干细胞移植治疗骨髓增生异常综合征13例

The effect of hematopoietic stem cell transplantation on myelodysplastic syndrome

目的 探讨干细胞移植（HSCT）在骨髓增生异常综合征（MDS）治疗中的疗效以及预处理方式的选择.方法 对13例MDS患者行异基因造血干细胞移植（allo-HSCT）（包括HLA配型全相合10例、半相合2例、脐血移植1例）.输注单个核细胞（MNC）6.92（2.65～21.33）×10^8/kg,CD34细胞4.47（1.49～10.22）×10^6/kg.其中,5例选择全身照射＋氟达拉滨＋环磷酰胺（TBI＋Flud＋Cy）方案预处理,3例白消安（BU）/Cy预处理,3例TBI＋CY,2例采用阿糖胞苷（Ara-C）＋BU＋Cy＋替尼泊苷（VM26）预处理.移植物抗宿主病（GVHD）的预防：2例HLA配型半相合者给予抗胸腺细胞球蛋白（ATG）联合环孢素A（CsA）加短程甲氨蝶呤（MTX）治疗,并于移植后1～28 d持续给予霉酚酸酯（MMF）,其他病例仅给予CsA加短程MTX.结果 13例患者中9例造血完全重建,移植相关死亡4例.结论 HSCT是可以治愈MDS的有效方法.预处理选择应采取个体化.

Objective To investigate the curative effect of allogeneic hematopoietic stem cell transplantation （allo-HSCT） and the method of the pretreatment choice on myelodysplastic syndrome （MDS）.Methods 13 cases who were undergoing allo-HSCT including 10 HLA-matched, 2 HLA partially matched,and one umbilical cord blood transplanted patients were enrolled in this study. The infusion number of MNC and CD34^＋ cells is 6.92 （2.65-21.33）×10^8/kg and 4.47 （1.49-10.22） ×10^6/kg, respectively. Of the 13 cases,5 adopted TBI, fludarabine（Flud）, and cyclophosphamide（Cy） pretreatment, 3 chose BU/Cy pretreatment, and 3 chose TBI and Cy pretreatment, 2 chose Ara-C＋BU＋Cy＋VM26 pretreatment. In order to prevent GVHD, 2cases of HLA partially matched were treated with ATG, cyclosporin A （CsA）, MTX and MMF aftertransplantation, while the others were treated with CsA and MTX only. Results 9 out of 13 cases achieved hematopoietic reconstruction completely. 4 died of complications. Conclusion The allogeneic hematopoietic stem cell transplantation is an efficient regimen to cure MDS. The pretreatment should adopt the individuation.