非清髓预处理后自体外周血造血干细胞移植治疗难治性重症肌无力远期疗效观察

Observation of long-term efficacy on patients with refractory myasthenia gravis treated by autologous peripheral blood stem cell transplantation with nonmyeloablative preconditioning regimen

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 study the long-term efficacy of autologous peripheral blood stem cell transplantation with nonmyeloablative preconditioning regimen for refractory myasthenia gravis （MG）. Methods We reviewed 5 cases of refractory MG treated by autologous peripheral blood CD34＋ cell transplantation in our hospital from February 2005 to February 2010. The FAC conditioning regimen included fludarabine 30 mg · m-2 · d-1 ×4 d; antithymocyte globulin 2.5 mg · kg-1 · d-1 ×4 d or antilymphocyte globulin 30 mg · kg-1 · d-1 ×4 d; Cyclophosphamide 50-60 mg· kg-1 · d-1 ×2 d. Results All patients achieved hematopoietic reconstitution and immune reconstitution with no transplantation-related death that occurred within 100 d after transplantation. The median follow-up time was 44 （24 - 84） months. Muscle strength of 4 patients was normal without medication, but 1 patient with palindromia at 12 months after transplantation accepted low dose of prednisone and pyridostigmine as a maintenance treatment and was back to normal life with stable condition. Conclusion Autologous peripheral blood CD34＋ cell transplantation with FAC conditioning regimen for refractory MG has favorable long-term efficacy.