2016年美国风湿病学会/欧洲抗风湿病联盟儿童皮肌炎最小、中度和主要临床反应标准：一项国际肌炎评估与临床研究组/儿科风湿病国际试验组织合作倡议。
2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative.
作者信息
Rider Lisa G, Aggarwal Rohit, Pistorio Angela, Bayat Nastaran, Erman Brian, Feldman Brian M, Huber Adam M, Cimaz Rolando, Cuttica Rubén J, de Oliveira Sheila Knupp, Lindsley Carol B, Pilkington Clarissa A, Punaro Marilynn, Ravelli Angelo, Reed Ann M, Rouster-Stevens Kelly, van Royen-Kerkhof Annet, Dressler Frank, Saad Magalhaes Claudia, Constantin Tamás, Davidson Joyce E, Magnusson Bo, Russo Ricardo, Villa Luca, Rinaldi Mariangela, Rockette Howard, Lachenbruch Peter A, Miller Frederick W, Vencovsky Jiri, Ruperto Nicolino
机构信息
NIEHS, NIH, Bethesda, Maryland, USA.
University of Pittsburgh, Pittsburgh, Pennsylvania, USA.
出版信息
Ann Rheum Dis. 2017 May;76(5):782-791. doi: 10.1136/annrheumdis-2017-211401.
To develop response criteria for juvenile dermatomyositis (DM). We analysed the performance of 312 definitions that used core set measures from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Paediatric Rheumatology International Trials Organisation (PRINTO) and were derived from natural history data and a conjoint analysis survey. They were further validated using data from the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis (RIM) trial. At a consensus conference, experts considered 14 top candidate criteria based on their performance characteristics and clinical face validity, using nominal group technique. Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute per cent change in core set measures of minimal (≥30), moderate (≥45), and major (≥70) improvement. The same criteria were chosen for adult DM/polymyositis, with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal improvement, 92-94% and 94-99% for moderate improvement, and 91-98% and 85-86% for major improvement, respectively, in juvenile DM patient cohorts using the IMACS and PRINTO core set measures. These criteria were validated in the PRINTO trial for differentiating between treatment arms for minimal and moderate improvement (p=0.009-0.057) and in the RIM trial for significantly differentiating the physician's rating for improvement (p<0.006). The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute per cent change in core set measures, with thresholds for minimal, moderate, and major improvement.
制定青少年皮肌炎（DM）的反应标准。我们分析了312种定义的性能，这些定义使用了来自国际肌炎评估与临床研究组（IMACS）或儿科风湿病国际试验组织（PRINTO）的核心指标集，并且源自自然史数据和联合分析调查。使用来自单独使用泼尼松与泼尼松联合甲氨蝶呤或环孢素的PRINTO试验以及肌炎利妥昔单抗（RIM）试验的数据对它们进行了进一步验证。在一次共识会议上，专家们使用名义群体技术，根据其性能特征和临床表面效度，审议了14项顶级候选标准。对于基于联合分析的连续模型达成了共识，该模型的总改善评分为0至100分，使用核心指标集最小（≥30）、中度（≥45）和显著（≥70）改善的绝对百分比变化。成人DM/多肌炎选择了相同的标准，但改善阈值不同。在使用IMACS和PRINTO核心指标集的青少年DM患者队列中，最小改善的敏感性和特异性分别为89%和91 - 98%，中度改善为92 - 94%和94 - 99%，显著改善为91 - 98%和85 - 86%。这些标准在PRINTO试验中得到验证，可用于区分最小和中度改善的治疗组（p = 0.009 - 0.057），在RIM试验中可显著区分医生对改善的评分（p < 0.006）。青少年DM的反应标准包括一个基于联合分析的模型，该模型使用基于核心指标集绝对百分比变化的连续改善评分，以及最小、中度和显著改善的阈值。
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