Lisa G Rider1, Nicolino Ruperto2, Angela Pistorio3, Brian Erman4, Nastaran Bayat1, Peter A Lachenbruch1, Howard Rockette5, Brian M Feldman6, Adam M Huber7, Paul Hansen8, Chester V Oddis9, Ingrid E Lundberg10, Anthony A Amato11, Hector Chinoy12,13, Robert G Cooper14, Lorinda Chung15, Katalin Danko16, David Fiorentino17, Ignacio García-De la Torre18, Ann M Reed19, Yeong Wook Song20, Rolando Cimaz21, Rubén J Cuttica22, Clarissa A Pilkington23, Alberto Martini2,24, Janjaap van der Net25, Susan Maillard23, Frederick W Miller1, Jiri Vencovsky26, Rohit Aggarwal9. 1. Environmental Autoimmunity Group, NIEHS, National Institutes of Health, Bethesda, MD, USA. 2. Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO. 3. Istituto Giannina Gaslini, Servizio di Epidemiologia e Biostatistica, Genoa, Italy. 4. Social and Scientific Systems, Inc., Durham, NC. 5. Department of Biostatistics, University of Pittsburgh, Pittsburgh, PA, USA. 6. Division of Rheumatology, Hospital for Sick Children, Toronto, Ontario. 7. Rheumatology Department, Izaak Walton Killam Health Centre, Halifax, Nova Scotia, Canada. 8. Department of Economics, University of Otago, Dunedin, New Zealand. 9. Department of Medicine, Division of Rheumatology and Clinical Immunology, University of Pittsburgh, Pittsburgh, PA, USA. 10. Rheumatology Unit, Karolinska University Hospital, Karolinska Institute, Stockholm, Sweden. 11. Department of Neurology, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA. 12. National Institute of Health Research Manchester Musculoskeletal Biomedical Research Unit, Central Manchester University Hospitals NHS Foundation Trust. 13. Manchester Academic Health Science Centre, The University of Manchester, Manchester. 14. Institute of Ageing and Chronic Disease, University of Liverpool, Liverpool, UK. 15. Division of Rheumatology, Stanford University, Redwood City, CA, USA. 16. 3rd Department of Internal Medicine, Division of Immunology, University of Debrecen, Debrecen, Hungary. 17. Department of Dermatology, Stanford University, Redwood City, CA, USA. 18. Hospital General de Occidente de la Secretaría de Salud, Guadalajara, Jalisco, Mexico. 19. Department of Pediatrics, Duke University, Durham, NC, USA. 20. Department of Molecular Medicine and Biopharmaceutical Sciences, Medical Research Center, Seoul National University, Seoul, Korea. 21. Pediatric Rheumatology, Azienda Ospedaliero Universitaria Meyer, University of Florence, Florence, Italy. 22. Department of Pediatric Rheumatology, Hospital de Niños Pedro de Elizalde, University of Buenos Aires, Buenos Aires, Argentina. 23. Department of Paediatric Rheumatology, Great Ormond Street Hospital for Children NHS Trust, London, UK. 24. Università degli Studi di Genova, Dipartimento di Pediatria, Genoa, Italy. 25. Wilhelmina Children's Hospital, University Medical Center, Utrecht, The Netherlands. 26. Institute of Rheumatology and Department of Rheumatology, Charles University, Prague, Czech Republic.
Abstract
Objective: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods: Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results: Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion: Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.
Objective: The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods:Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results:Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion: Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.
Authors: Tuhina Neogi; Daniel Aletaha; Alan J Silman; Raymond L Naden; David T Felson; Rohit Aggarwal; Clifton O Bingham; Neal S Birnbaum; Gerd R Burmester; Vivian P Bykerk; Marc D Cohen; Bernard Combe; Karen H Costenbader; Maxime Dougados; Paul Emery; Gianfranco Ferraccioli; Johanna M W Hazes; Kathryn Hobbs; Tom W J Huizinga; Arthur Kavanaugh; Jonathan Kay; Dinesh Khanna; Tore K Kvien; Timothy Laing; Katherine Liao; Philip Mease; Henri A Ménard; Larry W Moreland; Raj Nair; Theodore Pincus; Sarah Ringold; Josef S Smolen; Ewa Stanislawska-Biernat; Deborah Symmons; Paul P Tak; Katherine S Upchurch; Jirí Vencovský; Frederick Wolfe; Gillian Hawker Journal: Arthritis Rheum Date: 2010-09
Authors: Frank van den Hoogen; Dinesh Khanna; Jaap Fransen; Sindhu R Johnson; Murray Baron; Alan Tyndall; Marco Matucci-Cerinic; Raymond P Naden; Thomas A Medsger; Patricia E Carreira; Gabriela Riemekasten; Philip J Clements; Christopher P Denton; Oliver Distler; Yannick Allanore; Daniel E Furst; Armando Gabrielli; Maureen D Mayes; Jacob M van Laar; James R Seibold; Laszlo Czirjak; Virginia D Steen; Murat Inanc; Otylia Kowal-Bielecka; Ulf Müller-Ladner; Gabriele Valentini; Douglas J Veale; Madelon C Vonk; Ulrich A Walker; Lorinda Chung; David H Collier; Mary Ellen Csuka; Barri J Fessler; Serena Guiducci; Ariane Herrick; Vivien M Hsu; Sergio Jimenez; Bashar Kahaleh; Peter A Merkel; Stanislav Sierakowski; Richard M Silver; Robert W Simms; John Varga; Janet E Pope Journal: Arthritis Rheum Date: 2013-10-03
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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; 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: Lisa G Rider; Rohit Aggarwal; Angela Pistorio; Nastaran Bayat; Brian Erman; Brian M Feldman; Adam M Huber; Rolando Cimaz; Rubén J Cuttica; Sheila Knupp de Oliveira; Carol B Lindsley; Clarissa A Pilkington; Marilynn Punaro; Angelo Ravelli; Ann M Reed; Kelly Rouster-Stevens; Annet van Royen-Kerkhof; Frank Dressler; Claudia Saad Magalhaes; Tamás Constantin; Joyce E Davidson; Bo Magnusson; Ricardo Russo; Luca Villa; Mariangela Rinaldi; Howard Rockette; Peter A Lachenbruch; Frederick W Miller; Jiri Vencovsky; Nicolino Ruperto Journal: Ann Rheum Dis Date: 2017-05 Impact factor: 19.103
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Authors: Ida K Haugen; David Felson; Abhishek Abhishek; Francis Berenbaum; John James Edwards; Gabriel Herrero Beaumont; Merete Hermann-Eriksen; Catherine L Hill; Mariko Ishimori; Helgi Jonsson; Teemu Karjalainen; Ying Ying Leung; Emmanuel Maheu; Christian D Mallen; Rikke Helene Moe; Roberta Ramonda; Valentin Ritschl; Tanja A Stamm; Zoltan Szekanecz; Florus J van der Giesen; Marco J P F Ritt; Ruth Wittoek; Ingvild Kjeken; Nina Osteras; Lotte A van de Stadt; Martin Englund; Krysia S Dziedzic; M Marshall; Sita Bierma-Zeinstra; Paul Hansen; Elsie Greibrokk; Wilma Smeets; Margreet Kloppenburg Journal: RMD Open Date: 2022-02
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