Haplo-HSCT治疗重型再生障碍性贫血40例的疗效观察

Efficacy of haploidentical allogeneic hematopoietic stem cell transplantation for severe aplastic anemia: a report of 40 cases

目的探讨单倍体相合造血干细胞移植(Haplo-HSCT)治疗重型再生障碍性贫血(SAA)的临床疗效及预后。方法回顾性分析2013年9月至2018年02月接受Haplo-HSCT治疗的40例SAA的临床资料。预处理方案包括环磷酰胺、氟达拉滨及兔抗人胸腺细胞免疫球蛋白,部分受者加用白消安或减量全身照射。应用环孢素A、短程甲氨蝶呤、吗替麦考酚酯预防移植物抗宿主病(GVHD)。回输移植物单个核细胞(MNC)中位数5.3(2.0~13.5)×10^8/kg,CD34+细胞中位数5.6(1.6~15.9)×10^6/kg。结果40例受者中,36例(90.0 %)植活,达到造血重建。中性粒细胞及血小板植入中位时间为15(10~25)d及17(10~58)d。aGVHD发生率(35.0±6.8)%,cGVHD发生率(23.0±7.4)%,中位随访时间353(30~1226)d,28例(70.0 %)存活,累积总生存率(OS)为(67.8±7.8)%,平均生存时间(883±82)d。100 d内移植相关死亡率(TRM)为(10.0±3.1)%。结论Haplo-HSCT是治疗SAA的有效方法,但OS的提高尚需大样本临床研究的开展。

Objective To explore the efficacy and prognosis of haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) for severe aplastic anemia (SAA). Methods The clinical data were retrospectively analyzed for 40 SAA cases undergoing haplo-HSCT from September 2013 to February 2018. The conditioning regimen contained cyclophosphamide, fludarabine and antithymocyte globulin with or without busulfan or low-dose total body irradiation. Cyclosporin A, short-term methotrexate and mycophenolate mofetil were dosed for preventing graft versus host disease (GVHD). The median counts of mononuclear cell and CD34+ stem cell were 5.3(2.0~13.5)×108/kg and 5.6(1.6~15.9)×106/kg respectively. Results Among them, hematopoietic reconstitution was achieved (n=36, 90.0 %). The median times for myeloid engraftment and platelet engraftment were 15(10-25) and 17(10~58) days respectively. The incidence of acute graft-versus-host disease(aGVHD)was (35.0±6.8)%. The incidence of chronic GVHD (cGVHD) was (23.0±7.4)%. And 28 SAA cases (70.0 %) survived during a median follow-up period of 353(30~1226) days, The cumulative overall survival (OS) was (67.8±7.8)%, the average survival time (883±82)days and transplantation-related death (TRM) within 100 days (10.0±3.1)%. Conclusions Haplo-HSCT is an effective treatment for SAA patients. And a larger number of cases are required for enhancing OS.