{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,15]],"date-time":"2026-01-15T04:35:21Z","timestamp":1768451721331,"version":"3.49.0"},"reference-count":36,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2023,9,19]],"date-time":"2023-09-19T00:00:00Z","timestamp":1695081600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100003157","name":"Myasthenia Gravis Foundation of America","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100003157","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100016650","name":"Rare Diseases Clinical Research Network","doi-asserted-by":"publisher","award":["U54\u2010NS115054"],"award-info":[{"award-number":["U54\u2010NS115054"]}],"id":[{"id":"10.13039\/100016650","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>Myasthenia gravis (MG) is an autoantibody\u2010mediated neuromuscular junction disorder involving the acetylcholine receptors on the motor endplate. The safety and response to high\u2010dose chemotherapy (HDIT) and autologous hematopoietic cell transplantation (HCT) were assessed in a patient with severe refractory MG.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>As part of a pilot study of HDIT\/HCT for patients with treatment\u2010resistant autoimmune neurological disorders, a patient with severe refractory MG underwent treatment. After mobilization of hematopoietic stem cells with rituximab, prednisone, and G\u2010CSF, the patient had HDIT consisting of carmustine, etoposide, cytarabine, melphalan, and rabbit antithymocyte globulin, followed by autologous HCT. The effect of treatment on the autoantibody to the acetylcholine receptor (AChR) was assessed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The patient had been diagnosed with AChR antibody\u2010positive MG 14\u2009years before HDIT\/HCT and had failed thymectomy, therapeutic plasma exchange, and multiple immunomodulatory agents. The Myasthenia Gravis Foundation of America (MGFA) clinical classification was IVb before HDIT\/HCT. She tolerated HDIT\/HCT well and started to improve clinically within days of treatment. At both 1 and 2\u2009years after HDIT\/HCT, patients remained symptom\u2010free. After HDIT\/HCT, AChR\u2010binding autoantibodies persisted, and the relative frequency of immune cell subtypes shifted.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>HDIT\/HCT induced a complete response of disease activity in a patient with severe refractory MG. This response may suggest that a cell\u2010mediated etiology may be a significant contributing factor in refractory MG cases. A phase 2 clinical trial is warranted to establish if HDIT\/HCT can be an effective therapy for severe refractory MG and to gain a further understanding of disease pathogenesis.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.51898","type":"journal-article","created":{"date-parts":[[2023,9,20]],"date-time":"2023-09-20T04:57:20Z","timestamp":1695185840000},"page":"2105-2113","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Remission of severe myasthenia gravis after autologous stem cell transplantation"],"prefix":"10.1002","volume":"10","author":[{"given":"Monica I.","family":"Schlatter","sequence":"first","affiliation":[{"name":"Colorado Blood Cancer Institute  Denver Colorado 80218 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Soumya S.","family":"Yandamuri","sequence":"additional","affiliation":[{"name":"Department of Neurology Yale University School of Medicine  New Haven Connecticut 06520 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