Sarah L Mackie1,2, Helen Twohig3,4, Lorna M Neill3,4, Eileen Harrison3,4, Beverley Shea3,4, Rachel J Black3,4, Tanaz A Kermani3,4, Peter A Merkel3,4, Christian D Mallen3,4, Frank Buttgereit3,4, Chetan Mukhtyar3,4, Lee S Simon3,4, Catherine L Hill3,4. 1. From the Leeds Institute of Rheumatic and Musculoskeletal Medicine (LIRMM), University of Leeds, and UK National Institute for Health Research (NIHR), Leeds; Academic Unit of Primary Medical Care, University of Sheffield, Sheffield; PMR-GCA Scotland, Dundee; PMR-GCA North East, Gateshead; Primary Care and Health Sciences, Keele University, Keele; Norfolk and Norwich University Hospital, Norwich, UK; Ottawa Hospital Research Institute and School of Epidemiology, Public Health and Preventative Medicine, University of Ottawa, Ottawa, Ontario, Canada; Discipline of Medicine, The University of Adelaide; Rheumatology Unit, The Queen Elizabeth Hospital, Adelaide, Australia; Division of Rheumatology, University of California Los Angeles (UCLA), Los Angeles, California; Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, Pennsylvania; SDG LLC, Cambridge, Massachusetts, USA; Department of Rheumatology and Clinical Immunology, Charite University Hospital Berlin, Berlin, Germany. s.l.mackie@leeds.ac.uk. 2. S.L. Mackie, BM, BCh, PhD, Associate Clinical Professor and Honorary Consultant Rheumatologist, LIRM, University of Leeds; H. Twohig, MBChB, General Practitioner, Academic Unit of Primary Medical Care, University of Sheffield; L.M. Neill, BSc, PMR-GCA Scotland and OMERACT Patient Research Partner; E. Harrison, BSc, PMR-GCA North East and OMERACT Patient Research Partner; B. Shea, PhD, Senior Methodologist and Adjunct Professor, Ottawa Hospital Research Institute and School of Epidemiology, Public Health and Preventative Medicine, University of Ottawa; R.J. Black, MBBS, Consultant Rheumatologist and Clinical Lecturer, Discipline of Medicine, The University of Adelaide; T.A. Kermani, MD, MS, Assistant Clinical Professor, Division of Rheumatology, UCLA; P.A. Merkel, MD, MPH, Professor of Medicine and Epidemiology, Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania; C.D. Mallen, PhD, NIHR Research Professor in General Practice, Arthritis Research UK Primary Care Centre, Research Institute for Primary Care, Keele University; F. Buttgereit, MD, Professor of Rheumatology, Department of Rheumatology and Clinical Immunology, Charite University Hospital Berlin; C. Mukhtyar, MD, Consultant Rheumatologist, Norfolk and Norwich University Hospital; L.S. Simon, MD, Principal, SDG LLC; C.L. Hill, MD, Clinical Professor and Consultant Rheumatologist, Discipline of Medicine, The University of Adelaide, and Rheumatology Unit, The Queen Elizabeth Hospital. s.l.mackie@leeds.ac.uk. 3. From the Leeds Institute of Rheumatic and Musculoskeletal Medicine (LIRMM), University of Leeds, and UK National Institute for Health Research (NIHR), Leeds; Academic Unit of Primary Medical Care, University of Sheffield, Sheffield; PMR-GCA Scotland, Dundee; PMR-GCA North East, Gateshead; Primary Care and Health Sciences, Keele University, Keele; Norfolk and Norwich University Hospital, Norwich, UK; Ottawa Hospital Research Institute and School of Epidemiology, Public Health and Preventative Medicine, University of Ottawa, Ottawa, Ontario, Canada; Discipline of Medicine, The University of Adelaide; Rheumatology Unit, The Queen Elizabeth Hospital, Adelaide, Australia; Division of Rheumatology, University of California Los Angeles (UCLA), Los Angeles, California; Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, Pennsylvania; SDG LLC, Cambridge, Massachusetts, USA; Department of Rheumatology and Clinical Immunology, Charite University Hospital Berlin, Berlin, Germany. 4. S.L. Mackie, BM, BCh, PhD, Associate Clinical Professor and Honorary Consultant Rheumatologist, LIRM, University of Leeds; H. Twohig, MBChB, General Practitioner, Academic Unit of Primary Medical Care, University of Sheffield; L.M. Neill, BSc, PMR-GCA Scotland and OMERACT Patient Research Partner; E. Harrison, BSc, PMR-GCA North East and OMERACT Patient Research Partner; B. Shea, PhD, Senior Methodologist and Adjunct Professor, Ottawa Hospital Research Institute and School of Epidemiology, Public Health and Preventative Medicine, University of Ottawa; R.J. Black, MBBS, Consultant Rheumatologist and Clinical Lecturer, Discipline of Medicine, The University of Adelaide; T.A. Kermani, MD, MS, Assistant Clinical Professor, Division of Rheumatology, UCLA; P.A. Merkel, MD, MPH, Professor of Medicine and Epidemiology, Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania; C.D. Mallen, PhD, NIHR Research Professor in General Practice, Arthritis Research UK Primary Care Centre, Research Institute for Primary Care, Keele University; F. Buttgereit, MD, Professor of Rheumatology, Department of Rheumatology and Clinical Immunology, Charite University Hospital Berlin; C. Mukhtyar, MD, Consultant Rheumatologist, Norfolk and Norwich University Hospital; L.S. Simon, MD, Principal, SDG LLC; C.L. Hill, MD, Clinical Professor and Consultant Rheumatologist, Discipline of Medicine, The University of Adelaide, and Rheumatology Unit, The Queen Elizabeth Hospital.
Abstract
OBJECTIVE: To inform development of a core domain set for outcome measures for clinical trials in polymyalgia rheumatica (PMR), we conducted patient consultations, a systematic review, a Delphi study, and 2 qualitative studies. METHODS: Domains identified by 70% or more of physicians and/or patients in the Delphi study were selected. The conceptual framework derived from the 2 qualitative research studies helped inform the meaning of each domain and its relationship to the others. The draft core domain set was refined by further discussion with patients and physicians who had participated in the Delphi study. At the Outcome Measures in Rheumatology (OMERACT) 2016, the domains were discussed and prioritized by 8 breakout groups. Formal voting took place at the end of the workshop and in the final plenary. RESULTS: Ninety-three percent of voters in the final plenary agreed that the inner core of domains considered mandatory for clinical trials of PMR should consist the following: laboratory markers of systemic inflammation, pain, stiffness, and physical function. Patient's global and fatigue were considered important but not mandatory (outer core). The research agenda included psychological impact, weakness, physical activity, participation, sleep, imaging, and health-related quality of life. CONCLUSION: This core domain set was considered sufficiently well-defined that the next step will be to apply the OMERACT Filter 2.0 Instrument Selection Algorithm to select candidate instruments for a subsequent "deeper dive" into the data. This will allow instruments to be mapped onto each of our core domains to derive a core outcome set for PMR.
OBJECTIVE: To inform development of a core domain set for outcome measures for clinical trials in polymyalgia rheumatica (PMR), we conducted patient consultations, a systematic review, a Delphi study, and 2 qualitative studies. METHODS: Domains identified by 70% or more of physicians and/or patients in the Delphi study were selected. The conceptual framework derived from the 2 qualitative research studies helped inform the meaning of each domain and its relationship to the others. The draft core domain set was refined by further discussion with patients and physicians who had participated in the Delphi study. At the Outcome Measures in Rheumatology (OMERACT) 2016, the domains were discussed and prioritized by 8 breakout groups. Formal voting took place at the end of the workshop and in the final plenary. RESULTS: Ninety-three percent of voters in the final plenary agreed that the inner core of domains considered mandatory for clinical trials of PMR should consist the following: laboratory markers of systemic inflammation, pain, stiffness, and physical function. Patient's global and fatigue were considered important but not mandatory (outer core). The research agenda included psychological impact, weakness, physical activity, participation, sleep, imaging, and health-related quality of life. CONCLUSION: This core domain set was considered sufficiently well-defined that the next step will be to apply the OMERACT Filter 2.0 Instrument Selection Algorithm to select candidate instruments for a subsequent "deeper dive" into the data. This will allow instruments to be mapped onto each of our core domains to derive a core outcome set for PMR.
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