Santos, Eduardo Jose Ferreira;
Farisogullari, Bayram;
Yapp, Nicholas;
Townsley, Hermaleigh;
Sousa, Pedro;
Machado, Pedro M;
(2025)
Efficacy and safety of pharmacological treatments in inclusion body myositis: a systematic review.
RMD Open
, 11
(1)
, Article e005176. 10.1136/rmdopen-2024-005176.
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Abstract
OBJECTIVE: To identify the best evidence on the efficacy of treatment interventions for inclusion body myositis (IBM) and to describe their safety. METHODS: Systematic review of randomised controlled trials (RCTs) of pharmacological treatments of adults with IBM, conducted according to the Cochrane Handbook, updating a previous Cochrane review. The search strategy was run on Cochrane Neuromuscular Disease Group Specialized Register, CENTRAL, MEDLINE and EMBASE, ClinicalTrials.gov and the WHO International Clinical Trials Registry Platform. Assessment of risk of bias, data extraction and synthesis were performed independently by two reviewers. Data pooled in statistical meta-analyses, if possible. RESULTS: From a total of 487 records, 48 were selected for full-text review, 14 fulfilled the inclusion criteria, but only 2 RCTs were included in meta-analyses due to clinical heterogeneity (different drug interventions or dosages). Treatments included various immunosuppressive and immunomodulatory agents, alongside interventions modulating muscle growth and protein homoeostasis. Efficacy was assessed across multiple outcomes, namely muscle strength, physical function, mobility and muscle trophicity. Trials of methotrexate (MTX), intravenous immunoglobulin, interferon beta-1a and MTX, MTX and anti-T-lymphocyte immunoglobulin, oxandrolone, MTX and azathioprine, bimagrumab, arimoclomol, and sirolimus provided low-quality to high-quality evidence of having no effect on the progression of IBM. CONCLUSIONS: Drug interventions for IBM were not effective for most of the outcomes of interest. We observed inconsistency of outcome measures across trials. More RCTs are needed, of adequate size and duration, and using a standardised set of outcome measures.
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