Lisa G Rider1, Rohit Aggarwal2, Angela Pistorio3, Nastaran Bayat1, Brian Erman4, Brian M Feldman5, Adam M Huber6, Rolando Cimaz7, Rubén J Cuttica8, Sheila Knupp de Oliveira9, Carol B Lindsley10, Clarissa A Pilkington11, Marilynn Punaro12, Angelo Ravelli13, Ann M Reed14, Kelly Rouster-Stevens15, Annet van Royen-Kerkhof16, Frank Dressler17, Claudia Saad Magalhaes18, Tamás Constantin19, Joyce E Davidson20, Bo Magnusson21, Ricardo Russo22, Luca Villa23, Mariangela Rinaldi23, Howard Rockette2, Peter A Lachenbruch1, Frederick W Miller1, Jiri Vencovsky24, Nicolino Ruperto23. 1. NIH, Bethesda, Maryland. 2. University of Pittsburgh, Pittsburgh, Pennsylvania. 3. Istituto Giannina Gaslini, Servizio di Epidemiologia e Biostatistica, Genoa, Italy. 4. Social and Scientific Systems, Inc., Durham, North Carolina. 5. The Hospital for Sick Children, Toronto, Ontario, Canada. 6. IWK Health Centre, Halifax, Nova Scotia, Canada. 7. University of Firenze, Florence, Italy. 8. Hospital de Niños Pedro de Elizalde, University of Buenos Aires, Buenos Aires, Argentina. 9. Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil. 10. University of Kansas City Medical Center, Kansas City, Kansas. 11. Great Ormond Street Hospital for Children NHS Trust, London, UK. 12. University of Texas Southwestern Medical Center, Dallas. 13. Istituto Giannina Gaslini, Pediatria II - Reumatologia, and Università degli Studi di Genova, Genoa, Italy. 14. Duke University, Durham, North Carolina. 15. Emory University School of Medicine, Atlanta, Georgia. 16. University Medical Centre Utrecht, Wilhelmina Children's Hospital, Utrecht, The Netherlands. 17. Hannover Medical School, Hannover, Germany. 18. Universidade Estadual Paulista Júlio de Mesquita Filho, Botucatu, Saõ Paulo, Brazil. 19. Semmelweis University, Budapest, Hungary. 20. Royal Hospital for Sick Children, Glasgow, UK, and Royal Hospital for Sick Children, Edinburgh, UK. 21. Karolinska University Hospital, Stockholm, Sweden. 22. Hospital de Pediatría Garrahan, Buenos Aires, Argentina. 23. Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO, Genoa, Italy. 24. Charles University, Prague, Czech Republic.
Abstract
OBJECTIVE: To develop response criteria for juvenile dermatomyositis (DM). METHODS: We analyzed 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. RESULTS: Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute percent 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). CONCLUSION: The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute percent change in core set measures, with thresholds for minimal, moderate, and major improvement.
OBJECTIVE: To develop response criteria for juvenile dermatomyositis (DM). METHODS: We analyzed 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. RESULTS: Consensus was reached for a conjoint analysis-based continuous model with a total improvement score of 0-100, using absolute percent 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). CONCLUSION: The response criteria for juvenile DM consisted of a conjoint analysis-based model using a continuous improvement score based on absolute percent change in core set measures, with thresholds for minimal, moderate, and major improvement.
Authors: F W Miller; L G Rider; Y L Chung; R Cooper; K Danko; V Farewell; I Lundberg; C Morrison; L Oakley; I Oakley; C Pilkington; J Vencovsky; K Vincent; D L Scott; D A Isenberg Journal: Rheumatology (Oxford) Date: 2001-11 Impact factor: 7.580
Authors: Lisa G Rider; Edward H Giannini; Michael Harris-Love; Galen Joe; David Isenberg; Clarissa Pilkington; Peter A Lachenbruch; Frederick W Miller Journal: J Rheumatol Date: 2003-03 Impact factor: 4.666
Authors: Nicolino Ruperto; Angelo Ravelli; Kevin J Murray; Daniel J Lovell; Boel Andersson-Gare; Brian M Feldman; Stella Garay; Wietse Kuis; Claudia Machado; Lauren Pachman; Anne-Marie Prieur; Lisa G Rider; Earl Silverman; Elena Tsitsami; Pat Woo; Edward H Giannini; Alberto Martini Journal: Rheumatology (Oxford) Date: 2003-06-27 Impact factor: 7.580
Authors: Lisa G Rider; Edward H Giannini; Hermine I Brunner; Nicola Ruperto; Laura James-Newton; Ann M Reed; Peter A Lachenbruch; Frederick W Miller Journal: Arthritis Rheum Date: 2004-07
Authors: Nicolino Ruperto; Angelo Ravelli; Angela Pistorio; Virginia Ferriani; Immaculada Calvo; Gerd Ganser; Jurgen Brunner; Guenther Dannecker; Clovis Arthur Silva; Valda Stanevicha; Rebecca Ten Cate; Lisette W A van Suijlekom-Smit; Olga Voygioyka; Michel Fischbach; Ivan Foeldvari; Odete Hilario; Consuelo Modesto; Rotraud K Saurenmann; Marie-Josephe Sauvain; Iloite Scheibel; Danièle Sommelet; Lana Tambic-Bukovac; Roberto Barcellona; Riva Brik; Stephan Ehl; Mirjana Jovanovic; Jozef Rovensky; Francesca Bagnasco; Daniel J Lovell; Alberto Martini Journal: Arthritis Rheum Date: 2008-01-15
Authors: Lisa G Rider; Rohit Aggarwal; Pedro M Machado; Jean-Yves Hogrel; Ann M Reed; Lisa Christopher-Stine; Nicolino Ruperto Journal: Nat Rev Rheumatol Date: 2018-04-12 Impact factor: 20.543
Authors: Sara Sabbagh; Adriana Almeida de Jesus; SuJin Hwang; Hye Sun Kuehn; Hanna Kim; Lawrence Jung; Ruy Carrasco; Sergio Rosenzweig; Raphaela Goldbach-Mansky; Lisa G Rider Journal: Brain Date: 2019-11-01 Impact factor: 13.501
Authors: Rohit Aggarwal; Lisa G Rider; Nicolino Ruperto; Nastaran Bayat; Brian Erman; Brian M Feldman; Chester V Oddis; Anthony A Amato; Hector Chinoy; Robert G Cooper; Maryam Dastmalchi; David Fiorentino; David Isenberg; James D Katz; Andrew Mammen; Marianne de Visser; Steven R Ytterberg; Ingrid E Lundberg; Lorinda Chung; Katalin Danko; Ignacio García-De la Torre; Yeong Wook Song; Luca Villa; Mariangela Rinaldi; Howard Rockette; Peter A Lachenbruch; Frederick W Miller; Jiri Vencovsky Journal: Ann Rheum Dis Date: 2017-05 Impact factor: 19.103
Authors: Ruchi N Patel; Valeria G Esparza; Jin-Shei Lai; Elizabeth L Gray; Bryce B Reeve; Rowland W Chang; David Cella; Kaveh Ardalan Journal: Arthritis Care Res (Hoboken) Date: 2021-07-30 Impact factor: 5.178