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目的观察体外纯化的自体外周血CD34+细胞移植于经非清髓预处理方案治疗的难治性重症肌无力(MG)患者的远期疗效。方法回顾性分析浙江中医药大学附属第一医院血液科2005年2月至2010年2月期间经纯化的自体外周血CD34+细胞移植治疗5例难治性MG患者资料。采用FAC预处理方案:磷酸氟达拉滨30mg·m-2·d-1,共4d;抗胸腺细胞球蛋白2.5mg·kg-1·d-1或抗淋巴细胞球蛋白30mg·kg-1·d-1,共4d;环磷酰胺50～60mg·kg-1·d-1,共2d。结果 5例患者均获造血、免疫重建,无移植相关死亡。全部患者均随访至2012年2月,中位随访时间44(24～84)个月,其中肌力正常,恢复正常生活、工作,脱离药物治疗4例,另1例移植后12个月因疾病复发而再次应用小剂量泼尼松、溴吡斯的明维持治疗患者目前肌无力症状控制良好,生活自理。结论 FAC预处理方案应用于纯化的自体外周血CD34+细胞移植治疗难治性MG远期疗效较好,患者的耐受性良好,值得进一步临床研究。+
Objective To observe the long-term efficacy of in vitro purified CD34 + cells autologous peripheral blood transplanted in refractory myasthenia gravis (MG) treated with non-myeloablative preconditioning regimen. Methods The data of 5 patients with refractory MG were retrospectively analyzed in the Department of Hematology, the First Affiliated Hospital of Zhejiang University of Traditional Chinese Medicine from February 2005 to February 2010 with purified autologous peripheral blood CD34 + cell transplantation. FAC preconditioning regimen: fludarabine 30 mg · m-2 · d-1 for 4 days, antithymocyte globulin 2.5 mg · kg-1 · d-1 or anti-lymphocyte globulin 30 mg · kg-1 · D-1 for 4 days; cyclophosphamide 50-60 mg · kg-1 · d-1 for 2 days. Results All 5 patients received hematopoietic reconstitution and no transplant-related death. All patients were followed up until February 2012 with a median follow-up time of 44 (24-84) months. Among them, normal muscle strength, normal life, work, and withdrawal from medication were observed in 4 cases. Another case was 12 months after transplantation Recurrence and re-application of low-dose prednisone, pyridostigmine maintenance treatment of patients with current good muscle weakness symptoms, take care of themselves. Conclusion The FAC pretreatment scheme applied to the purification of autologous peripheral blood CD34 + cell transplantation for the treatment of refractory MG long-term effect is good, the patient’s well-tolerated, it is worth further clinical study. +