单倍型异基因造血干细胞移植治疗重型再生障碍性贫血：回顾性分析

摘要

BACKGROUND:Al ogeneic hematopoietic stem cel transplantation is currently recognized as the first-line therapy for severe aplastic anemia. However, with the popularity of the one-child families, the source of ful y matched hematopoietic stem cel transplantation is limited, so haploidentical hematopoietic stem cel transplantation is favored. OBJECTIVE:To retrospectively compare and analyze the clinical efficacy and safety of haploidentical al ogeneic hematopoietic stem cel transplantation and ful y matched hematopoietic stem cel transplantation for the treatment of severe aplastic anemia. METHODS:Clinical data of 15 patients with severe aplastic anemia (treatment group) who underwent haploidentical al ogeneic hematopoietic stem cel transplantation in the Department of Hematology General Hospital of Beijing Military Region from January 2013 to January 2015 were retrospectively analyzed. Pretreatment regimen was cyclophosphamide, fludarabine, Busulfex, combined with anti-human lymphocyte immune globulin. Donors received granulocyte colony-stimulating factor, and the transplantation method was bone marrow mobilization combined with peripheral blood stem cel transplantation. Combined immunosuppressive agents including cyclosporine A, methotrexate, tacrolimus, were adopted for prevention of graft versus host disease. Another 15 cases of severe aplastic anemia undergoing ful y matched hematopoietic stem cel transplantation served as control group over the same period. Complications and survival of the two groups were statistical y analyzed. RESULTS AND CONCLUSION:By the end of July 2015, the median fol ow-up time of the treatment group was 20.7 months (6-30 months), and hematopoietic reconstruction was achieved in al cases, including four cases of graft versus host disease, five cases of pulmonary infection, three cases of sepsis, and one case died of pulmonary infection, one cases died of sepsis, and two cases died of graft versus host disease. In the control group, the median fol ow-up time was 19.7 months (5-28 months), hematopoietic reconstruction was achieved in al cases. There were three cases of graft versus host disease, four cases of pulmonary infection, one case died of pulmonary infection, and two cases died of graft versus host disease. The total survival rates of the two groups were 73%and 80%respectively, with no significant difference (P=0.67). The haploidentical al ogeneic hematopoietic stem cel transplantation for severe aplastic anemia is safe and effective, and the clinical efficacy is comparable to the ful y matched hematopoietic stem cel transplantation.%背景：随着国内独生子女家庭的普及，全相合造血干细胞移植受干细胞来源限制，临床应用受到局限，因此单倍型造血干细胞移植越来受到亲睐。　　目的：回顾性对比分析单倍型异基因造血干细胞移植和全相合异基因造血干细胞移植治疗重型再生障碍性贫血的临床疗效及安全性。　　方法：选取解放军北京军区总医院血液科2013年1月至2015年1月接受单倍型异基因造血干细胞移植治疗的15例重型再生障碍性贫血患者(治疗组)病例资料，预处理方案为环磷酰胺、氟达拉滨、白舒非联合抗人淋巴细胞免疫球蛋白，供者接受粒细胞集落刺激因子动员，移植方式应用骨髓联合外周血干细胞移植。采用联合免疫抑制剂包括环孢素 A、甲氨蝶呤、他克莫司等预防移植物抗宿主病。同时选择同期行全相合异基因造血干细胞移植治疗的15例重型再生障碍性贫血患者病例资料作为对照组，统计两组患者移植相关并发症及存活情况。　　结果与结论：随访至2015年7月，治疗组中位随访时间20.7个月(6-30个月)，全部患者均获造血重建，4例发生移植物抗宿主病、5例合并肺部感染、3例合并败血症，因肺部感染死亡1例、败血症死亡1例、移植物抗宿主病死亡2例；对照组中位随访时间19.7个月(5-28个月)，全部患者均获造血重建，3例发生移植物抗宿主病、4例合并肺部感染，因移植物抗宿主病死亡2例、肺部感染死亡1例，两组患者总生存率分别为73%和80%，差异无显著性意义(P=0.67)。结果表明单倍型移植治疗重型再生障碍性贫血安全有效，临床疗效与全相合造血干细胞移植相当。