单倍体相合异基因造血干细胞移植治疗白血病

背景：对于无HLA全相合同胞供者的患者，采用单倍体相合造血干细胞移植面临移植物抗宿主病重、移植相关死亡率高的风险，但通过不同的移植模式，将有可能获取相近的疗效。 目的：观察亲缘HLA单倍体相合异基因造血干细胞移植治疗白血病的疗效，并与亲缘HLA全相合异基因造血干细胞移植相比较。 方法：45例白血病患者分为2组。单倍体组移植方式为外周血或联合骨髓干细胞移植，预处理方案为改良白消安与环磷酰胺或加抗胸腺细胞球蛋白，移植物抗宿主病的预防采用环孢素A+甲氨蝶呤+霉酚酸脂；全相合组移植方式为外周血干细胞移植，预处理方案为BuCY，移植物抗宿主病的预防采用环孢素A+甲氨蝶呤。 结果与结论：两组均获得造血重建时间差异无显著性意义。单倍体及全相合组急性移植物抗宿主病的累积发病率分别为73%对52%(P > 0.05)；慢性移植物抗宿主病的累积发病率分别为56%对45%(P > 0.05)；移植相关死亡率分别为36%对17% (P > 0.05)；单倍体组无复发，全相合组复发2例；两组的预计3年累积无病生存率分别为61%对60%(P > 0.05)。结果提示，亲缘单倍体异基因造血干细胞移植的总体疗效与亲缘全相合异基因造血干细胞移植相似，但中重度急性移植物抗宿主病的发生率较后者为高。

HLA haploidentical haematopoietic stem cell transplantation for leukemia

BACKGROUND: For the patients without HLA sibling matched donor, performing HLA haploidentical haematopoietic stem cell transplantation (HSCT) is confronted with problems such as severe graft versus host disease (GVHD) and high transplantation related mortality. Achieving the similar efficiency as sibling matched HSCT is possible by adopting different transplant methods. OBJECTIVE: To analyze the effect of HLA haploidentical haematopoietic stem cell transplantation for leukemia and compare with matched sibling HSCT. METHODS: A total of 45 patients diagnosed as leukemia were divided into two groups. For haploidentical cases, peripheral blood stem cell transplantation with or without combination of bone marrow transplantation was performed. Conditioning regimen included modified busulphan/ cyclophosphamide with or without adding antithymocyte globulin. Cyclosporine A, methotrexate and mycophenolate mofetile were used to prevent GVHD. For sibling matched patients, only peripheral blood stem cell transplantation was performed. Conditioning regimen included busulphan/ cyclophosphamide. Cyclosporine A and methotrexate were used for GVHD prevention. RESULTS AND CONCLUSION: There was no significant difference in the time of hematopoietic rebuilding between HLA-haploidentical and HLA-matched groups. The accumulative rates of acute GVHD were 73% versus 52% respectively between HLA-haploidentical and HLA-matched group (P > 0.05). The accumulative rate of chronic GVHD was 56% versus 45% (P > 0.05). Transplantation related mortality was 36% versus 17% (P > 0.05). There was no case relapse occurred in HLA-haploidentical group, but two cases relapse occurred in HLA-matched group. The expecting three-years accumulative rate of disease free survival was 61% versus 60% between HLA-haploidentical and HLA-matched groups (P > 0.05). Results indicate that the efficiency is similar between HLA haploidentical and matched sibling haematopoietic stem cell transplantation for leukemia, but moderate to severe acute GVHD occurred higher in HLA haploidentical HSCT than HLA-matched HSCT.