Department of Hematology, Xuan Wu Hospital, Capital Medical University, No. 45 Changchun Street, Xuan Wu District, Beijing, China.
Ann Hematol. 2011 Mar;90(3):343-8. doi: 10.1007/s00277-010-1071-5. Epub 2010 Sep 25.
To evaluate clinical outcomes of autologous peripheral blood stem cell transplantation (APBCST) between opticospinal multiple sclerosis (OSMS) and conventional multiple sclerosis (CMS) during disease progressive stage in a Chinese population. Thirty-six secondary progressive MS patients, among whom 21 were with OSMS and 15 with CMS, underwent APBSCT and were followed up for an average of 48.92 months (range, 10-91 months). Peripheral blood stem cells were obtained by leukapheresis after mobilization with granulocyte colony-stimulating factor. Modified BEAM conditioning regimen (Tiniposide, melphalan, carmustin, and cytosine arabinoside) were administered. Outcomes were evaluated using the expanded disability status scale (EDSS). No maintenance treatment was administered if there was no disease progression. No treatment-related mortality occurred. Among the 36 patients, one OSMS patient dropped during the follow-up. Among the 22 relapse-free patients, 20 were with continuous neurological improvement without any relapse events, and two remained in neurologically stable states. Among the 13 relapse patients, seven had experienced of neurological relapse, but with no progression during the follow-up period; and six experienced neurological deterioration after transplantation and needed further immunosuppressant treatment. The confirmed relapse-free survival rate was 62.9% and progression-free survival rate was 83.3% after 91 months according to Kaplan and Meier survival curves. Eleven of the 20 OSMS patients (55%) and two of the 15 CMS patients (13.3%) stayed in disease active group (P = 0.014). For the 20 OSMS patients, the overall EDSS score decreased significantly after transplantation (P = 0.016), while visual functions had no significant improvement (P = 0.716). Progressive OSMS has a higher relapse rate than CMS following APBSCT.
评估中国人群中活动期视神经脊髓型多发性硬化（OSMS）和普通型多发性硬化（CMS）患者接受自体外周血造血干细胞移植（APBSCT）的临床转归。36 例进展期 MS 患者接受 APBSCT 治疗，其中 21 例为 OSMS，15 例为 CMS，平均随访 48.92 个月（10-91 个月）。采用粒细胞集落刺激因子动员后采集外周血造血干细胞。采用改良的 BEAM 预处理方案（替尼泊苷、马法兰、卡莫司汀和阿糖胞苷）。采用扩展残疾状态量表（EDSS）评估转归。无疾病进展时不给予维持治疗。无治疗相关死亡事件发生。36 例患者中，1 例 OSMS 患者失访。22 例无复发患者中，20 例持续神经功能改善，无复发事件，2 例保持神经稳定状态。13 例复发患者中，7 例有神经复发，但随访期间无进展；6 例在移植后出现神经功能恶化，需要进一步免疫抑制治疗。Kaplan-Meier 生存曲线显示，91 个月时无复发生存率为 62.9%，无进展生存率为 83.3%。20 例 OSMS 患者中有 11 例（55%）和 15 例 CMS 患者中有 2 例（13.3%）仍处于疾病活动组（P=0.014）。20 例 OSMS 患者中，移植后 EDSS 总评分显著下降（P=0.016），而视力无明显改善（P=0.716）。APBSCT 后，进展性 OSMS 的复发率高于 CMS。
