2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects
2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects
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DOI：
10.1093/rheumatology/kex226
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发表时间：
2017-11-01
期刊：
影响因子：
5.5
通讯作者：
Aggarwal, Rohit
中科院分区：
文献类型：
--
作者：
Rider, Lisa G.;Ruperto, Nicolino;Aggarwal, Rohit
客观的。目的是描述用于制定成人 DM/PM 和 JDM 新反应标准的方法。方法。肌炎专家对来自前瞻性自然史数据和临床试验的患者概况进行评级，以制定最小、中度和重大改善的共识金标准评级。专家们完成了一项关于核心集测量 (CSM) 的临床有意义的改进的调查和一项联合分析调查（使用 1000Minds 软件），以得出 CSM 和候选定义的相对权重。使用调查结果、逻辑回归、联合分析、将联合分析权重应用于 CSM 和已发布的定义，得出了响应标准的六种候选定义。使用一致的患者概况数据定义候选标准的敏感性、特异性和曲线下面积，并使用临床试验数据验证选定的定义。结果。肌炎专家将 CSM 有临床意义的改善程度定义为最小、中度和重大改善。联合分析调查确定了 CSM 的相对权重，其中肌肉力量和医生整体活动最为重要。许多候选定义在共识概况中表现出出色的敏感性、特异性和曲线下面积。试验验证表明，许多候选标准在治疗组之间存在差异。共识会议上提出了顶级候选标准定义。结论。共识方法通过对患者资料进行测试并通过临床试验验证定义，得出了 18 个成人 PM/DM 定义和 14 个 JDM 定义，作为肌炎新反应标准最终共识的优秀候选者。
Objective. The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM.Methods. Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data.Results. Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference.Conclusion. Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.