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目的观察自体外周造血干细胞移植(APBSCT)治疗进展型多发硬化(PMS)的疗效及安全性。方法对2001-09~2004-07对首都医科大学宣武医院13例PMS患者进行了自体外周造血干细胞移植,单独使用粒细胞集落刺激因子(G-CSF)动员造血干细胞,7名患者进行了采集物的CD3+4细胞纯化。预处理采用BEAM(卡氮芥、依托泊甙、阿糖胞苷、马法兰)方案。中位随访期为22(3~36)个月,移植前后应用扩充神经功能残疾量表(EDSS)、年平均发病次数进行疗效评价。结果患者移植后18个月EDSS评分较移植前降低[移植后(4.05±0.66),移植前(6.00±0.30),P<0.05],年平均发病次数减少[移植后(0.40±0.15),移植前(1.63±0.18),P<0.01]。移植后3年疾病无活动存活率为(63.64±14.50)%,EDSS评分无进展存活率为(72.73±13.43)%。无移植相关死亡,造血功能恢复迅速,未出现严重的毒性反应及并发症。结论APBSCT治疗PMS安全有效
Objective To observe the efficacy and safety of autologous peripheral blood stem cell transplantation (APBSCT) in the treatment of advanced multiple sclerosis (PMS). Methods Thirteen patients with PMS at Xuanwu Hospital of Capital Medical University underwent autologous peripheral blood stem cell transplantation from 2001-09 to 2004-07. Hematopoietic stem cells were mobilized by granulocyte colony-stimulating factor (G-CSF) alone. Seven patients were collected Of CD3 + 4 cells. Pretreatment BEAM (Carmustine, etoposide, cytarabine, melphalan) program. The median follow-up period was 22 months (3-36 months). The EDSS was used before and after transplantation, and the average annual incidence was evaluated. Results The EDSS score at 18 months after transplantation was lower than that before transplantation [4.05 ± 0.66 after transplantation (6.00 ± 0.30), P <0.05], and the average annual incidence was decreased [after transplantation (0.40 ± 0.15) Before (1.63 ± 0.18), P <0.01]. Three years after transplantation, the disease-free survival rate was (63.64 ± 14.50)%, and the EDSS progression-free survival rate was (72.73 ± 13.43)%. No transplant-related death, rapid recovery of hematopoietic function, no serious toxicity and complications. Conclusion APBSCT is safe and effective in the treatment of PMS