F. Cronholm, MD, MSCE, FAAFP, Associate Professor, Department of Family Medicine and Community Health, University of Pennsylvania; K. Kellom, BA, Senior Research Coordinator, Department of Family Medicine and Community Health, University of Pennsylvania; J. Shea, PhD, Professor of Medici

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.

Keywords: ANCA; OUTCOMES; PATIENTS; VASCULITIS.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review
Validation Study

MeSH terms

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis / diagnosis
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis / therapy*
Clinical Trials as Topic / standards
Consensus Development Conferences as Topic*
Female
Humans
Male
Outcome Assessment, Health Care*
Patient Outcome Assessment*
Practice Guidelines as Topic / standards*
Severity of Illness Index

Abstract

Publication types

MeSH terms

Grants and funding