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

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

目的评价异基因造血干细胞移植（allo-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80．0％，难治性贫血伴有原始细胞过多（RAEB）-Ⅰ／Ⅱ组为81．0％，急性髓性白血病（AML）组为56．2％（P〉0．05）。以国际预后积分系统（1PSS）分组显示DFS在低危组为80．0％，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87．0％，5％～20％组为65．5％，〉20％组为75．O％（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60．0％，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。

Objective 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） was calculated by Kaplan-Meier analysis. Results Total DFS rate was 75.3%. The relapse rate was 20%. DFS rates in RA/RAS/Sq-, 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 haploidentical 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 clinical study is needed to evaluate the clinical outcomes of allo-HSCT in MDS.