异基因造血干细胞移植治疗骨髓增生异常综合征60例疗效分析

目的 评价异基因造血干细胞移植(silo-HSCT)治疗骨髓增生异常综合征(MDS)的疗效.方法 回顾性分析2001年8月-2009年2月在北京市道培医院接受allo-HSCT治疗的60例MDS患者.同胞相合移植采用马利兰+环磷酰胺/氟达拉滨预处理方案,非亲缘、单倍体移植采用马利兰+环磷酰胺/氟达拉滨+兔抗人胸腺细胞免疫球蛋白预处理方案.移植物抗宿主病(GVHD)预防采用环孢素A(CsA)、短程甲氨蝶呤(MTX)和霉酚酸酯(MMF)方案.采用Kaplan-Meier曲线计算无病生存率(DFS),率的比较采用Log-rank检验.结果 总体DFS为75.3%,复发率为20%;以WHO(2001年)分组显示DFS率在难治性贫血(RA)/难治性贫血伴环状铁粒幼细胞贫血(RARS)/5q-组为84.6%,难治性细胞减少伴有多系发育异常(RCMD)组为80.O%,难治性贫血伴有原始细胞过多(RAEB)-Ⅰ/Ⅱ组为81.O%,急性髓性白血病(AML)组为56.2%(P>0.05).以国际预后积分系统(IPSS)分组显示DFS在低危组为80.O%,中危-Ⅰ组为84.6%,中危-Ⅱ组为81.8%,高危组为65.4%(P>0.05).以移植前骨髓原始细胞百分比分组显示DFS在<5%组为87.O%,5%～20%组为65.5%,>20%组为75.0%(P>0.05).以移植类型分组显示DFS同胞相合组为79.2%,非血缘组为60.O%,单倍型组为76.9%(P>0.05),上述分组比较均未有统计学意义.结论 allo-HSCT治疗各种类型的MDS均获得较高的DFS,因此可作为MDS的一线治疗.非血缘移植以及单倍体移植治疗MDS疗效显著,因而在缺乏同胞相合供者时,可选择非血缘或单倍型供者.此外,除转化为明显的白血病患者,移植前的化疗不是必需的.但是评价allo-HSCT治疗MDS的影响因素尚需更大规模病例的临床研究.

Effects of allogeneic hematopoietic stem cell transplantation on 60 patients with myelodysplastic syndrome

0bjective To evaluate the clinical outcome of all-ogeneic hematopoietic stem cell transplantation (all-HSCT) for myelodysplastic syndrome (MDS). Methods From March 2001 to February 2009,60 patients with MDS underwent allo-HSCT in our hospital were enrolled in this study. The conditioning regimens were Myleran (BU)/Cyclophosphamide (Cy) or Flu for identical sibling HSCT, and BU/Cy or Flu plus anti-thymocyte globulin (ATG) for haploidentical and unrelated HSCT. Cyclosporine A and short-course MTX were used for graft-versus-host disease (GVHD) prophylaxis. Diseased free survival (DFS) wag calculated by Kaplan-Meier analysis. Results Total DFS rate was 75.3%., The relapse rate was 20%. DFS rates in RA/RAS/5q-, RCMD, RAEB-Ⅰ/RAEB-Ⅱ and acute myelocytic leukemia (AML) subgroups were 84.6%,80.0%,81.0%,56.2%, respectively(P>0.05).DFS rates in IPSS low risk group, intermediate-Ⅰ risk group, intermediate-Ⅰ risk group and high risk group were 80.0%, 84.6%, 81.8% and 65.4%, respectively (P>0.05). DFS rates for allo-HSCT from identical sibling. unrelated or hapioidentical donors were 79.2%, 60.0%, 76.9%, respectively(P=0.028).DFS rates for percentages of blasts in bone marrow pre-transplant were 87.0%, 65.5%, 75.0% in <5% blasts. 5%-20% blasts, >20% blasts subgroups, respectively (P>0.05). Conclusions Since favorable clinical outcomes have been seen in all kinds of MDS by allo-HSCT, HSCT should be the first-line therapy for MDS. No significant differences are found based on different stem cell donors and the percentages of bone marrow blasts pre-HSCT, unrelated or haploidentical donors should be important alternatives if there is no identical sibling available. Chemotherapy before transplantation is not necessary except overt acute leukemia. A larger chinical study is needed to evaluate the clinical outcomes of allo-HSCT in MDS.