TY - JOUR
T1 - 2016 American College of Rheumatology/European League Against Rheumatism criteria for minimal, moderate, and major clinical response in adult dermatomyositis and polymyositis
T2 - An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative
AU - International Myositis Assessment and Clinical Studies Group
AU - Paediatric Rheumatology International Trials Organisation
AU - Aggarwal, Rohit
AU - Rider, Lisa G.
AU - Ruperto, Nicolino
AU - Bayat, Nastaran
AU - Erman, Brian
AU - Feldman, Brian M.
AU - Oddis, Chester V.
AU - Amato, Anthony A.
AU - Chinoy, Hector
AU - Cooper, Robert G.
AU - Dastmalchi, Maryam
AU - Fiorentino, David
AU - Isenberg, David
AU - Katz, James D.
AU - Mammen, Andrew
AU - De Visser, Marianne
AU - Ytterberg, Steven R.
AU - Lundberg, Ingrid E.
AU - Chung, Lorinda
AU - Danko, Katalin
AU - García-De La Torre, Ignacio
AU - Song, Yeong Wook
AU - Villa, Luca
AU - Rinaldi, Mariangela
AU - Rockette, Howard
AU - Lachenbruch, Peter A.
AU - Miller, Frederick W.
AU - Vencovsky, Jiri
N1 - Funding Information:
We thank the following individuals for providing invaluable input and feedback on project development and support: members of the American College of Rheumatology Criteria Committee; Dr Daniel Aletaha (European League Against Rheumatism), Drs Suzette Peng and Sarah Yim (US Food and Drug Administration), Drs Thorsten Vetter and Richard Vesely (European Medicines Agency), Bob Goldberg and Theresa Curry (The Myositis Association), Rhonda McKeever and Patti Lawler (Cure JM Foundation), and Irene Oakley (Myositis UK). We also thank Drs Michael Ward, Steven Pavletic, and Adam Schiffenbauer for their critical review of the manuscript. Paul Hansen, who with Franz Ombler owns and co-invented the 1000Minds software referred to in the article, provided intellectual and logistic support for this project. 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 and D Project through the Korea Health Industry Development Institute funded by the Ministry of Health and 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).
Publisher Copyright:
© 2017, BMJ Publishing Group. All rights reserved.
PY - 2017/5/1
Y1 - 2017/5/1
N2 - 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.
AB - 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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U2 - 10.1136/annrheumdis-2017-211400
DO - 10.1136/annrheumdis-2017-211400
M3 - Article
C2 - 28385805
AN - SCOPUS:85019025865
VL - 76
SP - 792
EP - 801
JO - Annals of the Rheumatic Diseases
JF - Annals of the Rheumatic Diseases
SN - 0003-4967
IS - 5
ER -