TY - JOUR
T1 - 2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Juvenile Dermatomyositis
T2 - An International Myositis Assessment and Clinical Studies Group/Paediatric Rheumatology International Trials Organisation Collaborative Initiative
AU - Rider, Lisa G
AU - Aggarwal, Rohit
AU - Pistorio, Angela
AU - Bayat, Nastaran
AU - Erman, Brian
AU - Feldman, Brian M
AU - Huber, Adam M
AU - Cimaz, Rolando
AU - Cuttica, Rubén J
AU - de Oliveira, Sheila Knupp
AU - Lindsley, Carol B
AU - Pilkington, Clarissa A
AU - Punaro, Marilynn
AU - Ravelli, Angelo
AU - Reed, Ann M
AU - Rouster-Stevens, Kelly
AU - van Royen-Kerkhof, Annet
AU - Dressler, Frank
AU - Saad Magalhaes, Claudia
AU - Constantin, Tamás
AU - Davidson, Joyce E
AU - Magnusson, Bo
AU - Russo, Ricardo
AU - Villa, Luca
AU - Rinaldi, Mariangela
AU - Rockette, Howard
AU - Lachenbruch, Peter A
AU - Miller, Frederick W
AU - Vencovsky, Jiri
AU - Ruperto, Nicolino
AU - International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation
N1 - Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://www.bmj.com/company/products-services/rights-and-licensing/.
PY - 2017/5
Y1 - 2017/5
N2 - 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.
AB - 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.
KW - Adolescent
KW - Adult
KW - Child
KW - Child, Preschool
KW - Consensus
KW - Dermatomyositis
KW - Humans
KW - Outcome Assessment (Health Care)
KW - Randomized Controlled Trials as Topic
KW - Sensitivity and Specificity
KW - Severity of Illness Index
KW - Consensus Development Conference
KW - Journal Article
KW - Practice Guideline
KW - Validation Studies
U2 - 10.1136/annrheumdis-2017-211401
DO - 10.1136/annrheumdis-2017-211401
M3 - Article
C2 - 28385804
SN - 0003-4967
VL - 76
SP - 782
EP - 791
JO - Annals of the Rheumatic Diseases
JF - Annals of the Rheumatic Diseases
IS - 5
ER -