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

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

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4～6d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、鸟法兰、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。

Objective To investigate the efficacy and prognostic factors of autologous peripheral hematopoietie stem cell transplantation （anto-HSCT） for treatment of secondary progressive multiple sclerosis （SPMS）. Methods A total of 36 patients received subcutaneous injection with granuloeyte colony-stimulating factor （ G- CSF） for 4 - 6 days to mobilize hematopoietie stem cells and were then collected for peripheral blood mononuelear cells （PBMCs）. CD34 ＋ cell sorting was performed in 28 patients. The preparative regimen was BEAM （ earmustine,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.