From the Division of Rheumatology, Department of Internal Medicine, Medical Research Center, College of Medicine, and Department of Molecular Medicine and Biopharmaceutical Sciences, Seoul National University, Seoul, South Korea; Department of Neurology, Care Science and Society, Division of Physiotherapy, and Department of Learning, Informatics and Medical Education, Karolinska Institutet; Function Area Occupational Therapy and Physical Therapy, Allied Health Professionals Function, Karolinska University Hospital; Division of Rheumatology, Rheumatology Unit, Department of Medicine, Karolinska University Hospital in Solna, Sweden; Division of Rheumatology, Department of Medicine, Johns Hopkins University, Baltimore, Maryland, USA.
J.K. Park, MD, Division of Rheumatology, Department of Internal Medicine, Seoul National University Hospital; H. Alexanderson, PhD, Physiotherapist, Department of Neurology, Care Science and Society, Division of Physiotherapy, Karolinska Institutet, and Function Area Occupational Therapy and Physical Therapy, Allied Health Professionals Function, Karolinska University Hospital; M. Regardt, PhD, Occupational Therapist, Department of Learning, Informatics and Medical Education, Karolinska Institutet, and Function Area Occupational Therapy and Physical Therapy, Allied Health Professionals Function, Karolinska University Hospital; L. Christopher-Stine, MD, Division of Rheumatology, Department of Medicine, Johns Hopkins University; C.A. Mecoli, MD, Division of Rheumatology, Department of Medicine, Johns Hopkins University; M.C. Domínguez, MD, Division of Rheumatology, Department of Medicine, Johns Hopkins University; C. Sarver, Patient Research Partner; I. de Groot, Patient Research Partner; I.E. Lundberg, MD, PhD, Division of Rheumatology, Rheumatology Unit, Department of Medicine, Karolinska University, and Hospital in Solna, Karolinska Institutet; C.O. Bingham 3rd, MD, Division of Rheumatology, Department of Medicine, Johns Hopkins University; Y.W. Song, MD, PhD, Division of Rheumatology, Department of Internal Medicine, Medical Research Center, College of Medicine, Department of Molecular Medicine and Biopharmaceutical Sciences, Seoul National University. Dr. Park and Dr. Mecoli contributed equally to this work.
J Rheumatol. 2017 Nov;44(11):1683-1687. doi: 10.3899/jrheum.161252. Epub 2017 Aug 1.
To define a set of core patient-reported domains and respective instruments for use in idiopathic inflammatory myopathies (IIM). Previously, we reported a systematic literature review on patient-reported outcomes (PRO) in IIM followed by conducting international focus groups to elicit patient perspectives of myositis symptoms and effects.
Based on qualitative content analysis of focus groups, an initial list of 26 candidate domains was constructed. We subsequently conducted an international modified Delphi survey to identify the importance of each of the 26 domains. Participants were asked to rate each domain on a scale of 0-10 (0 = not important, 10 = very important).
In this first round of the Delphi survey, 643 patients participated from the United States (n = 543), Sweden (n = 49), and South Korea (n = 51). Of the 26 domains, 19 (73%) were rated of high importance (≥ 7/10). The top 5 domains were muscle symptoms, fatigue, interactions with healthcare, medication side effects, and pain. During Outcome Measures in Rheumatology (OMERACT) 2016, we discussed the goal for ultimate reduction in the number of domains and the importance of considering representation of healthcare providers from other specialties, caregivers, representatives of pharmaceutical industries, and regulatory authorities in the next rounds of Delphi to represent broader perspectives on IIM.
Further prioritization and a reduction in the number of domains will be needed for the next Delphi. At the next biennial OMERACT meeting, we aim to present and seek voting on a Myositis Preliminary PRO Core Set to enable ultimate measure selection and development.
定义一组用于特发性炎性肌病(IIM)的核心患者报告领域和相应的工具。此前,我们报道了一项关于 IIM 患者报告结局(PRO)的系统文献综述,随后进行了国际焦点小组讨论,以了解患者对肌炎症状和影响的看法。
基于焦点小组的定性内容分析,构建了一个包含 26 个候选领域的初始清单。随后,我们进行了一项国际改良 Delphi 调查,以确定 26 个领域中每一个的重要性。参与者被要求在 0-10 的量表上对每个领域进行评分(0 = 不重要,10 = 非常重要)。
在 Delphi 调查的第一轮中,来自美国(n = 543)、瑞典(n = 49)和韩国(n = 51)的 643 名患者参与了调查。在 26 个领域中,有 19 个(73%)被评为高度重要(≥7/10)。排名前五的领域是肌肉症状、疲劳、与医疗保健的互动、药物副作用和疼痛。在 2016 年风湿病结局测量(OMERACT)会议期间,我们讨论了最终减少领域数量的目标,以及在未来的 Delphi 中考虑来自其他专业的医疗保健提供者、护理人员、制药行业代表和监管机构的代表的代表性,以代表更广泛的 IIM 视角。
在未来的 Delphi 中,还需要进一步优先考虑和减少领域数量。在未来的两年一次的 OMERACT 会议上,我们旨在提出并寻求对肌炎初步 PRO 核心集进行投票,以实现最终的测量选择和发展。