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.

3rd Department of Internal Medicine Division of Immunology University of Debrecen Debrecen Hungary

Department of Biostatistics University of Pittsburgh Pittsburgh PA USA

Department of Dermatology Stanford University Redwood City CA USA

Department of Economics University of Otago Dunedin New Zealand

Department of Medicine Division of Rheumatology and Clinical Immunology University of Pittsburgh Pittsburgh PA USA

Department of Molecular Medicine and Biopharmaceutical Sciences Medical Research Center Seoul National University Seoul Korea

Department of Neurology Brigham and Women's Hospital and Harvard Medical School Boston MA USA

Department of Paediatric Rheumatology Great Ormond Street Hospital for Children NHS Trust London UK

Department of Pediatric Rheumatology Hospital de Niños Pedro de Elizalde University of Buenos Aires Buenos Aires Argentina

Department of Pediatrics Duke University Durham NC USA

Division of Rheumatology Hospital for Sick Children Toronto Ontario

Division of Rheumatology Stanford University Redwood City CA USA

Environmental Autoimmunity Group NIEHS National Institutes of Health Bethesda MD USA

Hospital General de Occidente de la Secretaría de Salud Guadalajara Jalisco Mexico

Institute of Ageing and Chronic Disease University of Liverpool Liverpool UK

Institute of Rheumatology and Department of Rheumatology Charles University Prague Czech Republic

Istituto Giannina Gaslini Pediatria 2 Reumatologia PRINTO

Istituto Giannina Gaslini Servizio di Epidemiologia e Biostatistica Genoa Italy

Manchester Academic Health Science Centre The University of Manchester Manchester

National Institute of Health Research Manchester Musculoskeletal Biomedical Research Unit Central Manchester University Hospitals NHS Foundation Trust

Pediatric Rheumatology Azienda Ospedaliero Universitaria Meyer University of Florence Florence Italy

Rheumatology Department Izaak Walton Killam Health Centre Halifax Nova Scotia Canada

Rheumatology Unit Karolinska University Hospital Karolinska Institute Stockholm Sweden

Social and Scientific Systems Inc Durham NC

Università degli Studi di Genova Dipartimento di Pediatria Genoa Italy

Wilhelmina Children's Hospital University Medical Center Utrecht The Netherlands

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