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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.

SUBMITTER: Rider LG

PROVIDER: S-EPMC6702032 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature

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Update on outcome assessment in myositis.

Rider Lisa G LG Aggarwal Rohit R Machado Pedro M PM Hogrel Jean-Yves JY Reed Ann M AM Christopher-Stine Lisa L Ruperto Nicolino N

Nature reviews. Rheumatology 20180412 5

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 ...[more]

PMID: 29651119

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Project description:Background Telemedicine has changed the landscape of patient care with wider use of patient-centered outcome measures (PCOMs). We evaluated two novel task–based PCOMs namely ten times arm lift (AL) test and two-minute walk distance (2MWD) in idiopathic myositis (IIM). Methods This was a cross-sectional observational study with the enrolment of adult IIM (ACR/EULAR criteria) patients with active/inactive disease. Active disease was defined as any two of increase in immunosuppression within 3 months, elevated muscle enzymes, physician VAS ≥ 2, worsened cutaneous disease, or fall in MMT8 < 76. Standard myositis core set measures (CSMs) were evaluated and test–retest validity [Cronbach’s alfa (CA)], construct validity (Pearson’s correlation), and discriminant validity (between active/inactive IIM) were assessed. The results were further validated in a separate tele-rheumatology cohort. Results Among 22 IIM patients (68%-female) of age 30.5(19–62) years, AL and 2MWD showed excellent test–retest reliability (CA-0.987, 0.99). AL exhibited moderate-strong correlation with all CSMs except CK levels and MDI. In contrast, 2MWD values were highly variable without CSM correlation. A higher AL time discriminated active and inactive myositis (16.6 vs 11 s, p = 0.006) with an AUC of 0.882 (p = 0.006). AL > 12.8 s had 94% negative predictive value (NPV) for active muscle disease. In the validation cohort (47 patient visits among 26 patients), AL significantly differentiated between active vs. inactive disease with an NPV of 95%. Conclusions AL test exhibits pilot evidence of construct and discriminant validity in patients with IIM requiring further evaluation. 2MWD was not a good test for outcome evaluation of IIM patients. Key Points • Novel task–based patient-centered outcome measures were evaluated for remote monitoring of muscle strength in IIM. • Ten times arm lift (AL) test showed strong test–retest reliability as well as provide pilot evidence of construct and discriminant validity in patients with IIM unlike 2-min walk distance. • This provides preliminary evidence to further evaluate the role of AL as patient-centered outcome measure in patients with IIM for virtual clinical trials. Supplementary Information The online version contains supplementary material available at 10.1007/s10067-021-05990-3.

Dataset Information

Update on outcome assessment in myositis.

Publications

Update on outcome assessment in myositis.

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