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

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 & 11 others Joyce E Davidson, Bo Magnusson, Ricardo Russo, Luca Villa, Mariangela Rinaldi, Howard Rockette, Peter A Lachenbruch, Frederick W Miller, Jiri Vencovsky, Nicolino Ruperto, International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

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.

Original languageEnglish
Pages (from-to)782-791
Number of pages10
JournalAnnals of the Rheumatic Diseases
Volume76
Issue number5
DOIs
Publication statusPublished - May 2017

Fingerprint

Pediatrics
Myositis
Rheumatology
Prednisone
Dermatomyositis
Natural History
Reproducibility of Results
Methotrexate
Cyclosporine
Physicians
Sensitivity and Specificity
Clinical Studies
Juvenile dermatomyositis
Rituximab
Therapeutics

Keywords

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Consensus
  • Dermatomyositis
  • Humans
  • Outcome Assessment (Health Care)
  • Randomized Controlled Trials as Topic
  • Sensitivity and Specificity
  • Severity of Illness Index
  • Consensus Development Conference
  • Journal Article
  • Practice Guideline
  • Validation Studies

Cite this

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. / Rider, Lisa G; Aggarwal, Rohit; Pistorio, Angela; Bayat, Nastaran; Erman, Brian; Feldman, Brian M; Huber, Adam M; Cimaz, Rolando; Cuttica, Rubén J; de Oliveira, Sheila Knupp; Lindsley, Carol B; Pilkington, Clarissa A; Punaro, Marilynn; Ravelli, Angelo; Reed, Ann M; Rouster-Stevens, Kelly; van Royen-Kerkhof, Annet; Dressler, Frank; Saad Magalhaes, Claudia; Constantin, Tamás; Davidson, Joyce E; Magnusson, Bo; Russo, Ricardo; Villa, Luca; Rinaldi, Mariangela; Rockette, Howard; Lachenbruch, Peter A; Miller, Frederick W; Vencovsky, Jiri; Ruperto, Nicolino; International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation.

In: Annals of the Rheumatic Diseases, Vol. 76, No. 5, 05.2017, p. 782-791.

Research output: Contribution to journalArticle

Rider, LG, Aggarwal, R, Pistorio, A, Bayat, N, Erman, B, Feldman, BM, Huber, AM, Cimaz, R, Cuttica, RJ, de Oliveira, SK, Lindsley, CB, Pilkington, CA, Punaro, M, Ravelli, A, Reed, AM, Rouster-Stevens, K, van Royen-Kerkhof, A, Dressler, F, Saad Magalhaes, C, Constantin, T, Davidson, JE, Magnusson, B, Russo, R, Villa, L, Rinaldi, M, Rockette, H, Lachenbruch, PA, Miller, FW, Vencovsky, J, Ruperto, N & International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation 2017, '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', Annals of the Rheumatic Diseases, vol. 76, no. 5, pp. 782-791. https://doi.org/10.1136/annrheumdis-2017-211401
Rider, Lisa G ; Aggarwal, Rohit ; Pistorio, Angela ; Bayat, Nastaran ; Erman, Brian ; Feldman, Brian M ; Huber, Adam M ; Cimaz, Rolando ; Cuttica, Rubén J ; de Oliveira, Sheila Knupp ; Lindsley, Carol B ; Pilkington, Clarissa A ; Punaro, Marilynn ; Ravelli, Angelo ; Reed, Ann M ; Rouster-Stevens, Kelly ; van Royen-Kerkhof, Annet ; Dressler, Frank ; Saad Magalhaes, Claudia ; Constantin, Tamás ; Davidson, Joyce E ; Magnusson, Bo ; Russo, Ricardo ; Villa, Luca ; Rinaldi, Mariangela ; Rockette, Howard ; Lachenbruch, Peter A ; Miller, Frederick W ; Vencovsky, Jiri ; Ruperto, Nicolino ; International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation. / 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. In: Annals of the Rheumatic Diseases. 2017 ; Vol. 76, No. 5. pp. 782-791.
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abstract = "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.",
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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

VL - 76

SP - 782

EP - 791

JO - Annals of the Rheumatic Diseases

JF - Annals of the Rheumatic Diseases

SN - 0003-4967

IS - 5

ER -