{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T02:10:00Z","timestamp":1782871800448,"version":"3.54.5"},"reference-count":43,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,12,31]],"date-time":"2024-12-31T00:00:00Z","timestamp":1735603200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>Patients with refractory myasthenia gravis (MG) have few treatment options. Autologous hematopoietic stem cell transplantation (HSCT) has been used to treat immune diseases; however, its use in the treatment of MG is not broadly considered. Our objective is to report on the efficacy and safety of HSCT in refractory MG.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Twenty\u2010one patients who underwent HSCT for MG were retrospectively reviewed. All patients had severe MG refractory to multiple therapies. Stem cells were mobilized with cyclophosphamide and granulocyte colony\u2010stimulating factor. The grafts were depleted of immune cells by selecting CD34+ cells. HSCT conditioning consisted of high\u2010dose cytoreductive therapy and anti\u2010thymocyte globulin. The primary efficacy outcome was achieving clinically stable remission or minimal manifestations without treatment and remaining as such until most recent follow\u2010up.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The median time from MG diagnosis to HSCT was 4.0\u2009years. The primary outcome was reached in 16 of 18 evaluable patients (89%) at a median of 1.7\u2009years and maintained with a median follow\u2010up of 6.7\u2009years (range 1.0\u201321.9\u2009years). Three patients were not evaluable for the primary outcome: one due to confounding illness and two died within 12\u2009months of transplant. The transplant\u2010related mortality at 100\u2009days was 9.5%. Two late deaths occurred, with uncertain relation to the HSCT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>After HSCT for refractory MG, most patients achieved sustained disease remission. However, HSCT\u2010related mortality in medically complex MG patients may be high. Prospective studies investigating the efficacy and safety of HSCT in the treatment of refractory MG are warranted.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.52246","type":"journal-article","created":{"date-parts":[[2024,12,31]],"date-time":"2024-12-31T12:14:40Z","timestamp":1735647280000},"page":"56-68","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Refractory myasthenia gravis treated with autologous hematopoietic stem cell transplantation"],"prefix":"10.1002","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9574-1412","authenticated-orcid":false,"given":"Benjamin","family":"Beland","sequence":"first","affiliation":[{"name":"Division of Neurology, Department of Clinical Neurosciences, Cumming School of Medicine University of Calgary  Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jan","family":"Storek","sequence":"additional","affiliation":[{"name":"Division of Hematology, Department of Medicine, Cumming School of Medicine University of Calgary  Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liam","family":"Quartermain","sequence":"additional","affiliation":[{"name":"Transplant and Cell Therapy Program, Division of Hematology, Department of Medicine The Ottawa Hospital  Ottawa Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher","family":"Hahn","sequence":"additional","affiliation":[{"name":"Division of Neurology, Department of Clinical Neurosciences, Cumming School of Medicine University of Calgary  Calgary Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. 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