What are other medical studies done for the treatment of sporadic inclusion body myositis (s-IBM)?

Updated: Jun 08, 2018
  • Author: Michael P Collins, MD; Chief Editor: Nicholas Lorenzo, MD, MHA, CPE  more...
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A pilot trial of etanercept, a tumor necrosis factor (TNF) alpha blocker, did not show significant benefit in composite muscle strength scores at 6 months. However, with 12 months of treatment, slight improvement in grip strength was noted. [110] . A double-blinded, randomized, placebo-controlled study is currently underway to assess the efficacy of etanercept treatment in patients with IBM.

A study of alemtuzumab, a T-cell–depleting monoclonal antibody, involved 13 patients who underwent infusion of 0.3 mg/kg/d for 4 days. It reported slowed disease progression, improvement of strength in some patients, and reduction in endomysial inflammation. [111] This preliminary study holds promise for future studies.

Follistatin, an antagonist of the myostatin pathway, has been shown to produce a dramatic increase in muscle mass in animals. [112] These results are promising for future gene therapy trials to improve muscle mass in patients with neuromuscular disease.

Arimoclomol, a heat shock protein (HSP) coinducer may slow down the process of protein misfolding and aggregation. A study of its safety and efficacy in IBM is underway.

Lithium is an inhibitor of the glycogen synthase kinase (GSK) enzyme, the latter of which is involved in the development of phosphorylated tau (p-tau). A recent study has shown that in biopsied s-IBM muscle fibers, GSK3b activity is increased, with increased ABPP phosphorylation. Treatment with lithium showed decreased GSK3b activity, decreased amounts of total and phosphorylated ABPP and AB oligomers, and increased proteosomal function. [113] These findings suggest that treatment of patients with s-IBM with lithium may be beneficial.

Empiric therapies include coenzyme Q10, carnitine, and antioxidants. They may provide benefit to some patients, but, to date, none of these has been studied in a controlled clinical trial.

Routine follow-up visits at intervals contingent upon the progression and severity of involvement are indicated to assess the patient's strength, tolerance of exertion, and compromise in occupation or activities of daily living. Hicks has outlined a strategy for care of patients with inflammatory myopathies, including s-IBM. [114]

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