Bryant A, Atkins H, Pringle CE, et al. Myasthenia Gravis Treated with Autologous Hematopoietic Stem Cell Transplantation. JAMA Neurol 2016;73:652-658.
Submitted by Niranjan N. Singh, MD, News Science Editorial Board
The authors report a series of seven patients (two of whom were seronegative) with severe myasthenia gravis treated with autologous hematopoietic stem cell transplantation. Patients were refractory to treatment with multiple treatment modalities including pyridostigmine, corticosteroids, azathioprine, mycophenolate mofetil, cyclosporine, intravenous immunoglobulin, plasma exchange, and thymectomy. The protocol included graft mobilization and purification, treatment with intensive conditioning chemotherapy, and graft reinfusion. All patients achieved complete stable remission with no residual myasthenia gravis symptoms or requirement for disease-modifying therapy.
Refractory myasthenia gravis accounts for 10% of disease. Hematopoietic stem cell transplantation offers a potential treatment option for patients in this group. It should be emphasized that this study is a retrospective case series, and that stem cell transplantation has not been studied in a prospective fashion in patients with myasthenia gravis.
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