增强预处理方案的造血干细胞移植对骨髓增生异常综合征的治疗效果

Effectiveness of idarubicin-intensified myeloablastive conditioning regimen in allogeneic peripheral blood stem cell transplantation in 12 patients with myelodysplastic syndromes

目的评价采用去甲氧柔红霉素增强预处理方案的外周血造血干细胞移植对骨髓增生异常综合征的治疗效果。方法2004年8月至2009年7月，采用去甲氧柔红霉素（IDA）＋白消安（Bu）＋环磷酰胺（Cy）的增强预处理方案对12例骨髓增生异常综合征患者进行了外周血造血干细胞移植。具体用法为：IDA15mg／m^2，每天持续静脉滴注20h（移植前12d～移植前10d给予）；Bu0．8mg／kg，每6h静脉滴注1次（移植前6d～移植前4d给予）；Cy1．8g／m^2，静脉滴注（移植前3d～移植前2d给予）。采用环孢素A（CsA）联合短程甲氨碟呤（MTX）预防急性移植物抗宿主病（aGVHD）。结果12例受者均移植成功，对该预处理方案耐受良好。8例受者存活，总存活率为66．7％，无病存活率为58．3％，2例受者原发病复发。以世界卫生组织（WHO）亚型分组和国际预后积分系统（IPSS）分组显示，总存活率的组间比较，差异均无统计学意义。结论采用去甲氧柔红霉素增强预处理方案的外周血造血干细胞移植对治疗骨髓增生异常综合征有效，且复发率低。

Objective To analyze the outcome of idarubicin-intensified myeloablastive conditioning regimen in allogeneic peripheral blood stem cell transplantation （allo-PBSCT） in patients with myelodysplastic syndromes （MDS）. Methods From August 2004 to July 2009, 12 patients with MDS were treated with allo-PBSCT following the idarubicin-intensified conditioning regimen. The conditioning regimen was idarubicin （15 mg/m2 ）, continuous intravenous infusion for 20 h, days -12 to-10; busulfan （0. 8 mg/kg）, intravenous infusion every 6 h, days-6 to-4; cyclophosphamide （1.8 g/m2）, intravenous infusion every 6 h, days -3 to -2; cyclosporine A combined with short-term methotrexate was used for the prophylaxes of acute graft versus host disease （aGVHD）. Results All twelve patients achieved Trilineage engraftment, and were well tolerated to this regimen. Eight patients survived, and the overall survival was 66. 7 %, disease-free survival （DFS） 58. 3 %. Two patients relapsed. OS for neither WHO subgroups nor IPSS subgroups had statistically significant difference. Conclusion Allo-PBSCT with idarubicin-intensified conditioning regimen is an effective treatment with reduction of the relapse rate for MDS patients.