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

• Type of Material: Article
• Authors: Lisa G. Rider, NIH; Rohit Aggarwal, University of Pittsburgh; Angela Pistorio, Istituto Giannina Gaslini; Nastaran Bayat, NIH; Brian Erman, Social and Scientific Systems, Inc.; Brian M. Feldman, The Hospital for Sick Children; Adam M. Huber, IWK Health Centre; Rolando Cimaz, University of Florence; Ruben J. Cuttica, University of Buenos Aires; Sheila Knupp de Oliveira, Universidade Federal do Rio de Janeiro; Carol B. Lindsley, University of Kansas; Clarissa A. Pilkington, Great Ormond Street Hospital for Children NHS Trust; Marilyn Punaro, University of Texas Dallas; Angelo Ravelli, University of Genoa; Ann M. Reed, Duke University; Kelly A. Rouster Stevens, Emory University; Annet van Royen-Kerkhof, University Medical Centre Utrecht – Wilhelmina Children's Hospital; Frank Dressler, Hannover Medical School; Claudia Saad Magalhaes, Universidade Estadual Paulista; Tamas Constantin, Semmelweis University; Joyce E. Davidson, Royal Hospitals for Sick Children; Bo Magnusson, Karolinska University Hospital; Ricardo Russo, Hospital de Pediatría Garrahan; Luca Villa, Istituto Giannina Gaslini; Mariangela Rinaldi, Istituto Giannina Gaslini; Howard Rockette, University of Pittsburgh; Peter A. Lachenbruch, NIH; Frederick W. Miller, NIH; Jiri Vencovsky, Charles University Prague; Nicolino Ruperto, Istituto Giannina Gaslini
• Language: English
• Date: 2017-05-01
• Publisher: BMJ Publishing Group
• Publication Version: Author Accepted Manuscript (After Peer Review)
• Copyright Statement: © 2017, BMJ Publishing Group. All rights reserved.
• Final Published Version (URL): http://dx.doi.org/10.1136/annrheumdis-2017-211401
• Title of Journal or Parent Work: Annals of the Rheumatic Diseases
• ISSN: 0003-4967
• Volume: 76
• Issue: 5
• Start Page: 782
• End Page: 791
• Grant/Funding Information: This work was supported in part by the American College of Rheumatology, the European League Against Rheumatism, the Intramural Research Program of the National Institutes of Health, the National Institute of Environmental Health Sciences, the National Center for Advancing Translational Sciences, and the National Institute of Arthritis and Musculoskeletal and Skin Diseases; Istituto Giannina Gaslini Genova (Italy) and the Paediatric Rheumatology International Trials Organisation (PRINTO); Cure JM Foundation; Myositis UK; and The Myositis Association.
• Keywords: ADULT POLYMYOSITIS; IMPROVEMENT; Rheumatology; DISEASE-ACTIVITY SCORE; Life Sciences & Biomedicine; Science & Technology; THERAPY; VALIDATION; CONSENSUS; CONJOINT-ANALYSIS; PRELIMINARY DEFINITION; CORE SET; ARTHRITIS
• Research Categories: Health Sciences, General

Abstract

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.