A new open-label study published in the European Journal of Neurology has provided evidence of efficacy of cladribine in difficult-to-treat cases of myasthenia gravis (MG) — a rare autoimmune neuromuscular disorder. Cladribine is an immunomodulatory drug that has been approved in some countries for the treatment of relapsing–remitting multiple sclerosis. The study included 13 patients with MG who had shown an insufficient response to standard therapy for this condition. Following 6 months of treatment with cladribine, 11 of the participants demonstrated significant clinical improvement, defined as a reduction of ≥3 points on the Myasthenia Gravis Composite scale. The investigators conclude that cladribine is a promising treatment for MG but additional studies are needed to refine the dosing regime and to determine long-term efficacy and safety.
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Rejdak, K. et al. Cladribine in myasthenia gravis: a pilot open-label study. Eur. J. Neurol. https://doi.org/10.1111/ene.14124 (2019)
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Wood, H. Cladribine shows promise in patients with difficult-to-treat myasthenia gravis. Nat Rev Neurol 16, 2 (2020). https://doi.org/10.1038/s41582-019-0292-z
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DOI: https://doi.org/10.1038/s41582-019-0292-z