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目的 评价自体外周造血干细胞移植术 (APBSCT)治疗进展型多发性硬化的疗效及其安全性和毒性。方法 移植组进展型多发性硬化患者 10例 ,造血干细胞动员应用惠尔血 ,预处理应用卡氮芥、依托泊苷、阿糖胞苷、马法兰 (BEAM方案 ) ,移植前采用CD+ 3 4 纯化和未纯化两种方法处理。对照组进展型多发性硬化患者 10例 ,应用糖皮质激素或免疫抑制剂治疗。中位随访时间 10个月 (范围 4～ 2 2个月 )。应用扩充神经功能残疾量表 (EDSS)、年平均发作次数和MRI进行疗效评价。结果移植组患者移植后 12个月EDSS评分较对照组降低 (分别为 3 4 1± 0 2 3和 6 31± 1 2 1,P <0 0 1) ,平均年发作次数降低 (分别为 0 37± 0 0 7和 1 83± 0 4 2 ,P <0 0 1)。移植组患者移植后MRI强化病灶数较移植前均减少 (P <0 0 1) ;移植前CD+ 3 4 纯化者较未纯化者治疗更有效 (P <0 0 1)。动员、预处理和移植期间无一例死亡 ,常见的不良反应为胃肠道反应和感染 ;在移植后 2例患者有一过性轻度神经功能损害 ,1例在 10个月时复发。结论 APBSCT治疗进展型MS患者近期治疗效果是明显的 ,安全性是可靠的 ,长期疗效仍需进一步随访观察。
Objective To evaluate the efficacy, safety and toxicity of autologous peripheral blood stem cell transplantation (APBSCT) for the treatment of multiple sclerosis. Methods Ten patients with progressive multiple sclerosis were enrolled in this study. The hematopoietic stem cells were mobilized and treated with Carvacizumab, etoposide, cytarabine and melphalan (BEAM regimen). CD34 + Unpurified two ways to deal with. Control group of patients with progressive multiple sclerosis in 10 cases, the application of glucocorticoid or immunosuppressive agents. The median follow-up time was 10 months (range 4 to 22 months). The EDSS, the number of annual seizures and MRI were used to evaluate the curative effect. Results The EDSS score at 12 months after transplantation in the transplant group was lower than that in the control group (34 1 ± 0 2 3 and 6 31 ± 1 2 1 respectively, P 0 01), and the average annual number of seizures decreased (0 37 ± 0 0 7 and 1 83 ± 0 4 2, P <0 0 1). The number of MRI enhancement lesion after transplantation in the transplantation group was significantly lower than that before transplantation (P <0.01). Pre-transplant CD + 3 4 purification was more effective than the unpurified treatment (P <0.01). None of the patients died during mobilization, pretreatment and transplantation. Common adverse reactions were gastrointestinal reactions and infections. Two patients had transient mild neurological impairment after transplantation and one patient relapsed at 10 months. Conclusion APBSCT treatment of advanced MS patients with short-term treatment effect is obvious, the safety is reliable, long-term efficacy still need further follow-up observation.