Allogeneic Hematopoietic Cell Transplantation for Refractory Myasthenia Gravis
Allogeneic Hematopoietic Cell Transplantation for Refractory Myasthenia Gravis
复制标题
DOI：
10.1001/archneurol.2009.28
复制
发表时间：
2009-05-01
影响因子：
--
通讯作者：
Horn, Biljana N.
中科院分区：
文献类型：
--
作者：
Strober, Jonathan;Cowan, Morton J.;Horn, Biljana N.
目的：描述1例难治性重症肌无力(MG)患者接受相合同胞外周血干细胞移植治疗。设计：病例报告：患者为17岁男孩，11个月时确诊为重症肌无力，曾接受吡斯的明、静脉注射免疫球蛋白、皮质类固醇、胸腺切除术、硫唑嘌呤、霉酚酸酯、血浆蛋白原、利妥昔单抗和大剂量环磷酰胺等治疗。结果：患者接受了静脉注射白花丹、氟达拉滨和阿罗珠单抗的减毒预适应，随后接受了来自其相合兄弟姐妹的外周血干细胞输注。在移植前，患者经常接受血浆置换、静脉注射免疫球蛋白和吡斯的明。他有眼肌麻痹，口咽和四肢肌肉受累，活动受限。在移植后40个月，他的口咽和骨骼肌无力已经完全消失，他没有服用任何治疗MG的药物，他是一名狂热的运动员。结论：异基因造血干细胞移植后，抗乙酰胆碱受体抗体的存在不足以引起MG的症状。这证实了额外的免疫机制在该病的发病机制中起重要作用。同种异体移植可能是重症难治性MG患者的一种治疗选择。然而，对同种异体移植治疗这种疾病的长期疗效知之甚少，需要进行长期的随访。
Objective: To describe a patient with intractable myasthenia gravis (MG) who was treated with a matched sibling peripheral blood stem cell transplantation.Design: Case report.Patient: A 17-year-old boy with MG diagnosed at 11 months of age who was previously treated with pyridostigmine, intravenous immunoglobulin, corticosteroids, thymectomies, azathioprine, mycophenolate mofetil, plasmaphereses, rituximab, and high-dose cyclophosphamide.Results: The patient underwent a reduced-toxicity conditioning with intravenous busulfan, fludarabine, and alemtuzumab, followed by a peripheral blood stem cell infusion from his HLA-matched sibling. Before transplantation, the patient was receiving frequent plasmaphereses, intravenous immunoglobulin, and pyridostigmine. He had ophthalmoplegia, oropharyngeal and limb muscle involvement, and limited mobility. At 40 months posttransplantation, his oropharyngeal and skeletal muscle weakness has completely resolved, he is not taking any medications for MG, and he is an avid athlete. However, his ophthalmoplegia persists, and his anti-acetylcholine receptor antibody levels remain elevated.Conclusions: Following allogeneic hematopoietic stem cell transplantation, the presence of anti-acetylcholine receptor antibodies was not sufficient for inducing symptoms of MG. This confirms that additional immune mechanisms are important in pathogenesis of this disease. Allogeneic transplantation may be a therapeutic option for patients with severe, refractory MG. However, little is known about the long-term efficacy of allogeneic transplantation for this disease, and long-term follow-up is warranted.