title: 2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis: 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 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.
  
  
  
  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).
  
  
  
  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.affiliation: NIH, Bethesda, Maryland.
authors.forename: Lisa G
authors.initials: LG
authors.lastname: Rider
authors.affiliation: University of Pittsburgh, Pittsburgh, Pennsylvania.
authors.forename: Rohit
authors.initials: R
authors.lastname: Aggarwal
authors.affiliation: Istituto Giannina Gaslini, Servizio di Epidemiologia e Biostatistica, Genoa, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Pistorio
authors.affiliation: NIH, Bethesda, Maryland.
authors.forename: Nastaran
authors.initials: N
authors.lastname: Bayat
authors.affiliation: Social and Scientific Systems, Inc., Durham, North Carolina.
authors.forename: Brian
authors.initials: B
authors.lastname: Erman
authors.affiliation: The Hospital for Sick Children, Toronto, Ontario, Canada.
authors.forename: Brian M
authors.initials: BM
authors.lastname: Feldman
authors.affiliation: IWK Health Centre, Halifax, Nova Scotia, Canada.
authors.forename: Adam M
authors.initials: AM
authors.lastname: Huber
authors.affiliation: University of Firenze, Florence, Italy.
authors.forename: Rolando
authors.initials: R
authors.lastname: Cimaz
authors.affiliation: Hospital de Niños Pedro de Elizalde, University of Buenos Aires, Buenos Aires, Argentina.
authors.forename: Rubén J
authors.initials: RJ
authors.lastname: Cuttica
authors.affiliation: Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil.
authors.forename: Sheila Knupp
authors.initials: SK
authors.lastname: de Oliveira
authors.affiliation: University of Kansas City Medical Center, Kansas City, Kansas.
authors.forename: Carol B
authors.initials: CB
authors.lastname: Lindsley
authors.affiliation: Great Ormond Street Hospital for Children NHS Trust, London, UK.
authors.forename: Clarissa A
authors.initials: CA
authors.lastname: Pilkington
authors.affiliation: University of Texas Southwestern Medical Center, Dallas.
authors.forename: Marilynn
authors.initials: M
authors.lastname: Punaro
authors.affiliation: Istituto Giannina Gaslini, Pediatria II - Reumatologia, and Università degli Studi di Genova, Genoa, Italy.
authors.forename: Angelo
authors.initials: A
authors.lastname: Ravelli
authors.affiliation: Duke University, Durham, North Carolina.
authors.forename: Ann M
authors.initials: AM
authors.lastname: Reed
authors.affiliation: Emory University School of Medicine, Atlanta, Georgia.
authors.forename: Kelly
authors.initials: K
authors.lastname: Rouster-Stevens
authors.affiliation: University Medical Centre Utrecht, Wilhelmina Children's Hospital, Utrecht, The Netherlands.
authors.forename: Annet
authors.initials: A
authors.lastname: van Royen-Kerkhof
authors.affiliation: Hannover Medical School, Hannover, Germany.
authors.forename: Frank
authors.initials: F
authors.lastname: Dressler
authors.affiliation: Universidade Estadual Paulista Júlio de Mesquita Filho, Botucatu, Saõ Paulo, Brazil.
authors.forename: Claudia Saad
authors.initials: CS
authors.lastname: Magalhaes
authors.affiliation: Semmelweis University, Budapest, Hungary.
authors.forename: Tamás
authors.initials: T
authors.lastname: Constantin
authors.affiliation: Royal Hospital for Sick Children, Glasgow, UK, and Royal Hospital for Sick Children, Edinburgh, UK.
authors.forename: Joyce E
authors.initials: JE
authors.lastname: Davidson
authors.affiliation: Karolinska University Hospital, Stockholm, Sweden.
authors.forename: Bo
authors.initials: B
authors.lastname: Magnusson
authors.affiliation: Hospital de Pediatría Garrahan, Buenos Aires, Argentina.
authors.forename: Ricardo
authors.initials: R
authors.lastname: Russo
authors.affiliation: Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO, Genoa, Italy.
authors.forename: Luca
authors.initials: L
authors.lastname: Villa
authors.affiliation: Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO, Genoa, Italy.
authors.forename: Mariangela
authors.initials: M
authors.lastname: Rinaldi
authors.affiliation: University of Pittsburgh, Pittsburgh, Pennsylvania.
authors.forename: Howard
authors.initials: H
authors.lastname: Rockette
authors.affiliation: NIH, Bethesda, Maryland.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Lachenbruch
authors.affiliation: NIH, Bethesda, Maryland.
authors.forename: Frederick W
authors.initials: FW
authors.lastname: Miller
authors.affiliation: Charles University, Prague, Czech Republic.
authors.forename: Jiri
authors.initials: J
authors.lastname: Vencovsky
authors.affiliation: Istituto Giannina Gaslini, Pediatria II - Reumatologia, PRINTO, Genoa, Italy.
authors.forename: Nicolino
authors.initials: N
authors.lastname: Ruperto
mesh_terms: D000293:Adolescent; D000410:Alanine Transaminase / Q000378:metabolism; D018501:Antirheumatic Agents / Q000627:therapeutic use*; D001219:Aspartate Aminotransferases / Q000378:metabolism; D002648:Child; D003402:Creatine Kinase / Q000378:metabolism; D016572:Cyclosporine / Q000627:therapeutic use; D003882:Dermatomyositis / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D005060:Europe; D005634:Fructose-Bisphosphate Aldolase / Q000378:metabolism; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D007770:L-Lactate Dehydrogenase / Q000378:metabolism; D016015:Logistic Models; D008727:Methotrexate / Q000627:therapeutic use; D053580:Muscle Strength; D017063:Outcome Assessment, Health Care; D000071066:Patient Reported Outcome Measures; D011241:Prednisone / Q000627:therapeutic use; D015203:Reproducibility of Results; D012219:Rheumatology; D000069283:Rituximab / Q000627:therapeutic use; D012955:Societies, Medical; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D014481:United States
chemical_list: D018501:Antirheumatic Agents; D005938:Glucocorticoids; D000069283:Rituximab; D016572:Cyclosporine; D007770:L-Lactate Dehydrogenase; D001219:Aspartate Aminotransferases; D000410:Alanine Transaminase; D003402:Creatine Kinase; D005634:Fructose-Bisphosphate Aldolase; D011241:Prednisone; D008727:Methotrexate