2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects

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

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

Objective. The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods. Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results. Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion. Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

Original languageEnglish (US)
Pages (from-to)1884-1893
Number of pages10
JournalRheumatology (United Kingdom)
Volume56
Issue number11
DOIs
StatePublished - Jan 1 2017

Fingerprint

Dermatomyositis
Myositis
Clinical Trials
Weights and Measures
Area Under Curve
Sensitivity and Specificity
Muscle Strength
Natural History
Gold
Software
Logistic Models
Regression Analysis
Physicians
Juvenile dermatomyositis
Surveys and Questionnaires
Therapeutics

Keywords

  • 1000Minds software
  • Conjoint analysis
  • Dermatomyositis
  • Hybrid measure
  • Juvenile dermatomyositis
  • Outcome assessment
  • Polymyositis
  • Response criteria

ASJC Scopus subject areas

  • Rheumatology
  • Pharmacology (medical)

Cite this

the International Myositis Assessment and Clinical Studies Group, & Paediatric Rheumatology International Trials Organisation (2017). 2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects. Rheumatology (United Kingdom), 56(11), 1884-1893. https://doi.org/10.1093/rheumatology/kex226

2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects. / the International Myositis Assessment and Clinical Studies Group; Paediatric Rheumatology International Trials Organisation.

In: Rheumatology (United Kingdom), Vol. 56, No. 11, 01.01.2017, p. 1884-1893.

Research output: Contribution to journalArticle

the International Myositis Assessment and Clinical Studies Group & Paediatric Rheumatology International Trials Organisation 2017, '2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects', Rheumatology (United Kingdom), vol. 56, no. 11, pp. 1884-1893. https://doi.org/10.1093/rheumatology/kex226
the International Myositis Assessment and Clinical Studies Group, Paediatric Rheumatology International Trials Organisation. 2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects. Rheumatology (United Kingdom). 2017 Jan 1;56(11):1884-1893. https://doi.org/10.1093/rheumatology/kex226
the International Myositis Assessment and Clinical Studies Group ; Paediatric Rheumatology International Trials Organisation. / 2016 ACR-EULAR adult dermatomyositis and polymyositis and juvenile dermatomyositis response criteria-methodological aspects. In: Rheumatology (United Kingdom). 2017 ; Vol. 56, No. 11. pp. 1884-1893.
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abstract = "Objective. The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods. Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results. Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion. Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.",
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AU - Ruperto, Nicolino

AU - Pistorio, Angela

AU - Erman, Brian

AU - Bayat, Nastaran

AU - Lachenbruc, Peter A.

AU - Rockette, Howard

AU - Feldman, Brian M.

AU - Huber, Adam M.

AU - Hansen, Paul

AU - Oddis, Chester V.

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AU - Amato, Anthony A.

AU - Chinoy, Hector

AU - Cooper, Robert G.

AU - Chung, Lorinda

AU - Danko, Katalin

AU - Fiorentino, David

AU - De la Torre, Ignacio García

AU - Reed, Ann M.

AU - Song, Yeong Wook

AU - Cimaz, Rolando

AU - Cuttica, Rubén J.

AU - Pilkington, Clarissa A.

AU - Martini, Alberto

AU - van der Net, Janjaap

AU - Maillard, Susan

AU - Miller, Frederick W.

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AU - Aggarwal, Rohit

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AU - Criscione-Schreiber, Lisa

AU - Crofford, Leslie

AU - Cronin, Mary E.

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AU - Schiopu, Elena

AU - Wolfe, Gil

AU - Wortmann, Robert

AU - Apaz, Maria

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AU - Ytterberg, Steven R

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N2 - Objective. The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods. Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results. Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion. Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

AB - Objective. The objective was to describe the methodology used to develop new response criteria for adult DM/PM and JDM. Methods. Patient profiles from prospective natural history data and clinical trials were rated by myositis specialists to develop consensus gold-standard ratings of minimal, moderate and major improvement. Experts completed a survey regarding clinically meaningful improvement in the core set measures (CSM) and a conjoint-analysis survey (using 1000Minds software) to derive relative weights of CSM and candidate definitions. Six types of candidate definitions for response criteria were derived using survey results, logistic regression, conjoint analysis, application of conjoint-analysis weights to CSM and published definitions. Sensitivity, specificity and area under the curve were defined for candidate criteria using consensus patient profile data, and selected definitions were validated using clinical trial data. Results. Myositis specialists defined the degree of clinically meaningful improvement in CSM for minimal, moderate and major improvement. The conjoint-analysis survey established the relative weights of CSM, with muscle strength and Physician Global Activity as most important. Many candidate definitions showed excellent sensitivity, specificity and area under the curve in the consensus profiles. Trial validation showed that a number of candidate criteria differentiated between treatment groups. Top candidate criteria definitions were presented at the consensus conference. Conclusion. Consensus methodology, with definitions tested on patient profiles and validated using clinical trials, led to 18 definitions for adult PM/DM and 14 for JDM as excellent candidates for consideration in the final consensus on new response criteria for myositis.

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KW - Conjoint analysis

KW - Dermatomyositis

KW - Hybrid measure

KW - Juvenile dermatomyositis

KW - Outcome assessment

KW - Polymyositis

KW - Response criteria

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