自体外周血造血干细胞移植治疗进展型多发性硬化的临床研究

Clinical study of antologous peripheral blood stem cell transplantation on progressive multiple sclerosis

目的观察自体外周血造血干细胞移植(APBSCT)治疗进展型多发性硬化(PMS)的疗效及安全性。方法16例进展型多发性硬化患者接受APBSCT治疗,造血干细胞动员采用环磷酰胺/粒细胞集落刺激因子(CY/GCSF)方案,预处理采用环磷酰胺/全身放疗(CY/TBl)或卡氮芥、足叶乙甙、阿糖胞苷和马法兰(BEAM)方案。采用无进展生存率和无复发生存率进行疗效评估,并记录移植、随访期间的毒副作用。结果中位数随访时间24个月,无进展生存率为80%,无事件生存率为50%。常见的不良反应有恶心、呕吐、感染、脱发、一过性转氨酶升高、短暂性神经症状加重,2例患者于移植后4.5个月和15个月分别死于严重肺部感染和不明原因的进行性黄疸。结论APBSCT治疗可明显阻止PMS患者的病情进展,其长期疗效和安全性仍需进一步随访观察。

Objective To observe the effects and toxicities of autologous peripheral blood stem cell transplantation (APBSCT) on progressive multiple sclerosis (PMS).Methods Sixteen patients suffering from PMS were treated with APBSCT. Autologous peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte-colony-stimulating factor(CY/G-SCF). CY/TBI (cyclophosphamide and total body irradiation) or BEAM (BCNU, etoposide, arabinosylcytosine, melphalan) was used as conditioning regimen. The probabilities of progression-free survival and the event-free survival were used to assess the effects and adverse experiences were recorded to detect the toxicities of APBSCT. Results The median follow-up time was 24 months. The probabilities of progression-free survival and the event-free survival wer 80%and 50%, respectively. The principal adverse events included nausea, vomiting, infections, trichomadesis, transient and mild abnormal liver enzymes and neurotoxicities. Two patients died due to severe infection and jaundice, respectively. Conclusions APBSCT appears feasible in PMS. However, more patients and longer follow-up would be required.