Joanna C Robson1,2, Gunnar Tomasson1,2, Nataliya Milman1,2, Sue Ashdown1,2, Annelies Boonen1,2, George C Casey1,2, Peter F Cronholm1,2, David Cuthbertson1,2, Jill Dawson1,2, Haner Direskeneli1,2, Ebony Easley1,2, Tanaz A Kermani1,2, John T Farrar1,2, Don Gebhart1,2, Georgia Lanier1,2, Raashid A Luqmani1,2, Alfred Mahr1,2, Carol A McAlear1,2, Jacqueline Peck1,2, Beverley Shea1,2, Judy A Shea1,2, Antoine G Sreih1,2, Peter S Tugwell1,2, Peter A Merkel3,4. 1. From the University of the West of England; University of Bristol; University Hospitals Bristol UK National Health Service (NHS) Trust, Bristol; Nuffield Department of Population Health, University of Oxford; Rheumatology, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK; Department of Public Health Sciences, University of Iceland, Reykjavik, Iceland; Division of Rheumatology, University of Ottawa; Division of Rheumatology, The Ottawa Hospital; Department of Clinical Epidemiology, Ottawa Hospital Research Institute; Ottawa Hospital Research Institute, School of Epidemiology, Public Health and Preventative Medicine, Faculty of Medicine, University of Ottawa; Department of Medicine, Faculty of Medicine, University of Ottawa; Ottawa Hospital Research Institute, Clinical Epidemiology Program, Ottawa, Ontario, Canada; Division of Rheumatology, Maastricht University Medical Centre; Caphri Graduate School, Maastricht, the Netherlands; Vasculitis Foundation, Denver, Colorado; Department of Family Medicine and Community Health, University of Pennsylvania; Epidemiology, Neurology, and Anesthesiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania; Department of Medicine, University of Pennsylvania; Clinical Medicine, Division of Rheumatology, University of Pennsylvania; Medicine and Epidemiology, Division of Rheumatology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania, Philadelphia, Pennsylvania; Division of Biostatistics and Informatics, Department of Pediatrics, University of South Florida, Tampa, Florida; Division of Rheumatology, Department of Medicine, University of California, Los Angeles, USA; Division of Rheumatology, Marmara University, School of Medicine, Marmara, Turkey; Medicine, Hopital Saint Louis, Paris, France. 2. J.C. Robson, MRCP, PhD, Consultant Senior Lecturer, University of the West of England, and Honorary Senior Lecturer, University of Bristol, and Honorary Consultant in Rheumatology, University Hospitals Bristol UK NHS Trust; G. Tomasson, MD, PhD, Assistant Professor of Medicine, Department of Public Health Sciences, University of Iceland; N. Milman, MD, Assistant Professor, Division of Rheumatology, University of Ottawa, and Division of Rheumatology, The Ottawa Hospital, and Department of Clinical Epidemiology, Ottawa Hospital Research Institute; S. Ashdown, RGN, RM, Patient-Partner; A. Boonen, MD, PhD, Professor of Rheumatology, Division of Rheumatology, Maastricht University Medical Centre, and Caphri Graduate School; George C. Casey, MBA, Patient-Partner, Vasculitis Foundation; P.F. Cronholm, MD, MSCE, Associate Professor of Family Medicine, Department of Family Medicine and Community Health, University of Pennsylvania; D. Cuthbertson, MA, Division of Biostatistics and Informatics, Department of Pediatrics, University of South Florida; J. Dawson, MA, MSc, DPhil, SRN, SCM, Associate Professor, Nuffield Department of Population Health, University of Oxford; H. Direskeneli, MD, Professor of Medicine, Division of Rheumatology, Marmara University, School of Medicine; E. Easley, Department of Family Medicine and Community Health, University of Pennsylvania; T.A. Kermani, MD, MS, Assistant Professor of Medicine, Division of Rheumatology, Department of Medicine, University of California; J.T. Farrar, MD, PhD, Associate Professor of Epidemiology, Neurology, and Anesthesiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania; D. Gebhart, MD, Patient-Partner; G. Lanier, Patient-Partner; R.A. Luqmani, DM, FRCP, FRCP(E), Professor of Rheumatology, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford; A. Mahr, MD, PhD, Professor of Medicine, Hopital Saint Louis; C.A. McAlear, MA, Division of Rheumatology, University of Pennsylvania; J. Peck, RN, Patient-Partner; B. Shea, MSc, PhD, Senior Methodologist, Ottawa Hospital Research Institute, Adjunct Professor School of Epidemiology, Public Health and Preventative Medicine, Faculty of Medicine, University of Ottawa; J.A. Shea, PhD, Professor of Medicine, Department of Medicine, University of Pennsylvania; A.G. Sreih, MD, Assistant Professor of Clinical Medicine, Division of Rheumatology, University of Pennsylvania; P.S. Tugwell, MD, MSc, Professor, Department of Medicine, Faculty of Medicine, University of Ottawa, and Ottawa Hospital Research Institute, Clinical Epidemiology Program, and School of Epidemiology, Public Health and Preventative Medicine, Faculty of Medicine, University of Ottawa; P.A. Merkel, MD, MPH, Professor of Medicine and Epidemiology, Division of Rheumatology, Department of Biostatistics, Epidemiology, and Informatics, and Department of Medicine, University of Pennsylvania. 3. From the University of the West of England; University of Bristol; University Hospitals Bristol UK National Health Service (NHS) Trust, Bristol; Nuffield Department of Population Health, University of Oxford; Rheumatology, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK; Department of Public Health Sciences, University of Iceland, Reykjavik, Iceland; Division of Rheumatology, University of Ottawa; Division of Rheumatology, The Ottawa Hospital; Department of Clinical Epidemiology, Ottawa Hospital Research Institute; Ottawa Hospital Research Institute, School of Epidemiology, Public Health and Preventative Medicine, Faculty of Medicine, University of Ottawa; Department of Medicine, Faculty of Medicine, University of Ottawa; Ottawa Hospital Research Institute, Clinical Epidemiology Program, Ottawa, Ontario, Canada; Division of Rheumatology, Maastricht University Medical Centre; Caphri Graduate School, Maastricht, the Netherlands; Vasculitis Foundation, Denver, Colorado; Department of Family Medicine and Community Health, University of Pennsylvania; Epidemiology, Neurology, and Anesthesiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania; Department of Medicine, University of Pennsylvania; Clinical Medicine, Division of Rheumatology, University of Pennsylvania; Medicine and Epidemiology, Division of Rheumatology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania, Philadelphia, Pennsylvania; Division of Biostatistics and Informatics, Department of Pediatrics, University of South Florida, Tampa, Florida; Division of Rheumatology, Department of Medicine, University of California, Los Angeles, USA; Division of Rheumatology, Marmara University, School of Medicine, Marmara, Turkey; Medicine, Hopital Saint Louis, Paris, France. pmerkel@upenn.edu. 4. J.C. Robson, MRCP, PhD, Consultant Senior Lecturer, University of the West of England, and Honorary Senior Lecturer, University of Bristol, and Honorary Consultant in Rheumatology, University Hospitals Bristol UK NHS Trust; G. Tomasson, MD, PhD, Assistant Professor of Medicine, Department of Public Health Sciences, University of Iceland; N. Milman, MD, Assistant Professor, Division of Rheumatology, University of Ottawa, and Division of Rheumatology, The Ottawa Hospital, and Department of Clinical Epidemiology, Ottawa Hospital Research Institute; S. Ashdown, RGN, RM, Patient-Partner; A. Boonen, MD, PhD, Professor of Rheumatology, Division of Rheumatology, Maastricht University Medical Centre, and Caphri Graduate School; George C. Casey, MBA, Patient-Partner, Vasculitis Foundation; P.F. Cronholm, MD, MSCE, Associate Professor of Family Medicine, Department of Family Medicine and Community Health, University of Pennsylvania; D. Cuthbertson, MA, Division of Biostatistics and Informatics, Department of Pediatrics, University of South Florida; J. Dawson, MA, MSc, DPhil, SRN, SCM, Associate Professor, Nuffield Department of Population Health, University of Oxford; H. Direskeneli, MD, Professor of Medicine, Division of Rheumatology, Marmara University, School of Medicine; E. Easley, Department of Family Medicine and Community Health, University of Pennsylvania; T.A. Kermani, MD, MS, Assistant Professor of Medicine, Division of Rheumatology, Department of Medicine, University of California; J.T. Farrar, MD, PhD, Associate Professor of Epidemiology, Neurology, and Anesthesiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania; D. Gebhart, MD, Patient-Partner; G. Lanier, Patient-Partner; R.A. Luqmani, DM, FRCP, FRCP(E), Professor of Rheumatology, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford; A. Mahr, MD, PhD, Professor of Medicine, Hopital Saint Louis; C.A. McAlear, MA, Division of Rheumatology, University of Pennsylvania; J. Peck, RN, Patient-Partner; B. Shea, MSc, PhD, Senior Methodologist, Ottawa Hospital Research Institute, Adjunct Professor School of Epidemiology, Public Health and Preventative Medicine, Faculty of Medicine, University of Ottawa; J.A. Shea, PhD, Professor of Medicine, Department of Medicine, University of Pennsylvania; A.G. Sreih, MD, Assistant Professor of Clinical Medicine, Division of Rheumatology, University of Pennsylvania; P.S. Tugwell, MD, MSc, Professor, Department of Medicine, Faculty of Medicine, University of Ottawa, and Ottawa Hospital Research Institute, Clinical Epidemiology Program, and School of Epidemiology, Public Health and Preventative Medicine, Faculty of Medicine, University of Ottawa; P.A. Merkel, MD, MPH, Professor of Medicine and Epidemiology, Division of Rheumatology, Department of Biostatistics, Epidemiology, and Informatics, and Department of Medicine, University of Pennsylvania. pmerkel@upenn.edu.
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.
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.
Authors: Gunnar Tomasson; Maarten Boers; Michael Walsh; Michael LaValley; David Cuthbertson; Simon Carette; John C Davis; Gary S Hoffman; Nader A Khalidi; Carol A Langford; Carol A McAlear; W Joseph McCune; Paul A Monach; Philip Seo; Ulrich Specks; Robert Spiera; E William St Clair; John H Stone; Steven R Ytterberg; Peter A Merkel Journal: Arthritis Care Res (Hoboken) Date: 2012-02 Impact factor: 4.794
Authors: Peter P Cheung; Maarten de Wit; Clifton O Bingham; John R Kirwan; Amye Leong; Lyn M March; Pam Montie; Marieke Scholte-Voshaar; Laure Gossec Journal: J Rheumatol Date: 2015-04-15 Impact factor: 4.666
Authors: Peter A Merkel; Sibel Z Aydin; Maarten Boers; Haner Direskeneli; Karen Herlyn; Philip Seo; Ravi Suppiah; Gunnar Tomasson; Raashid A Luqmani Journal: J Rheumatol Date: 2011-07 Impact factor: 4.666
Authors: L Gossec; M Dougados; N Rincheval; A Balanescu; D T Boumpas; S Canadelo; L Carmona; J-P Daurès; M de Wit; B A C Dijkmans; M Englbrecht; Z Gunendi; T Heiberg; J R Kirwan; E M Mola; M Matucci-Cerinic; K Otsa; G Schett; T Sokka; G A Wells; G J Aanerud; A Celano; A Dudkin; C Hernandez; K Koutsogianni; F N Akca; A-M Petre; P Richards; M Scholte-Voshaar; G Von Krause; T K Kvien Journal: Ann Rheum Dis Date: 2008-12-03 Impact factor: 19.103
Authors: 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 Journal: J Rheumatol Date: 2015-09-01 Impact factor: 4.666
Authors: Neil Basu; Andrew McClean; Lorraine Harper; Esther Nicole Amft; Neeraj Dhaun; Raashid A Luqmani; Mark A Little; David Rw Jayne; Oliver Flossmann; John McLaren; Vinod Kumar; Lars P Erwig; David M Reid; Gareth T Jones; Gary J Macfarlane Journal: Ann Rheum Dis Date: 2013-01-25 Impact factor: 19.103
Authors: Joanna Robson; Helen Doll; Ravi Suppiah; Oliver Flossmann; Lorraine Harper; Peter Höglund; David Jayne; Alfred Mahr; Kerstin Westman; Raashid Luqmani Journal: Ann Rheum Dis Date: 2013-11-15 Impact factor: 19.103
Authors: Nataliya Milman; Eilish McConville; Joanna C Robson; Annelies Boonen; Peter Tugwell; George A Wells; Dipayan Chaudhuri; Jill Dawson; Gunnar Tomasson; Susan Ashdown; Don Gebhart; Georgia Lanier; Jacqueline Peck; Carol A McAlear; Katherine S Kellom; Peter F Cronholm; Peter A Merkel Journal: J Rheumatol Date: 2019-02-01 Impact factor: 4.666