To develop response criteria for adult dermatomyositis (DM) and polymyositis (PM). 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. Consensus was reached for a conjoint analysis-based continuous model using absolute per cent 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). The response criteria for adult DM/PM consisted of the conjoint analysis model based on absolute per cent change in 6 core set measures, with thresholds for minimal, moderate, and major improvement.
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Handling editor Tore K Kvien
See Appendix A for members of the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation who contriuted to developing the response criteria.
RA and LGR contributed equally and FWM and JV contributed equally.
This article is published simultaneously in the May 2017 issue of Arthritis & Rheumatology.
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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. Adult core set survey group: Anthony A Amato, Hector Chinoy, Lisa Christopher-Stine, Lorinda Chung, Robert G Cooper, Lisa Criscione-Schreiber, Leslie Crofford, Mary E Cronin, Katalin Dankó, David Fiorentino, Ignacio García-De la Torre, Patrick Gordon, Gerald Hengstman, James D Katz, Andrew Mammen, Galina Marder, Neil McHugh, Chester V Oddis, Elena Schiopu, Albert Selva-O'Callaghan, Yeong Wook Song, Jiri Vencovsky, Gil Wolfe, Robert Wortmann. Clinical trial or natural history study data set contributions: Anthony A Amato, Hector Chinoy, Lorinda Chung, Robert G Cooper, Katalin Dankó, David Fiorentino, Ignacio García-De la Torre, Mark Gourley, Ingrid Lundberg, Frederick W Miller, Chester V Oddis, Paul Plotz, Lisa G Rider, Yeong Wook Song, Jiri Vencovsky. Adult patient profile working group: Rohit Aggarwal, Anthony A Amato, Dana Ascherman, Richard Barohn, Olivier Benveniste, Jan De Bleecker, Jeffrey Callen, Christina Charles-Schoeman, Hector Chinoy, Lisa Christopher-Stine, Lorinda Chung, Robert G Cooper, Leslie Crofford, Mary E Cronin, Katalin Dankó, Sonye Danoff, Maryam Dastmalchi, Marianne de Visser, Mazen Dimachkie, Steve DiMartino, Lyubomir Dourmishev, Floranne Ernste, David Fiorentino, Ignacio García-De la Torre, Takahisa Gono, Patrick Gordon, Mark Gourley, David Isenberg, Yasuhiro Katsumata, James D Katz, John Kissel, Richard L Leff, Todd Levine, Ingrid Lundberg, Andrew Mammen, Herman Mann, Galina Marder, Isabelle Marie, Neil McHugh, Joseph Merola, Frederick W Miller, Chester V Oddis, Marzena Olesinska, Nancy Olsen, Nicolo Pipitone, Sindhu Ramchandren, Seward Rutkove, Lesley Ann Saketkoo, Adam Schiffenbauer, Albert Selva-O'Callaghan, Samuel Katsuyuki Shinjo, Rachel Shupak, Yeong Wook Song, Katarzyna Swierkocka, Jiri Vencovsky, Julia Wanschitz, Victoria Werth, Irene Whitt, Robert Wortmann, Steven R Ytterberg. Conjoint analysis, adult group: Rohit Aggarwal, Anthony A Amato, Hector Chinoy, Lisa Christopher-Stine, Lorinda Chung, Robert G Cooper, Mary E Cronin, Katalin Dankó, Mazen Dimachkie, Steve DiMartino, David Fiorentino, Ignacio García-De la Torre, Patrick Gordon, Ingrid Lundberg, Herman Mann, Frederick W Miller, Chester V Oddis, Albert Selva-O'Callaghan, Jiri Vencovsky, Victoria Werth, Robert Wortmann, Steven R Ytterberg. Participants in consensus conference, adult working group: Anthony A Amato, Hector Chinoy, Robert G Cooper, Maryam Dastmalchi, Marianne de Visser, David Fiorentino, David Isenberg, James D Katz, Andrew Mammen, Chester V Oddis, Jiri Vencovsky, Steven R Ytterberg. 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.
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. Drs Aggarwal and Rider 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. All authors: Study conception and design; Acquisition of data; and Analysis and interpretation of data.
Funding Supported in part by the American College of Rheumatology, the European League Against Rheumatism, Cure JM Foundation, Myositis UK, Istituto G. Gaslini and the Paediatric Rheumatology International Trials Organisation (PRINTO), the Myositis Association, and the NIH (National Institute of Environmental Health Sciences (NIEHS), National Center for Advancing Translational Sciences, and National Institute of Arthritis and Musculoskeletal and Skin Diseases). IG-DlT's work was supported in part by CONACYT (Programa Nacional de Posgrados de Calidad). YWS's work was supported by the Korea Health Technology R & D Project through the Korea Health Industry Development Institute funded by the Ministry of Health & Welfare, Republic of Korea (grant HI14C1277). JV's work was supported by the Ministry of Health, Czech Republic (Institute of Rheumatology project for conceptual development of a research organisation, 00023728).
Competing interests None declared.
Provenance and peer review Not commissioned; internally peer reviewed.
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