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目的:评价自体外周造血干细胞移植(APBSCT)治疗继发进展型多发性硬化(MS)的疗效及安全性。方法:回顾性分析2001-2010年在首都医科大学宣武医院接受APBSCT治疗的41例继发进展型MS患者的临床资料。女性31例,男性10例,年龄24～56岁,中位年龄36岁。所有患者连续5d皮下注射粒细胞集落刺激因子(G-CSF)5μg/kg动员造血干细胞。应用细胞分离机采集外周血单个核细胞(PBMC),对28例患者应用CliniMACS免疫磁珠系统分选CD34+细胞,将PBMC或CD34+细胞置-80℃冰箱冻存。患者采用BEAM[becenum(camustine);etoposide(teniposide);cytosine arabinoside;melphalan]预处理方案:卡莫司汀300mg/m2×1d;替尼泊苷150mg/m2×4d;阿糖胞苷200mg/m2×4d;美法仑140mg/m2×1d。预处理后经静脉回输低温保存复苏的自体CD34+细胞或PBMC。应用扩充神经功能残疾量表评判患者APBSCT后神经系统功能的恢复程度,根据不良事件常用术语标准3.0版评判APBSCT的不良反应。结果:2例患者失访。39例患者治疗后随访6～100个月,平均51个月,其中16例缓解,8例稳定,15例移植后复发且病情进展。100个月累计疾病无进展生存率为55.5%。预处理后41例患者的中性粒细胞均<0.5×109/L,血小板均<25×109/L。经支持治疗后所有患者均获得造血重建;37例发生腹泻;3例发生替尼泊苷过敏反应;26例发生感染,其中发热性中性粒细胞减少19例,会阴肛门周围皮肤软组织脓肿2例,败血症2例,肺部真菌感染、静脉置管处皮肤感染和消化道感染各1例;6例发生植入综合征;无移植相关死亡。结论:APBSCT是治疗继发进展型MS较安全有效的方法,但使用期间应严密观察可能出现的不良反应。
Objective: To evaluate the efficacy and safety of autologous peripheral blood stem cell transplantation (APBSCT) in the treatment of secondary progressive multiple sclerosis (MS). Methods: The clinical data of 41 patients with MS who underwent APBSCT at Xuanwu Hospital, Capital Medical University from 2001 to 2010 were retrospectively analyzed. 31 females, 10 males, aged 24 to 56 years old, the median age of 36 years. All patients underwent subcutaneous injection of 5μg / kg granulocyte colony-stimulating factor (G-CSF) for 5 days to mobilize hematopoietic stem cells. Peripheral blood mononuclear cells (PBMCs) were collected using a cell separator, CD34 + cells were sorted by CliniMACS immunomagnetic beads system in 28 patients, and PBMC or CD34 + cells were stored in -80 ℃ freezer. Patients were treated with BEAM [becenum (camustine); etoposide (teniposide); cytosine arabinoside; melphalan] Pretreatment regimen: Carmustine 300mg / m2 x 1d; Teniposide 150mg / m2 x 4d; Cytarabine 200mg / m2 × 4d; melphalan 140mg / m2 × 1d. After preconditioning, resuscitated autologous CD34 + cells or PBMCs were cryogenically transfused intravenously. The degree of recovery of the nervous system function after APBSCT was evaluated by the extended neurological disability scale. The adverse reactions of APBSCT were evaluated according to the 3.0 standard version of the commonly used terminology standard of adverse events. Results: Two patients were lost. Thirty-nine patients were followed up for 6 to 100 months with an average of 51 months, of which 16 were relieved, 8 were stable, and 15 were relapsed after transplantation. 100 months cumulative disease progression-free survival rate was 55.5%. After pretreatment of 41 patients with neutrophils were <0.5 × 109 / L, platelets were <25 × 109 / L. All patients underwent hematopoietic reconstitution after receiving supportive care; 37 cases developed diarrhea; 3 patients had an allergic reaction to teniposide; 26 patients had an infection, including 19 cases of febrile neutropenia, 2 cases of skin and soft tissue abscess around perineal anus , 2 cases of sepsis, 1 case of pulmonary fungal infection, skin infection of the venous catheter and alimentary canal infection, 6 cases of implantation syndrome and no graft-related death. Conclusion: APBSCT is a safe and effective method for the treatment of secondary progressive MS. However, adverse reactions should be closely observed during use.