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2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis
  1. Lisa G Rider1,
  2. Rohit Aggarwal2,
  3. Angela Pistorio3,
  4. Nastaran Bayat1,
  5. Brian Erman4,
  6. Brian M Feldman5,
  7. Adam M Huber6,
  8. Rolando Cimaz7,
  9. Rubén J Cuttica8,
  10. Sheila Knupp de Oliveira9,
  11. Carol B Lindsley10,
  12. Clarissa A Pilkington11,
  13. Marilynn Punaro12,
  14. Angelo Ravelli13,
  15. Ann M Reed14,
  16. Kelly Rouster-Stevens15,
  17. Annet van Royen-Kerkhof16,
  18. Frank Dressler17,
  19. Claudia Saad Magalhaes18,
  20. Tamás Constantin19,
  21. Joyce E Davidson20,21,
  22. Bo Magnusson22,
  23. Ricardo Russo23,
  24. Luca Villa24,
  25. Mariangela Rinaldi24,
  26. Howard Rockette2,
  27. Peter A Lachenbruch1,
  28. Frederick W Miller1,
  29. Jiri Vencovsky25,
  30. Nicolino Ruperto24
  31. for the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation
    1. 1NIEHS, NIH, Bethesda, Maryland, USA
    2. 2University of Pittsburgh, Pittsburgh, Pennsylvania, USA
    3. 3Istituto Giannina Gaslini, Servizio di Epidemiologia e Biostatistica, Genoa, Italy
    4. 4Social and Scientific Systems, Inc., Durham, North Carolina, USA
    5. 5The Hospital for Sick Children, Toronto, Ontario, Canada
    6. 6IWK Health Centre, Halifax, Nova Scotia, Canada
    7. 7University of Firenze, Florence, Italy
    8. 8Hospital de Niños Pedro de Elizalde, University of Buenos Aires, Buenos Aires, Argentina
    9. 9Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil
    10. 10University of Kansas City Medical Center, Kansas City, Kansas, USA
    11. 11Great Ormond Street Hospital for Children NHS Trust, London, UK
    12. 12University of Texas Southwestern Medical Center, Dallas, Texas, USA
    13. 13Istituto Giannina Gaslini, Pediatria II - Reumatologia, and Università degli Studi di Genova, Genoa, Italy
    14. 14Duke University, Durham, North Carolina, USA
    15. 15Emory University School of Medicine, Atlanta, Georgia, USA
    16. 16University Medical Centre Utrecht, Wilhelmina Children's Hospital, Utrecht, The Netherlands
    17. 17Hannover Medical School, Hannover, Germany
    18. 18Universidade Estadual Paulista Júlio de Mesquita Filho, Botucatu, Saõ Paulo, Brazil
    19. 19Semmelweis University, Budapest, Hungary
    20. 20Royal Hospital for Sick Children, Glasgow, UK
    21. 21Royal Hospital for Sick Children, Edinburgh, UK
    22. 22Karolinska University Hospital, Stockholm, Sweden
    23. 23Hospital de Pediatría Garrahan, Buenos Aires, Argentina
    24. 24Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO, Genoa, Italy
    25. 25Charles University, Prague, Czech Republic
    1. Correspondence to Dr Nicolino Ruperto, Istituto Giannina Gaslini, Pediatria II, PRINTO, Via G. Gaslini 5, Genoa 16147, Italy; nicolaruperto{at}gaslini.org

    An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative

    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.

    • Dermatomyositis
    • Polymyositis
    • Treatment

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    Footnotes

    • 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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