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.
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Handling editor Tore K Kvien
LGR and RA contributed equally and JV and NR contributed equally.
This article is published simultaneously in the May 2017 issue of Arthritis & Rheumatology.
Submitted for publication 11 February 2016; accepted in revised form 31 January 2017
Collaborators Appendix A MEMBERS OF THE INTERNATIONAL MYOSITIS ASSESSMENT AND CLINICAL STUDIES GROUP AND THE PAEDIATRIC RHEUMATOLOGY INTERNATIONAL TRIALS ORGANISATION WHO CONTRIBUTED TO DEVELOPING THE RESPONSE CRITERIA
Steering committee: Lisa G Rider (co-principal investigator), Nicolino Ruperto (co-principal investigator), Rohit Aggarwal (methodology lead), Frederick W Miller, Jiri Vencovsky.
Statistical team: Rohit Aggarwal, Brian Erman, Nastaran Bayat, Angela Pistorio, Adam M. Huber, Brian M Feldman, Paul Hansen, Howard Rockette, Peter A Lachenbruch, Nicolino Ruperto, Lisa G. Rider.
Paediatric core set survey group: Maria Apaz, Suzanne Bowyer, Rolando Cimaz, Tamás Constantin, Megan Curran, Joyce Davidson, Brian M. Feldman, Thomas Griffin, Adam H. Huber, Olcay Jones, Susan Kim, Bianca Lang, Carol Lindsley, Daniel Lovell, Claudia Saad Magalhaes, Lauren M. Pachman, Clarissa Pilkington, Andrea Ponyi, Marilynn Punaro, Pierre Quartier, Athimalaipet V Ramanan, Angelo Ravelli, Ann Reed, Robert Rennebohm, David D Sherry, Clovis A Silva, Elizabeth Stringer, Annet van Royen-Kerkhof, Carol Wallace.
Clinical trial or natural history study data set contributors: Frederick W. Miller, Chester V. Oddis, Ann Reed, Lisa G. Rider, Nicolino Ruperto, and PRINTO members.
Paediatric patient profile working group: Maria Apaz, Tadej Avcin, Mara Becker, Michael W. Beresford, Rolando Cimaz, Tamás Constantin, Megan Curran, Ruben Cuttica, Joyce Davidson, Frank Dressler, Jeffrey Dvergsten, Sheila Knupp Feitosa de Oliveira, Brian M. Feldman, Virginia Paes Leme Ferriani, Berit Flato, Valeria Gerloni, Thomas Griffin, Michael Henrickson, Claas Hinze, Mark Hoeltzel, Adam M. Huber, Maria Ibarra, Norman Ilowite, Lisa Imundo, Olcay Jones, Susan Kim, Daniel Kingsbury, Bianca Lang, Carol Lindsley, Daniel Lovell, Alberto Martini, Claudia Saad Magalhaes, Bo Magnusson, Sheilagh Maguiness, Susan Maillard, Pernille Mathiesen, Liza McCann, Susan Nielsen, Lauren M. Pachman, Murray Passo, Clarissa Pilkington, Marilynn Punaro, Pierre Quartier, Egla Rabinovich, Athimalaipet V. Ramanan, Angelo Ravelli, Ann Reed, Robert Rennebohm, Lisa G. Rider, Rafael Rivas-Chacon, Angela Byun Robinson, Kelly Rouster-Stevens, Ricardo Russo, Lidia Rutkowska-Sak, Adriana Sallum, Helga Sanner, Heinrike Schmeling, Duygu Selcen, Bracha Shaham, David D. Sherry, Clovis A. Silva, Charles H. Spencer, Robert Sundel, Marc Tardieu, Akaluck Thatayatikom, Janjaap van der Net, Annet van Royen-Kerkhof, Dawn Wahezi, Carol Wallace, Francesco Zulian.
Conjoint analysis, paediatric group: Rolando Cimaz, Tamás Constantin, Ruben Cuttica, Joyce Davidson, Frank Dressler, Brian M. Feldman, Thomas Griffin, Michael Henrickson, Adam M. Huber, Lisa Imundo, Bianca Lang, Carol Lindsley, Claudia Saad Magalhaes, Bo Magnusson, Susan Maillard, Lauren M. Pachman, Murray Passo, Clarissa Pilkington, Marilynn Punaro, Angelo Ravelli, Ann Reed, Lisa G. Rider, Kelly Rouster-Stevens, Ricardo Russo, Bracha Shaham, Robert Sundel, Janjaap van der Net, Annet van Royen-Kerkhof.
Participants in consensus conference, paediatric working group: Rolando Cimaz, Rubén Cuttica, Sheila Knupp Feitosa de Oliveira, Brian M. Feldman, Adam M. Huber, Carol B. Lindsley, Clarissa Pilkington, Marilynn Punaro, Angelo Ravelli, Ann Reed, Kelly Rouster-Stevens, Annet van Royen-Kerkhof.
Participants in consensus conference, adult working group: Anthony Amato, Hector Chinoy, Robert G Cooper, Maryam Dastmalchi, Marianne de Visser, David Fiorentino, David Isenberg, James Katz, Andrew Mammen, Chester V. Oddis, Jiri Vencovsky, Steven R Ytterberg.
See Appendix A for members of the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation who contributed to developing the response criteria.
Contributors All authors were involved in drafting the article or revising it critically for important intellectual content, and all authors approved the final version to be published. LGR and NR had full access to all of the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. Study conception and design: LGR, RA, FWM, JV, NR. Acquisition of data: LGR, RA, AP, NB, BE, BMF, AMH, RC, RJC, SKO, CBL, CAP, MP, AR, AMR, KR, AR, FD, CS, TC, JED, BM, RR, LV, MR, HR, PAL, JV, NR. Analysis and interpretation of data: LGR, RA, CAP, NB, BE, BMF, AMH, HR, PAL, FWM, JV, NR.
Competing interests None declared.
Provenance and peer review Not commissioned; internally peer reviewed.
Supported in part by the American College of Rheumatology, the European League Against Rheumatism, the NIH (Intramural Research Programs of the National Institute of Environmental Health Sciences (NIEHS), National Center for Advancing Translational Sciences, and National Institute of Arthritis and Musculoskeletal and Skin Diseases), Istituto G. Gaslini and the Paediatric Rheumatology International Trials Organisation (PRINTO), Cure JM Foundation, Myositis UK, and the Myositis Association. Dr. Vencovsky's work was supported by the Ministry of Health, Czech Republic (Institute of Rheumatology project for conceptual development of a research organisation, 00023728).
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