自体造血干细胞移植治疗继发进展型多发性硬化36例疗效及预后影响因素分析

目的分析继发进展型多发性硬化(SPMS)患者接受自体造血干细胞移植(auto-HSCT)疗效及预后因素分析。方法收集2001—2009年首都医科大学宣武医院收治的36例SPMS患者。粒细胞集落刺激因子(G-CSF)5μg/kg皮下注射4~6 d动员造血干细胞,采集外周血单个核细胞,其中28例患者分选CD34+细胞。预处理方案为BEAM(卡氮芥、马法兰、替尼泊甙及阿糖胞苷)。结果15例临床缓解,5例疾病稳定,14例移植后复发,2例失访。88个月预期无复发存活率55%。发病时有视神经损伤是移植预后不良的危险因素。结论auto-HSCT是SPMS患者获得长期疾病缓解的有效方法,视神经损伤是移植预后不良因素。

Autologous peripheral hematopoietic stem cell transplantation for treatment of multiple sclerosis in 36 cases:efficacy and prognostic factors analysis.

Objective To investigate the efficacy and prognostic factors of autologous peripheral hematopoietic stem cell transplantation(auto-HSCT) for treatment of secondary progressive multiple sclerosis(SPMS).Methods A total of 36 patients received subcutaneous injection with granulocyte colony-stimulating factor(G-CSF) for 4~6 days to mobilize hematopoietic stem cells and were then collected for peripheral blood mononuclear cells(PBMCs).CD34+ cell sorting was performed in 28 patients.The preparative regimen was BEAM(carmustine,melphalan,teniposide and cytosine arabinoside).Results Clinical improvement was shown in 15 cases and stability in 5 cases.14 cases recurred after auto-HSCT,Two cases were lost to follow-up.The expected relapse free survival(RFS) was 55% at 88 months.In addition,optic nerve injury at the onset of MS was considered as a risk factor for unfavorable outcome of auto-HSCT.Conclusion Auto-HSCT is effective for long-term remission in patients with SPMS.Optic nerve injury may be a risk factor for unfavorable prognosis of auto-HSCT.