Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies
Proposed preliminary core set measures for disease outcome assessment in adult and juvenile idiopathic inflammatory myopathies
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DOI：
10.1093/rheumatology/40.11.1262
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发表时间：
2001-11-01
期刊：
影响因子：
5.5
通讯作者：
Isenberg, DA
中科院分区：
文献类型：
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作者：
Miller, FW;Rider, LG;Isenberg, DA
为了开发一套用于特发性炎性肌病（IIM）临床试验的初步核心疾病结局指标，我们评估了以前试验中使用的指标，评估了已发表工具的有效性，并在国际共识会议上讨论了这些指标。最初的建议进一步完善了一个多学科小组的成人和儿科专家经验丰富的IIM使用德尔菲方法。拟议的疾病活动性指标初步核心集包括五个领域：医生和患者父母对疾病活动性的总体评估;肌肉力量：身体功能，肌肉酶的血清活性;以及用于捕获骨骼肌外疾病活动的评估工具。该小组建议进一步制定一套核心疾病损害指标，以评估至少持续6个月的解剖、病理和功能的持续变化。该小组建议，患者报告的结局应包括使用成人IIM患者的医学结局研究36项简表（SF-36）健康调查进行的一般健康相关生活质量评估，以及儿科患者经验证的生活质量工具。我们建议将核心结局指标集作为所有IIM治疗研究中的最小评估组。使用这一核心集应有助于标准化的结果测量和优化肌炎的治疗试验。
In order to develop a preliminary core set of disease outcome measures for use in clinical trials of idiopathic inflammatory myopathies (IIM), we evaluated those measures used in previous trials, assessed the validation of published instruments and discussed these at an international consensus conference. The initial proposals were further refined by a multidisciplinary group of adult and paediatric specialists experienced in IIM using the Delphi method. The proposed preliminary core set of disease activity measures consists of five domains: physician and patient parent global assessments of disease activity; muscle strength: physical function, serum activity of muscle enzymes; and an assessment tool to capture extra-skeletal muscle disease activity. The group recommended further development of a core set of disease damage measures for assessment of persistent changes in anatomy, pathology and function of at least 6 months' duration. The group recommended that patient-reported outcomes should include generic health-related quality of life assessments using the Medical Outcomes Study 36-item Short Form (SF-36) health survey in adult IIM patients and a validated quality of life instrument for paediatric patients. We propose the core set of outcome measures as a minimum group of assessments to include in all IIM therapeutic studies. The use of this core set should assist in standardizing outcome measurement and in optimizing therapeutic trials in myositis.