Kozák T, Havrdová E, Pit'ha J, Gregora E, Pytlík R, Maaloufová J, Marecková H, Kobylka P, Vodvárková S
Department of Clinical Haematology, University Hospital Královské Vinohrady, Prague, Czech Republic.
Bone Marrow Transplant. 2000 Mar;25(5):525-31. doi: 10.1038/sj.bmt.1702180.
High-dose immunoablative chemotherapy with autologous haematopoietic cell support might be beneficial in the treatment of intractable forms of MS. We mobilised PBPC in 11 patients with secondary progressive MS and finally eight patients were grafted after high-dose BEAM chemotherapy with either in vitro or in vivo T cell depletion. Median EDSS and SNRS scores at the time of inclusion were 6.5 (6.5-7.5) and 56 (44-65), respectively. PBPC mobilisation was safe with no serious adverse effects, and without significant aggravation of disability. One patient improved significantly (by 1.0 point on EDSS) after the mobilisation. Two mobilisation failures were observed. No life-threatening events occurred during the transplantation. All grafted patients, except one, at least stabilised their disability status. One patient improved significantly (by 1.5 points on EDSS), two patients improved slightly (by 0.5 points on EDSS), one patient worsened by 1.0 point on the EDSS in 10 months. Improvement occurred with a delay of 2-4 months. Median EDSS and SNRS of grafted patients at the last follow up were 6.5 (5.5-8.5) and 64 (39-73), respectively with median follow-up of 8.5 months. Further follow-up is needed to determine the disease course after complete immune reconstitution. Bone Marrow Transplantation (2000) 25, 525-531.
大剂量免疫清除化疗联合自体造血细胞支持可能对治疗难治性多发性硬化症有益。我们对11例继发进展型多发性硬化症患者进行了外周血造血干细胞动员，最终8例患者在接受大剂量BEAM化疗并进行体外或体内T细胞清除后接受了移植。纳入时的EDSS中位数和SNRS评分分别为6.5（6.5 - 7.5）和56（44 - 65）。外周血造血干细胞动员是安全的，没有严重不良反应，也没有导致残疾显著加重。1例患者在动员后显著改善（EDSS评分提高1.0分）。观察到2例动员失败。移植过程中未发生危及生命的事件。除1例患者外，所有接受移植的患者至少稳定了其残疾状态。1例患者显著改善（EDSS评分提高1.5分），2例患者略有改善（EDSS评分提高0.5分），1例患者在10个月内EDSS评分恶化1.0分。改善在2 - 4个月后出现。末次随访时移植患者的EDSS中位数和SNRS分别为分别为6.5（5.5 - 8.5）和64（39 - 73），中位随访时间为8.5个月。需要进一步随访以确定完全免疫重建后的疾病进程。《骨髓移植》（2000年）第25卷，第525 - 531页 。
