风湿性多肌痛未来研究中使用的结局指标的可行性和表面效度：一项OMERACT研究
Feasibility and Face Validity of Outcome Measures for Use in Future Studies of Polymyalgia Rheumatica: An OMERACT Study.
作者信息
Yates Max, Owen Claire E, Muller Sara, Graham Karly, Neill Lorna, Twohig Helen, Boers Maarten, Pujades Rodriguez Mar, Goodman Susan M, Cheah Jonathan, Dejaco Christian, Mukhtyar Chetan, Nielsen Berit Dalsgaard, Robson Joanna, Simon Lee S, Shea Beverley, Mackie Sarah L, Hill Catherine L
机构信息
From the Epidemiology Centre Versus Arthritis, Norwich Medical School, University of East Anglia, Norwich, UK; Department of Rheumatology, East Suffolk and North Essex Foundation Trust, Ipswich, UK; Department of Rheumatology, Austin Health; Department of Medicine, University of Melbourne, Melbourne, Australia; Primary Care Centre Versus Arthritis, Research Institute for Primary Care and Health Sciences, Keele University, Keele; PMRGCA Scotland, Scotland, UK; Department of Epidemiology and Biostatistics, and Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands; Leeds Institute of Health Sciences, University of Leeds, Leeds, UK; Department of Rheumatology, and Department of Medicine, Hospital for Special Surgery, New York, New York, USA; Department of Rheumatology and Immunology, Medical University Graz, Graz, Austria; Department of Rheumatology, Hospital of Bruneck, Bruneck, Italy; Department of Rheumatology, Norfolk and Norwich University Hospital, Norwich, UK; Department of Rheumatology, Aarhus University Hospital, Aarhus, Denmark; Department of Rheumatology, Faculty of Health and Applied Sciences, University of the West of England, Bristol, UK; SDG LLC, Cambridge, Massachusetts, USA; School of Epidemiology and Public Health, University of Ottawa, Ottawa, Ontario, Canada; Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds; National Institute for Health Research (NIHR) Leeds Biomedical Research Centre, Leeds Teaching Hospitals National Health Service (NHS) Trust, Leeds, UK; Discipline of Medicine, The University of Adelaide; the Rheumatology Unit, The Queen Elizabeth and Royal Adelaide Hospitals, Adelaide, Australia.
M. Yates, BSc (Hons), MBBS, MSc, PhD, Epidemiology Centre Versus Arthritis, Norwich Medical School, University of East Anglia, and Department of Rheumatology, East Suffolk and North Essex Foundation Trust; C.E. Owen, MBBS (Hons), Department of Rheumatology, Austin Health, Department of Medicine, University of Melbourne; S. Muller, BSc (Hons), MSc, PhD, Primary Care Centre Versus Arthritis, Research Institute for Primary Care and Health Sciences, Keele University; K. Graham, Epidemiology Centre Versus Arthritis, Norwich Medical School, University of East Anglia; L. Neill, BSc (Hons), Chair, PMRGCA Scotland; H. Twohig, MBChB, BMedSci, PhD, Primary Care Centre Versus Arthritis, Research Institute for Primary Care and Health Sciences, Keele University; M. Boers, MSc, MD, PhD, Department of Epidemiology and Biostatistics, and Amsterdam Rheumatology and Immunology Center, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam; M. Pujades Rodriguez, MSc, PhD, Leeds Institute of Health Sciences, University of Leeds; S.M. Goodman, MD, Department of Rheumatology, Hospital for Special Surgery; J. Cheah, MD, Department of Medicine, Hospital for Special Surgery; C. Dejaco, MD, PhD, MBA, Department of Rheumatology and Immunology, Medical University Graz, Department of Rheumatology, Hospital of Bruneck; C. Mukhtyar, MBBS, MSc, MD, Department of Rheumatology, Norfolk and Norwich University Hospital; B. Dalsgaard Nielsen, MD, Department of Rheumatology, Norfolk and Norwich University Hospital; J. Robson, MBBS, PhD, Department of Rheumatology, Faculty of Health and Applied Sciences, University of the West of England; L.S. Simon, MD, SDG LLC; B. Shea, RN, BScN, MSc, PhD (epi), School of Epidemiology and Public Health, University of Ottawa; S.L. Mackie, BM, BCh, PhD, Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals NHS Trust; C.L. Hill, MBBS, MD, MSc, Discipline of Medicine, The University of Adelaide, and Rheumatology Unit, The Queen Elizabeth and Royal Adelaide Hospitals.
出版信息
J Rheumatol. 2020 Sep 1;47(9):1379-1384. doi: 10.3899/jrheum.190575. Epub 2020 Feb 1.
OBJECTIVE
To survey participants with polymyalgia rheumatica (PMR) to evaluate the face validity, acceptability, and domain match of proposed candidate outcome measures.
METHODS
A structured, online, anonymous survey was disseminated by patient support groups through their networks and online forums. The candidate outcome measures comprised (1) visual analog scale (VAS) and numerical rating score (NRS) to assess pain; (2) VAS, NRS, and duration to assess stiffness; (3) the modified Health Assessment Questionnaire and Health Assessment Questionnaire Disability Index to assess physical function; and (4) C-reactive protein and erythrocyte sedimentation rate to assess inflammation. Free-text answers were analyzed using descriptive thematic analysis to determine respondents' views of the candidate instruments.
RESULTS
Seventy-eight people with PMR from 6 countries (UK, France, USA, Canada, Australia, and New Zealand) participated in the survey. Most respondents agreed candidate instruments were acceptable or "good to go." Free-text analysis identified 5 themes that participants considered inadequately covered by the proposed instruments. These related to (1) the variability, context, and location of pain; (2) the variability of stiffness; (3) fatigue; (4) disability; and (5) the correlation of inflammatory marker levels and severity of symptoms, sometimes reflecting disease activity and other times not.
CONCLUSION
Participants reported additional aspects of their experience that are not covered by the proposed instruments, particularly for the experience of stiffness and effect of fatigue. New patient-reported outcome measures are required to increase the relevance of results from clinical trials to patients with PMR.
目的
对风湿性多肌痛（PMR）患者进行调查，以评估拟议的候选结局指标的表面效度、可接受性和领域匹配度。
方法
患者支持小组通过其网络和在线论坛开展了一项结构化、在线、匿名调查。候选结局指标包括：（1）用于评估疼痛的视觉模拟量表（VAS）和数字评定量表（NRS）；（2）用于评估僵硬的VAS、NRS和持续时间；（3）用于评估身体功能的改良健康评估问卷和健康评估问卷残疾指数；以及（4）用于评估炎症的C反应蛋白和红细胞沉降率。使用描述性主题分析对自由文本答案进行分析，以确定受访者对候选工具的看法。
结果
来自6个国家（英国、法国、美国、加拿大、澳大利亚和新西兰）的78名PMR患者参与了调查。大多数受访者认为候选工具是可接受的或“可以使用”。自由文本分析确定了5个主题，参与者认为拟议的工具未充分涵盖这些主题。这些主题涉及：（1）疼痛的变异性、背景和部位；（2）僵硬的变异性；（3）疲劳；（4）残疾；以及（5）炎症标志物水平与症状严重程度的相关性，有时反映疾病活动，有时则不然。
结论
参与者报告了其经历中拟议工具未涵盖的其他方面，特别是僵硬的体验和疲劳的影响。需要新的患者报告结局指标，以提高临床试验结果与PMR患者的相关性。
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