Update on outcome assessment in myositis

Lisa G. Rider, Rohit Aggarwal, Pedro M. MacHado, Jean Yves Hogrel, Ann M. Reed, Lisa Christopher-Stine, Nicolino Ruperto

Research output: Contribution to journalReview article

21 Scopus citations

Abstract

The adult and juvenile myositis syndromes, commonly referred to collectively as idiopathic inflammatory myopathies (IIMs), are systemic autoimmune diseases with the hallmarks of muscle weakness and inflammation. Validated, well-standardized measures to assess disease activity, known as core set measures, were developed by international networks of myositis researchers for use in clinical trials. Composite response criteria using weighted changes in the core set measures of disease activity were developed and validated for adult and juvenile patients with dermatomyositis and adult patients with polymyositis, with different thresholds for minimal, moderate and major improvement in adults and juveniles. Additional measures of muscle strength and function are being validated to improve content validity and sensitivity to change. A health-related quality of life measure, which incorporates patient input, is being developed for adult patients with IIM. Disease state criteria, including criteria for inactive disease and remission, are being used as secondary end points in clinical trials. MRI of muscle and immunological biomarkers are promising approaches to discriminate between disease activity and damage and might provide much-needed objective outcome measures. These advances in the assessment of outcomes for myositis treatment, along with collaborations between international networks, should facilitate further development of new therapies for patients with IIM.

Original languageEnglish (US)
Pages (from-to)303-318
Number of pages16
JournalNature Reviews Rheumatology
Volume14
Issue number5
DOIs
StatePublished - May 1 2018

ASJC Scopus subject areas

  • Rheumatology

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    Rider, L. G., Aggarwal, R., MacHado, P. M., Hogrel, J. Y., Reed, A. M., Christopher-Stine, L., & Ruperto, N. (2018). Update on outcome assessment in myositis. Nature Reviews Rheumatology, 14(5), 303-318. https://doi.org/10.1038/nrrheum.2018.33