自体外周造血干细胞移植后对多发性硬化患者MRI强化病灶的影响

Autologous peripheral blood stem cell transplantation suppresses Gd-enhanced MRI activity in progressive multiple sclerosis

目的 评价自体外周造血干细胞移植术对进展型多发性硬化患者 MRI强化病灶的抑制作用、临床神经功能改善情况和毒副作用。方法 应用自体外周造血干细胞移植治疗进展型多发性硬化患者 10例 ,用惠尔血进行造血干细胞动员 ,预处理应用 BEAM方案 (卡氮芥、依托泊苷、阿糖胞苷、马法兰 ) ,移植前 1、2个月及移植后每间隔 3个月分别进行脑、脊髓的 MRI及强化扫描 ,同时评价扩充神经功能残疾量表 ( EDSS)。结果 中位随访时间 10个月 (范围 4～ 2 2月 )。移植后 1、3、6、9、12、15、18个月 MRI强化病灶数较移植前均明显减少 ,差异有显著意义 ( P<0 .0 1) ;移植后患者 EDSS评分较移植降低 ,差异有显著意义 ( P<0 .0 1)。造血干细胞动员、预处理和移植期间无 1例死亡 ,常见的副反应为发热感染等 ;2例患者在 3～ 6个月神经功能一过性加重 ,1例在 10个月后复发。结论 自体外周造血干细胞移植术对进展型多发性硬化患者 MRI强化病灶有明显的抑制作用 ,同时临床神经功能得到改善 ,无严重毒副作用 。

Objective To determine in progressive multiple sclerosis the effect of autologous peripheral blood stem cell transplantation(APBSCT)on gadolinium(Gd)-enhanced MRI and to obtain information on clinical course and safety.Methods 10 patients with progressive MS were transplanted,after BEAM conditioning regimen(carmustine,etoposide,cytosine-arabinoside,melphalan),with APBSCT mobilized with granulocyte-colony-stimulating factor(G-CSF).Gd-enhanced MRI scans were performed monthly for a pretreatment period of 2 months and compared with serial Gd-enhanced MRI once every 3 months for posttreatment following-up.At same time,EDSS was used for evaluating clinicalneurological function.Results The median follow-up is now 10 months(range 4 to 22 months).The number of Gd-enhancing MRI lesions decreased immediately after APBSCT and finally dropped to zero in 9 cases after the conditioning regimen.The mean EDSS rating scores of 10 subjects in one year after APBSCTwas significantly decreased( P <0.01) than before. Vomiting and nausea and infections were the principal toxic complications.Mild and transient worsening ofneurological signs were observed in 2 patients in the post-transplant period.One patient relapsed in 10th month.Conclusions These results demonstrate that the APBSCT has the capacity to significantly suppress MRI-enhancing activity and the effect is sustained with time.The final impact of this procedure on disease course remains to be long-term followed up.