【摘 要】
：
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APBSCF治疗,造血干细胞动员采用环磷酰胺/粒细胞集落
【机 构】
：
南京大学医学院附属鼓楼医院神经内科,南京,210008;南京大学医学院附属鼓楼医院血液科,南京,210008
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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APBSCF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/TBI)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.
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