International consensus on preliminary definitions of improvement in adult and juvenile myositis

Lisa G. Rider, Edward H. Giannini, Hermine I. Brunner, Nicola Ruperto, Laura James-Newton, Ann M. Reed, Peter A. Lachenbruch, Frederick W. Miller

Research output: Contribution to journalArticle

158 Citations (Scopus)

Abstract

Objective. To use a core set of outcome measures to develop preliminary definitions of improvement for adult and juvenile myositis as composite end points for therapeutic trials. Methods. Twenty-nine experts in the assessment of myositis achieved consensus on 102 adult and 102 juvenile paper patient profiles as clinically improved or not improved. Two hundred twenty-seven candidate definitions of improvement were developed using the experts' consensus ratings as a gold standard and their judgment of clinically meaningful change in the core set of measures. Seventeen additional candidate definitions of improvement were developed from classification and regression tree analysis, a data-mining decision tree tool analysis. Six candidate definitions specifying percentage change or raw change in the core set of measures were developed using logistic regression analysis. Adult and pediatric working groups ranked the 13 top-performing candidate definitions for face validity, clinical sensibility, and ease of use, in which the sensitivity and specificity were ≥75% in adult, pediatric, and combined data sets. Nominal group technique was used to facilitate consensus formation. Results. The definition of improvement (common to the adult and pediatric working groups) that ranked highest was 3 of any 6 of the core set measures improved by ≥20%, with no more than 2 worse by ≥25% (which could not include manual muscle testing to assess strength). Five and 4 additional preliminary definitions of improvement for adult and juvenile myositis, respectively, were also developed, with several definitions common to both groups. Participants also agreed to prospectively test 6 logistic regression definitions of improvement in clinical trials. Conclusion. Consensus preliminary definitions of improvement were developed for adult and juvenile myositis, and these incorporate clinically meaningful change in all myositis core set measures in a composite end point. These definitions require prospective validation, but they are now proposed for use as end points in all myositis trials.

Original languageEnglish (US)
Pages (from-to)2281-2290
Number of pages10
JournalArthritis and Rheumatism
Volume50
Issue number7
DOIs
StatePublished - Jul 2004

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Myositis
Consensus
Pediatrics
Logistic Models
Regression Analysis
Decision Trees
Data Mining
Reproducibility of Results
Outcome Assessment (Health Care)
Clinical Trials
Sensitivity and Specificity
Muscles
Juvenile dermatomyositis
Therapeutics

ASJC Scopus subject areas

  • Immunology
  • Rheumatology

Cite this

Rider, L. G., Giannini, E. H., Brunner, H. I., Ruperto, N., James-Newton, L., Reed, A. M., ... Miller, F. W. (2004). International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis and Rheumatism, 50(7), 2281-2290. https://doi.org/10.1002/art.20349

International consensus on preliminary definitions of improvement in adult and juvenile myositis. / Rider, Lisa G.; Giannini, Edward H.; Brunner, Hermine I.; Ruperto, Nicola; James-Newton, Laura; Reed, Ann M.; Lachenbruch, Peter A.; Miller, Frederick W.

In: Arthritis and Rheumatism, Vol. 50, No. 7, 07.2004, p. 2281-2290.

Research output: Contribution to journalArticle

Rider, LG, Giannini, EH, Brunner, HI, Ruperto, N, James-Newton, L, Reed, AM, Lachenbruch, PA & Miller, FW 2004, 'International consensus on preliminary definitions of improvement in adult and juvenile myositis', Arthritis and Rheumatism, vol. 50, no. 7, pp. 2281-2290. https://doi.org/10.1002/art.20349
Rider LG, Giannini EH, Brunner HI, Ruperto N, James-Newton L, Reed AM et al. International consensus on preliminary definitions of improvement in adult and juvenile myositis. Arthritis and Rheumatism. 2004 Jul;50(7):2281-2290. https://doi.org/10.1002/art.20349
Rider, Lisa G. ; Giannini, Edward H. ; Brunner, Hermine I. ; Ruperto, Nicola ; James-Newton, Laura ; Reed, Ann M. ; Lachenbruch, Peter A. ; Miller, Frederick W. / International consensus on preliminary definitions of improvement in adult and juvenile myositis. In: Arthritis and Rheumatism. 2004 ; Vol. 50, No. 7. pp. 2281-2290.
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abstract = "Objective. To use a core set of outcome measures to develop preliminary definitions of improvement for adult and juvenile myositis as composite end points for therapeutic trials. Methods. Twenty-nine experts in the assessment of myositis achieved consensus on 102 adult and 102 juvenile paper patient profiles as clinically improved or not improved. Two hundred twenty-seven candidate definitions of improvement were developed using the experts' consensus ratings as a gold standard and their judgment of clinically meaningful change in the core set of measures. Seventeen additional candidate definitions of improvement were developed from classification and regression tree analysis, a data-mining decision tree tool analysis. Six candidate definitions specifying percentage change or raw change in the core set of measures were developed using logistic regression analysis. Adult and pediatric working groups ranked the 13 top-performing candidate definitions for face validity, clinical sensibility, and ease of use, in which the sensitivity and specificity were ≥75{\%} in adult, pediatric, and combined data sets. Nominal group technique was used to facilitate consensus formation. Results. The definition of improvement (common to the adult and pediatric working groups) that ranked highest was 3 of any 6 of the core set measures improved by ≥20{\%}, with no more than 2 worse by ≥25{\%} (which could not include manual muscle testing to assess strength). Five and 4 additional preliminary definitions of improvement for adult and juvenile myositis, respectively, were also developed, with several definitions common to both groups. Participants also agreed to prospectively test 6 logistic regression definitions of improvement in clinical trials. Conclusion. Consensus preliminary definitions of improvement were developed for adult and juvenile myositis, and these incorporate clinically meaningful change in all myositis core set measures in a composite end point. These definitions require prospective validation, but they are now proposed for use as end points in all myositis trials.",
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