Description
- Abstract:
- "Introduction: Hematopoietic stem cell transplantation (HSCT) treatment is a combined therapy that involves autoimmune cell depletion and infusion of healthy hematopoietic stem cells (HSCs) to restore blood cell production. HSCT has been shown to be an effective treatment for many severe autoimmune diseases including myasthenia gravis (MG). There have been a number of reported cases of successful HSCT in severe MG that are refractory to standard treatment. However, a summary of its efficacy in MG from the published literature has not been performed. Methods: Databases including PubMed, clinicaltrials.gov, and Cochrane Reviews were searched for research articles containing “hematopoietic stem cell transplantation,” “stem cell,” and “myasthenia gravis.” Articles were excluded if they were not written in English. Results: Literature review identified and included sixteen patients (12 females, 4 males) from eight publications describing HSCT treatment for MG. Fifteen patients received autologous HSCT while one received allogeneic. Patients ranged in age from 17–64 years (median 46), with MG duration of 1–38 years (median 6). Most had severe disease (MGFA classes IV or V). Before HSCT, all had received multiple standard treatments, most commonly pyridostigmine, prednisone, plasmapheresis, IVIG, and other immunosuppressants. After a median follow-up of 38 months following HSCT treatment, all patients showed significant clinical improvement, typically beginning within 7 weeks post-transplantation. Fourteen (87.5%) discontinued all MG treatment, and eleven (68.8%) achieved complete stable remission. The remaining patients had marked improvement with fewer exacerbations. Common adverse effects included neutropenic fever, cytomegalovirus viremia or reactivation, mucositis, and upper respiratory tract infection. Conclusion: Literature review supports that HSCT provides sustained benefits for a group of patients with MG who are refractory to conventional treatment. A large-scale prospective study is warranted to better define its role in the treatment of refractory MG."
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Citation
Li, Grace , Heber, Morgan , Pike-Lee, Tiffany,
"An updated review on the efficacy of hematopoietic stem cell transplantation for refractory myasthenia gravis"
(2025).
Student Neurosurgery/Neurology Research Conference.
Brown Digital Repository. Brown University Library.
https://doi.org/10.26300/rx7d-gf97
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Student Neurosurgery/Neurology Research Conference
Brown University Department of Neurosurgery and Department of Neurology and Warren Alpert Medical School annually hosts the Student Neurosurgery/Neurology Research Conference, a national conference for medical students conducting research in neurosurgery/neurology or related neuroscience fields....