Sirolimus leads to rapid and sustained clinical improvement of motor deficits in a patient with inclusion body myositis.

To provide further evidence for sirolimus, a mammalian target of rapamycin inhibitor, as a treatment strategy for patients with inclusion body myositis (IBM). We acquired longitudinal clinical data and immunological assessments of CD8+ T-cell subsets in peripheral blood for evaluation of potential anti-inflammatory treatment effects of sirolimus. Therapy with sirolimus 2 mg/day by mouth led to rapid and sustained clinical improvement of motor symptoms for an observation period of more than 1 year. Treatment was well tolerated, with no occurrence of adverse effects. We did not observe a meaningful alteration of CD8+ T-cell subsets in our patient after 9 and 12 months compared to baseline. The significant and persistent clinical improvement highlights the use of sirolimus as a potential treatment option in patients with IBM. In light of the lack of immunological treatment effects observed for cytotoxic CD8+ T cells, further studies should investigate the potential myoprotective effects of sirolimus. © 2022 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.

Citation

Marc Pawlitzki, Christopher Nelke, Melanie Korsen, Sven G Meuth, Tobias Ruck. Sirolimus leads to rapid and sustained clinical improvement of motor deficits in a patient with inclusion body myositis. European journal of neurology. 2022 Apr;29(4):1284-1287

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PMID: 35253967

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