Update on outcome assessment in myositis

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.

Acute Exacerbation of Interstitial Lung Disease in Adult Patients With Idiopathic Inflammatory Myopathies: A Retrospective Case-Control Study

“…The predictive role of MYOACT score in this study might lie in the partially overlapped pathological mechanism between AE-ILD and IIM. Elevated levels of several cytokines and chemokines, namely IL-6, IL-8, IL-17, IL-23, etc., were seen in peripheral blood, muscle or skin of IIM patients, and were consistent with disease activity (37). Meanwhile several studies also observed significant elevation of cytokines and chemokines including IL-6, IL-8 in patients with ILD exacerbation, and the elevation was found to be related to worse outcome (38,39).…”

Neutrophil dysregulation is pathogenic in idiopathic inflammatory myopathies

“…MSAs were detected by standardized immunoprecipitation and immunoprecipitation-immunoblot methods (34) at the NIH Clinical Center Central Laboratory. Clinical characterization and measurements of IIM disease activity and damage were obtained as previously described (35,36). Videocapillaroscopy was quantified as described previously (37).…”

Real-world Experience of Nintedanib Therapy in Idiopathic-inflammatory-myopathy-related Interstitial Lung Disease: Efficacy and Tolerability

“…As a core set measure for IIM patients, MYOACT score work as an overall assessment of IIM disease activity by taking muscular and various extra-muscular manifestations into consideration. It has been demonstrated to predict outcome of IIM patients and re ect in ammatory level [47,48]. Meanwhile RP-ILD was also found to be associated with cytokines like tumor necrosis factor, etc [49].…”