2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative.
2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis: An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative.
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DOI：
10.1002/art.40064
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发表时间：
2017-05
期刊：
影响因子：
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通讯作者：
International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation
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作者：
Aggarwal R;Rider LG;Ruperto N;Bayat N;Erman B;Feldman BM;Oddis CV;Amato AA;Chinoy H;Cooper RG;Dastmalchi M;Fiorentino D;Isenberg D;Katz JD;Mammen A;de Visser M;Ytterberg SR;Lundberg IE;Chung L;Danko K;García-De la Torre I;Song YW;Villa L;Rinaldi M;Rockette H;Lachenbruch PA;Miller FW;Vencovsky J;International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation
制定幼年型皮肌炎（JDM）的反应标准。我们分析了312个定义的性能，这些定义使用来自国际肌炎评估和临床研究组（IMACS）或儿科流变学国际试验组织（PRINTO）的核心集测量（CSM），并来自自然病史数据和联合分析调查。在PRINTO试验中，单独使用泼尼松与泼尼松联合甲氨蝶呤或环孢霉素以及利妥昔单抗治疗肌炎的试验进行了进一步验证。专家们在一次共识会议上考虑了14个表现最好的候选标准，这些标准基于其性能特征和临床表面效度，使用名义组技术。对于基于联合分析的连续模型达成共识，总改善评分为0-100，使用CSM的绝对百分比变化，阈值为极轻微（≥30分）、中度（≥45）和重大改善（≥70）。对于具有不同改善阈值的成人皮肌炎/多发性肌炎选择相同的标准。在使用IMACS和PRINTO CSM的JDM患者队列中，极轻微改善的灵敏度和特异性分别为89%和91-98%，中度改善的灵敏度和特异性分别为92-94%和94-99%，重大改善的灵敏度和特异性分别为91-98%和85-85%。这些标准在PRINTO试验中得到了验证，用于区分治疗组的轻微和中度改善（P=0.009-0.057），并在利妥昔单抗试验中得到了验证，用于显著区分医生的改善评级（P<0.006）。JDM的缓解标准是一种基于联合分析的模型，使用基于CSM绝对百分比变化的持续改善评分，阈值为极轻微、中度和重大改善。
Develop response criteria for juvenile dermatomyositis (JDM). We analyzed the performance of 312 definitions that used core set measures (CSM) from either the International Myositis Assessment and Clinical Studies Group (IMACS) or the Pediatric Rheumatology International Trials Organization (PRINTO) and were derived from natural history data and a conjoint-analysis survey. They were further validated in the PRINTO trial of prednisone alone compared to prednisone with methotrexate or cyclosporine and the Rituximab in Myositis trial. Experts considered 14 top-performing candidate criteria based on their performance characteristics and clinical face validity using nominal group technique at a consensus conference. Consensus was reached for a conjoint analysis–based continuous model with a Total Improvement Score of 0-100, using absolute percent change in CSM with thresholds for minimal (≥30 points), moderate (≥45), and major improvement (≥70). The same criteria were chosen for adult dermatomyositis/polymyositis with differing thresholds for improvement. The sensitivity and specificity were 89% and 91-98% for minimal, 92-94% and 94-99% for moderate, and 91-98% and 85-85% for major improvement, respectively, in JDM patient cohorts using the IMACS and PRINTO CSM. 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 Rituximab trial for significantly differentiating the physician rating of improvement (P<0.006). The response criteria for JDM was a conjoint analysis–based model using a continuous improvement score based on absolute percent change in CSM, with thresholds for minimal, moderate, and major improvement.