自体造血干细胞移植治疗进展型多发性硬化的疗效评价  

The evaluation of curative effect of autologous hematopoietic stem cell transplantation in treatment of patients with progressive multiple sclerosis

作　　者：冯楠[1] 张普[1] 董会卿[1] 徐娟[2] 苏力[2] 

机构地区：[1]首都医科大学宣武医院神经内科,北京100049 [2]首都医科大学宣武医院血液科,北京100049

出　　处：《脑与神经疾病杂志》2011年第6期425-430,共6页Journal of Brain and Nervous Diseases

基　　金：北京市教育委员会科技发展计划面上项目(KM200910025015)

摘　　要：目的评价自体造血干细胞移植(AHSCT)治疗进展型多发性硬化(PMS)的疗效及安全性。方法选取2001年11月至2010年3月我院收治的82例PMS患者,行AHSCT治疗的41例为AHSCT组,行皮质醇激素治疗的41例PMS患者为激素治疗组。随访观察临床疗效,分析指标包括疾病无进展生存率(PFS)、神经功能残疾评分(EDSS)、总发作次数、平均年发作次数、生活质量评分(ADL)和MRI病灶变化情况、移植相关死亡率及毒副反应。结果 AHSCT组41例患者均完成了AHSCT,但3例失访,38例完成临床试验。其移植后EDSS评分、发作总次数、年均发作次数、病灶总容积及生活质量评分均较移植前下降(<0.05);5.2年PFS为78.9%。激素治疗组41例患者失访29例,随访12例患者治疗后EDSS评分、发作总次数及年均发作次数均较治疗前增加(P<0.05);2.8年PFS仅为8.3%。结论 PMS应用其他方法治疗效果欠佳时,采取自体造血干细胞移植,可有效降低EDSS评分、发作次数及病灶容积,提高生活质量。Objective To evaluate the therapeutic effect and the safety of autologous haematopoietic stem cell transplantation in progressive multiple sclerosis. Methods A retrospective analysis of 82 patients with PMS who were treated between November 2001 and March 2010 were carried out in our hospital. 41 cases with AHSCT therapy were defined as test group, and another 41 cases with eortisol hormone therapy were defined as control group. Compared their therapeutic effect by observing progression-free survival, EDSS, total numbers of relapses, the per capital average annal number of relapses, ADL, image changes of MRI, transplantation related mortality and toxic effect. Results 38 of 41 patients completed the trial in test group as planned. The results showed that there were significant differences（ all P 〈 0. 05 ）after AHSCT in test group with lower mean EDSS, total nmnberof relapses, the per capital average annal number of relapses, total focus volume and mean ADL. And the PFS of 5.2 years is 78.9% 29 in 41 patients were lost in control group and we observed a higher mean EDSS, tolal number of relapses and the per capital average annal number of relapses than before（ P 〈 0.05 ） in the other 12 cases. And the PFS of 2. $ years was only 8.3%. Conclusion AHSCT can bring PMS patients a better daily life with lower EDSS, h）wer number of relapses and smaller lesion volume for those who do not response well to other treatmeut methods.

关 键 词：进展型多发性硬化 自体造血干细胞移植 临床疗效 

分 类 号：R774.51[医药卫生—眼科]