改良FC/ATG预处理在异基因造血干细胞移植治疗重型再生障碍性贫血中的疗效观察  

Effect of modified FC/ATG preconditioning on allogeneic hematopoietic stem cell transplantation for severe aplastic anemia

作　　者：任瑞瑞 马梁明 王涛 朱秋娟 高志林 Ren Ruirui;Ma Liangming;Wang Tao;Zhu Qiujuan;Gao Zhilin(Shanxi Bethune Hospital,Shanxi Academy of Medical Sciences,Taiyuan,030032,China)

机构地区：[1]山西白求恩医院(山西医学科学院)血液科,太原030032

出　　处：《中华器官移植杂志》2021年第11期646-651,共6页Chinese Journal of Organ Transplantation

基　　金：山西省重点研发计划(201803D31144)。

摘　　要：目的评价改良FC/ATG预处理行异基因造血干细胞移植治疗重型再生障碍性贫血(SAA)疗效及安全性。方法回顾性分析2012年6月至2020年6月在山西白求恩医院接受改良FC/ATG(氟达拉滨30 mg·m-2·d-1、环磷酰胺50 mg·kg-1·d-1、抗胸腺细胞球蛋白2.5 mg·kg-1·d-1, 移植前5 d~移植前2 d)预处理行异基因造血干细胞移植64例SAA的临床资料, 其中HLA相合同胞供者造血干细胞移植(MSD-HSCT)29例, 血缘半相合造血干细胞移植(Haplo-HSCT)35例。结果 1例受者在移植前死于脑出血, 其余63例受者全部获得完全植入, 中位随访时间14.5个月(1~95个月), 总存活率为92.2%, 明显高于国外含低剂量TBI预处理方案治疗SAA的总存活率67.2%, 略高于国内采用含FC+马利兰/TBI预处理方案治疗SAA总存活率91.3%。Haplo-HSCT组和MSD-HSCT 3年总存活率分别为85.7%和93.5%(P=0.058), 与"北京方案"(BU/CY+ATG)治疗SAA的Haplo-HSCT组和MSD-HSCT组的总存活率89%、91%相当。Haplo-HSCT组EB病毒、巨细胞病毒感染率明显高于MSD-HSCT组, 两组差异有统计学意义(P<0.05)。单因素分析显示, 二者对受者生存时间无影响(P=0.403、P=0.132);单因素分析显示受者生存时间与有无Ⅲ~Ⅳ°急性移植物抗宿主病(aGVHD)及是否合并严重并发症显著相关(P=0.007、P=0.001);进一步行多因素分析显示是否有严重并发症是影响受者生存期的独立危险因素(P=0.003)。结论与国内、国际其他预处理方案比较, 改良FC/ATG预处理在MSD-HSCT或Haplo-HSCT治疗SAA的总存活率较高, 安全且有效;是否有严重并发症和是否合并Ⅲ~Ⅳ°aGVHD是影响受者生存时间的因素。Objective To evaluate the efficacy and safety of modified FC/ATG pretreatment in the treatment of severe aplastic anemia(SAA).Methods From June 2012 to June 2020,clinical data of 64 patients with severe aplastic anemia undergoing allogeneic hematopoietic stem cell transplantation with modified FC/ATG(Flu 30 mg·m-2·d-l,-5~-2 d、CTX 50 mg·kg-1·d-1~-2 d、ATG:2.5 mg·kg-1·d-1,-5~-2 d)pretreatment were retrospectively analyzed.There were MSD-HSCT(n=29)and Haplo-HSCT(n=35).Results One patient died of intracerebral hemorrhage before transplantation and the remainders were completely implanted.During a median follow-up period of 14.5(1-95)months,overall survival(OS)rate of 92.2%.It was significantly higher than OS rate of 67.2%in the treatment of SAA by foreign pretreatment regimens containing low-dose TBI.And pretreatment scheme containing FC+BU/TBI had an OS of slightly>91.3%in the treatment of SAA.The 3-year OS rates were 85.7%and 93.5%in Haplo-HSCT and MSD-HSCT groups(P=0.058).The OS rate of SAA Haplo-HSCT/MSD-HSCT group was similar to that of"Beijing Protocol"(BU/CY+ATG)(89%,91%).The viral infection rates of EB and CMV were significantly higher in haplo-HSCT group than those in MSD-HSCT group and inter-group difference was statistically significant(P<0.05).However,univariate analysis showed that two groups had no effect on survival time(P=0.403,P=0.132).Univariate analysis showed that survival time was significantly associated with the presence ofⅢ-Ⅳ°aGVHD and the presence of severe complications(P=0.007,P=0.001).Further multivariate analysis revealed that severe complication was an independent risk factor for survival(P=0.003).Conclusions The efficacy of improved FC/ATG pretreatment in the treatment of SAA in MSD-HSCT or Haplo-HSCT is higher than other domestic and international pretreatment schemes in OS rate,safety and effectiveness.Onset of severe complication and association withⅢ~Ⅳ°aGVHD are the influencing factors for patient survival.The efficacy of Haplo-HSCT group is similar to that of MSD

关 键 词：造血干细胞移植 再生障碍性贫血 单倍体 

分 类 号：R556.5[医药卫生—血液循环系统疾病]