皮肌炎患者的体验及皮肌炎特异性患者报告结局指标的进一步验证——确立核心领域并扩大患者对皮肌炎临床评估的投入。OMERACT 12报告
Patients' Experience of Myositis and Further Validation of a Myositis-specific Patient Reported Outcome Measure - Establishing Core Domains and Expanding Patient Input on Clinical Assessment in Myositis. Report from OMERACT 12.
作者信息
Regardt Malin, Basharat Pari, Christopher-Stine Lisa, Sarver Catherine, Björn Anita, Lundberg Ingrid E, Wook Song Yeong, Bingham Clifton O, Alexanderson Helene
机构信息
From the Department of Occupational Therapy, Karolinska University Hospital; Department of Learning, Informatics and Medical Education, Karolinska Institutet, Stockholm, Sweden; Division of Rheumatology, Department of Medicine, Johns Hopkins University, Baltimore, Maryland, USA; Division of Rheumatology, Rheumatology Unit, Department of Medicine, Karolinska University Hospital in Solna, Stockholm, Sweden; Division of Rheumatology, Department of Internal Medicine, Medical Research Center, College of Medicine, Department of Molecular Medicine and Biopharmaceutical Sciences, Seoul National University, Seoul, Korea; Department of Care Science and Society, Division of Physiotherapy, Karolinska Institutet; and Department of Physical Therapy, Karolinska University Hospital, Stockholm, Sweden.M. Regardt, PhD, Occupational Therapist, Department of Occupational Therapy, Karolinska University Hospital, and Department of Learning, Informatics and Medical Education, Karolinska Institutet; P. Basharat, MD; L. Christopher-Stine, MD, Division of Rheumatology, Department of Medicine, Johns Hopkins University; C. Sarver, Patient Research Partner, Baltimore, Maryland, USA; A. Björn, Patient Research Partner, Stockholm, Sweden; I.E. Lundberg, MD, Division of Rheumatology, Rheumatology Unit, Department of Medicine, Karolinska University Hospital in Solna, Karolinska Institutet; Y. Wook Song, MD, Division of Rheumatology, Department of Internal Medicine, Medical Research Center, College of Medicine, Department of Molecular Medicine and Biopharmaceutical Sciences, Seoul National University; C.O. Bingham 3rd, MD, Division of Rheumatology, Department of Medicine, Johns Hopkins University; H. Alexanderson, Associate Professor, Physiotherapist, Department of Care Science and Society, Division of Physiotherapy, Karolinska Institutet, and Department of Physical Therapy, Karolinska University Hospital.
出版信息
J Rheumatol. 2015 Dec;42(12):2492-5. doi: 10.3899/jrheum.141243. Epub 2015 May 1.
OBJECTIVE
The Outcome Measures in Rheumatology (OMERACT) myositis working group was established to examine patient-reported outcomes (PRO) as well as to validate patient-reported outcome measures (PROM) in myositis.
METHODS
Qualitative studies using focus group interviews and cognitive debriefing of the myositis-specific Myositis Activities Profile (MAP) were used to explore the experience of adults living with polymyositis (PM) and dermatomyositis (DM).
RESULTS
Preliminary results underscore the importance of patient input in the development of PROM to ensure content validity. Results from multicenter focus groups indicate the range of symptoms experienced including pain, fatigue, and impaired cognitive function, which are not currently assessed in myositis. Preliminary cognitive debriefing of the MAP indicated that while content was deemed relevant and important, several activities were not included; and that questionnaire construction and wording may benefit from revision. A research agenda was developed to continue work toward optimizing PRO assessment in myositis with 2 work streams. The first would continue to conduct and analyze focus groups until saturation in the thematic analysis was achieved to develop a framework that encompassed the patient-relevant aspects of myositis. The second would continue cognitive debriefing of the MAP to identify potential areas for revision. There was agreement that further work would be needed for inclusion body myositis and juvenile dermatomyositis, and that the inclusion of additional contributors such as caregivers and individuals from the pharmaceutical/regulatory spheres would be desirable.
CONCLUSIONS
The currently used PROM do not assess symptoms or the effects of disease that are most important to patients; this emphasizes the necessity of patient involvement. Our work provides concrete examples for PRO identification.
目的
成立风湿病结局评估（OMERACT）肌炎工作组，以检查患者报告结局（PRO）并验证肌炎患者报告结局测量指标（PROM）。
方法
采用焦点小组访谈和对肌炎特异性肌炎活动概况（MAP）进行认知性详细询问的定性研究，以探索成年多发性肌炎（PM）和皮肌炎（DM）患者的经历。
结果
初步结果强调了患者参与PROM制定以确保内容效度的重要性。多中心焦点小组的结果表明，患者经历的症状范围包括疼痛、疲劳和认知功能受损，而这些目前在肌炎评估中未涉及。对MAP的初步认知性详细询问表明，虽然内容被认为相关且重要，但一些活动未被纳入；并且问卷的构建和措辞可能需要修订。制定了一项研究议程，通过两个工作流程继续致力于优化肌炎的PRO评估。第一个流程将继续开展并分析焦点小组，直到主题分析达到饱和，以制定一个涵盖肌炎患者相关方面的框架。第二个流程将继续对MAP进行认知性详细询问，以确定潜在的修订领域。大家一致认为，对于包涵体肌炎和青少年皮肌炎还需要进一步开展工作，并且纳入更多的参与者，如护理人员以及来自制药/监管领域的人员是可取的。
结论
目前使用的PROM未评估对患者最重要的症状或疾病影响；这强调了患者参与的必要性。我们的工作为PRO识别提供了具体实例。
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