Treatment for inclusion body myositis

Abstract

Data collection and analysis We used standard Cochrane methodological procedures. Main results The review included 10 trials (249 participants) using different treatment regimens. Seven of the 10 trials assessed single agents, and 3 assessed combined agents. Many of the studies did not present adequate data for the reporting of the primary outcome of the review, which was the percentage change in muscle strength score at six months. Pooled data from two trials of interferon beta-1a (n = 58) identified no important difference in normalised manual muscle strength sum scores from baseline to six months (mean difference (MD)-0.06, 95% CI-0.15 to 0.03) between IFN beta-1a and placebo (moderate-quality evidence). A single trial of methotrexate (MTX) (n = 44) provided moderate-quality evidence that MTX did not arrest or slow disease progression, based on reported percentage change in manual muscle strength sum scores at 12 months. None of the fully published trials were adequately powered to detect a treatment effect. We assessed six of the nine fully published trials as providing very low-quality evidence in relation to the primary outcome measure. Three trials (n = 78) compared intravenous immunoglobulin (combined in one trial with prednisone) to a placebo, but we were unable to perform meta-analysis because of variations in study analysis and presentation of trial data, with no access to the primary data for re-analysis. Other comparisons were also reported in single trials. An open trial of anti-T lymphocyte immunoglobulin (ATG) combined with MTX versus MTX provided very low-quality evidence in favour of the combined therapy, based on percentage change in quantitative muscle strength sum scores at 12 months (MD 12.50%, 95% CI 2.43 to 22.57). Data from trials of oxandrolone versus placebo, azathioprine (AZA) combined with MTX versus MTX, and arimoclomol versus placebo did not allow us to report either normalised or percentage change in muscle strength sum scores. A complete analysis of the effects of arimoclomol is pending data publication. Studies of simvastatin and bimagrumab (BYM338) are ongoing. All analysed trials reported adverse events. Only 1 of the 10 trials interpreted these for statistical significance. None of the trials included prespecified criteria for significant adverse events. Authors' conclusions Trials of interferon beta-1a and MTX provided moderate-quality evidence of having no effect on the progression of IBM. Overall trial design limitations including risk of bias, low numbers of participants, and short duration make it difficult to say whether or not any of the drug treatments included in this review were effective. An open trial of ATG combined with MTX versus MTX provided very lowquality evidence in favour of the combined therapy based on the percentage change data given. We were unable to draw conclusions from trials of IVIg, oxandrolone, and AZA plus MTX versus MTX. We need more randomised controlled trials that are larger, of longer duration, and that use fully validated, standardised, an...

Longitudinal Changes in MRI Muscle Morphometry and Composition in People With Inclusion Body Myositis

“…Treatments targeting the inflammatory component have not been successful in sIBM. 16,17 For this reason, new efforts are now geared toward the development of muscle-regenerative drugs and drugs that target protein dyshomeostasis. [16][17][18][19][20] Outcome assessment in sIBM has included measures of healthrelated quality of life, physical function, mobility, strength, and endurance.…”

“…16,17 For this reason, new efforts are now geared toward the development of muscle-regenerative drugs and drugs that target protein dyshomeostasis. [16][17][18][19][20] Outcome assessment in sIBM has included measures of healthrelated quality of life, physical function, mobility, strength, and endurance. 21,22 Although these assessments seek to provide a simple measurement of patients' functional status, the inherent variability in ambulatory outcomes can make these outcome measures relatively insensitive to small changes.…”

The host defense peptide LL-37 a possible inducer of the type I interferon system in patients with polymyositis and dermatomyositis

“…sIBM is even more difficult to treat and there are no data about beneficial effects of pharmacological treatment in sIBM [28]. Randomized placebo-controlled trials of large patient groups, long duration of treatment, more useful validation, better outcome measures are highly warranted to investigate the effects of different therapies on the progression of sIBM [31].…”

Efficacy of immunosuppressive treatment in a systemic lupus erythematosus patient presenting with inclusion body myositis

“…IBM is considered to be resistant to treatment with corticosteroids and immunosuppressive drugs, as the degeneration of muscle fibres is the main feature promoting slow disease progression 3 17. However, this case, together with other reports, serves to support the concept that patients with underlying SLE may have a more prominent immune-mediated inflammatory component in the disease manifestations, thus explaining the response to therapy.…”

“…IBM tends to more frequently affect males over the age of 50 years and carries a poor prognosis 2. This form of myopathy is considered to be, in most cases, refractory to treatment with conventional immunosuppressive medications and those who respond, subsequently have a progressive slow decay in function 3. IBM usually presents as an isolated disease but there are some reports of IBM occurring in patients with autoimmune connective diseases, including systemic lupus erythematosus (SLE) 4–6.…”