OBJECTIVE: To develop response criteria for adult dermatomyositis (DM) and polymyositis (PM). METHODS: Expert surveys, logistic regression, and conjoint analysis were used to develop 287 definitions using core set measures. Myositis experts rated greater improvement among multiple pairwise scenarios in conjoint analysis surveys, where different levels of improvement in 2 core set measures were presented. The PAPRIKA (Potentially All Pairwise Rankings of All Possible Alternatives) method determined the relative weights of core set measures and conjoint analysis definitions. The performance characteristics of the definitions were evaluated on patient profiles using expert consensus (gold standard) and were validated using data from a clinical trial. The nominal group technique was used to reach consensus. RESULTS: Consensus was reached for a conjoint analysis-based continuous model using absolute percent change in core set measures (physician, patient, and extramuscular global activity, muscle strength, Health Assessment Questionnaire, and muscle enzyme levels). A total improvement score (range 0-100), determined by summing scores for each core set measure, was based on improvement in and relative weight of each core set measure. Thresholds for minimal, moderate, and major improvement were ≥20, ≥40, and ≥60 points in the total improvement score. The same criteria were chosen for juvenile DM, with different improvement thresholds. Sensitivity and specificity in DM/PM patient cohorts were 85% and 92%, 90% and 96%, and 92% and 98% for minimal, moderate, and major improvement, respectively. Definitions were validated in the clinical trial analysis for differentiating the physician rating of improvement (P < 0.001). CONCLUSION: The response criteria for adult DM/PM consisted of the conjoint analysis model based on absolute percent change in 6 core set measures, with thresholds for minimal, moderate, and major improvement.

Academic Medical Center Amsterdam The Netherlands

Brigham and Women's Hospital and Harvard Medical School Boston Massachusetts

Central Manchester University Hospitals NHS Foundation Trust University of Manchester Manchester UK

Charles University Prague Czech Republic

Graduate School of Convergence Science and Technology and Seoul National University Hospital Seoul Korea

Hospital General de Occidente de la Secretaría de Salud and University of Guadalajara Guadalajara México

Istituto Giannina Gaslini Pediatria 2 Reumatologia PRINTO Genoa Italy

Johns Hopkins University School of Medicine Baltimore Maryland

Karolinska University Hospital Karolinska Institute Stockholm Sweden

Mayo Clinic Rochester Minnesota

NIH Bethesda Maryland

Social and Scientific Systems Inc Durham North Carolina

Stanford University Redwood City California

The Hospital for Sick Children Toronto Ontario Canada

University College London London UK

University of Debrecen Debrecen Hungary

University of Liverpool Liverpool UK

University of Pittsburgh Pittsburgh Pennsylvania

Arthritis Rheumatol. 2017 May;69(5):878-884. doi: 10.1002/art.40063. PubMed

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