用于巨细胞动脉炎临床试验结局测量的 OMERACT 核心域集。
The OMERACT Core Domain Set for Outcome Measures for Clinical Trials in Polymyalgia Rheumatica.
机构信息
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, 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.
出版信息
J Rheumatol. 2017 Oct;44(10):1515-1521. doi: 10.3899/jrheum.161109. Epub 2017 Aug 1.
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.
目的
为了制定用于巨细胞动脉炎临床试验的结局测量核心领域集，我们进行了患者咨询、系统评价、德尔菲研究和 2 项定性研究。
方法
在德尔菲研究中，有 70%或以上的医生和/或患者确定的领域被选中。从 2 项定性研究中得出的概念框架有助于理解每个领域的含义及其与其他领域的关系。通过与参与德尔菲研究的患者和医生进一步讨论，对草案核心领域集进行了完善。在 2016 年风湿病结局测量（OMERACT）会议上，8 个分组讨论并确定了这些领域的优先级。在研讨会结束时和全体会议上进行了正式投票。
结果
在最终全体会议上，93%的投票者同意，巨细胞动脉炎临床试验中必须包含以下核心领域：系统性炎症的实验室标志物、疼痛、僵硬和身体功能。患者的整体状况和疲劳被认为是重要的，但不是强制性的（外核心）。研究议程包括心理影响、乏力、身体活动、参与度、睡眠、影像学和健康相关生活质量。
结论
该核心领域集被认为定义得足够明确，下一步将应用 OMERACT Filter 2.0 仪器选择算法来选择候选仪器，以便对数据进行更深入的研究。这将允许将仪器映射到我们的每个核心领域，为巨细胞动脉炎制定一个核心结局集。
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