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

for the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation, Carol Wallace

Research output: Contribution to journalArticle

17 Citations (Scopus)

Abstract

Objective: To develop response criteria for juvenile dermatomyositis (DM). Methods: 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. Results: 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). Conclusion: 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.

Original languageEnglish (US)
Pages (from-to)911-923
Number of pages13
JournalArthritis and Rheumatology
Volume69
Issue number5
DOIs
StatePublished - May 1 2017

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

ASJC Scopus subject areas

  • Immunology and Allergy
  • Rheumatology
  • Immunology

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. / for the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation; Wallace, Carol.

In: Arthritis and Rheumatology, Vol. 69, No. 5, 01.05.2017, p. 911-923.

Research output: Contribution to journalArticle

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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 = "Objective: To develop response criteria for juvenile dermatomyositis (DM). Methods: 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. Results: 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). Conclusion: 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.",
author = "{for the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation} and Rider, {Lisa G.} and Rohit Aggarwal and Angela Pistorio and Nastaran Bayat and Brian Erman and Feldman, {Brian M.} and Huber, {Adam M.} and Rolando Cimaz and Cuttica, {Rub{\'e}n J.} and {de Oliveira}, {Sheila Knupp} and Lindsley, {Carol B.} and Pilkington, {Clarissa A.} and Marilynn Punaro and Angelo Ravelli and Reed, {Ann M.} and Kelly Rouster-Stevens and {van Royen-Kerkhof}, Annet and Frank Dressler and Magalhaes, {Claudia Saad} and Tam{\'a}s Constantin and Davidson, {Joyce E.} and Bo Magnusson and Ricardo Russo and Luca Villa and Mariangela Rinaldi and Howard Rockette and Lachenbruch, {Peter A.} and Miller, {Frederick W.} and Jiri Vencovsky and Nicolino Ruperto and Rider, {Lisa G.} and Nicolino Ruperto and Miller, {Frederick W.} and Jiri Vencovsky and Rohit Aggarwal and Brian Erman and Nastaran Bayat and Angela Pistorio and Huber, {Adam M.} and Feldman, {Brian M.} and Paul Hansen and Howard Rockette and Lachenbruch, {Peter A.} and Nicolino Ruperto and Rider, {Lisa G.} and Maria Apaz and Suzanne Bowyer and Rolando Cimaz and Carol Wallace and Ilowite, {Norman Todd}",
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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 - for the International Myositis Assessment and Clinical Studies Group and the Paediatric Rheumatology International Trials Organisation

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 - Magalhaes, Claudia Saad

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 - Rider, Lisa G.

AU - Ruperto, Nicolino

AU - Miller, Frederick W.

AU - Vencovsky, Jiri

AU - Aggarwal, Rohit

AU - Erman, Brian

AU - Bayat, Nastaran

AU - Pistorio, Angela

AU - Huber, Adam M.

AU - Feldman, Brian M.

AU - Hansen, Paul

AU - Rockette, Howard

AU - Lachenbruch, Peter A.

AU - Ruperto, Nicolino

AU - Rider, Lisa G.

AU - Apaz, Maria

AU - Bowyer, Suzanne

AU - Cimaz, Rolando

AU - Wallace, Carol

AU - Ilowite, Norman Todd

PY - 2017/5/1

Y1 - 2017/5/1

N2 - Objective: To develop response criteria for juvenile dermatomyositis (DM). Methods: 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. Results: 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). Conclusion: 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.

AB - Objective: To develop response criteria for juvenile dermatomyositis (DM). Methods: 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. Results: 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). Conclusion: 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.

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U2 - 10.1002/art.40060

DO - 10.1002/art.40060

M3 - Article

VL - 69

SP - 911

EP - 923

JO - Arthritis and Rheumatology

JF - Arthritis and Rheumatology

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ER -