单倍型异基因造血干细胞移植联合后置环磷酰胺(PT/Cy)治疗重型再生障碍性贫血患者的临床研究  

Clinical Study of Haploid Allogeneic Hematopoietic Stem Cell Transplantation Combined with Post-transplant Cyclophosphamide in Severe Aplastic Anemia Patients

作　　者：李俊南[1] 李平 刘林[1] 肖青[1] 唐晓琼[1] 张红宾[1] 王欣[1] 罗小华[1] 王利[1] LI Jun-Nan;LI Ping;LIU Lin;XIAO Qing;TANG Xiao-Qiong;ZHANG Hong-Bin;WANG Xin;LUO Xiao-Hua;WANG Li(Department of Hematology,The First Affiliated Hospital of Chongqing Medical University,Chongqing 400016,China;Department of Bone Orthopaedics,Chongqing Hospital of Traditional Chinese Medicine,Chongqing 400021,China)

机构地区：[1]重庆医科大学附属第一医院血液内科,重庆400016 [2]重庆市中医院骨一科,重庆400021

出　　处：《中国实验血液学杂志》2022年第1期227-231,共5页Journal of Experimental Hematology

基　　金：重庆市科卫联合医学科研项目重点项目(2018ZDXM001);重庆市渝中区科委重点项目(20190121)。

摘　　要：目的:评估单倍型异基因造血干细胞移植(haplo-HSCT)治疗重型再生障碍性贫血(SAA)患者的疗效;探讨联合后置环磷酰胺(PT/Cy)与标准剂量ATG方案预防GVHD疗效的区别。方法:收集并回顾性分析2012年1月至2019年12月于我院接受haplo-HSCT的SAA患者32例的临床资料,评价治疗疗效。将接受haplo-HSCT治疗的患者按预防移植物抗宿主病(GVHD)方案不同,分为低剂量ATG联合PT/Cy方案和标准剂量ATG组,比较两组患者血细胞造血重建时间、GVHD发生率、死亡率及生存时间的差异。结果:32例患者中,30例获得造血重建,重建率93.75%,中性粒细胞、血小板中位植入时间分别为15(10-22)d、13(7-30)d。GVHD发生率为21.89%,感染发生率为93.75%,2年总生存率为84.38%。低剂量ATG联合PT/Cy组和标准剂量ATG组患者造血重建时间、GVHD发生率、死亡率、生存时间差异均无统计学意义(P>0.05)。结论:Haplo-HSCT是治疗SAA的有效方法,低剂量ATG联合PT/Cy方案可减轻患者的经济负担,副作用小,是治疗SAA可行的方案。Objective:To evaluate the clinical effect of haploid allogeneic hematopoietic stem cell transplantation(haplo-HSCT) in the treatment of severe aplastic anemia(SAA),and to explore the efficacy different between posttransplant cyclophosphamide(PT/Cy) and standard-dose ATG.Methods:The clinical data of 38 patients with SAA in our hospital from January 2012 to December 2019 were collected and retrospectively analyzed.The efficacy was evaluated.The patients with haplo-HSCT were divided into low-dose ATG combined with PT/Cy group and standarddose ATG group,and the blood cell hematopoietic reconstruction time,GVHD incidence,mortality and survival time of the patients in the two groups was compared.Results:Among the 32 patients,hematopoietic reconstitution were detected in 93.75%(30/32) recipients.The median time of neutrophil and platelet engraftment was 15(10-22) days and 13(7-30) days,respectively.The incidence of GVHD was 21.89%,the incidence of infection was 93.75%,and the 2-year overall survival rate was 84.38%.The hematopoietic reconstitution time,incidence of GVHD,mortality rate and survival time were no statistical differences between the patients in the two groups(all P> 0.05).Conclusion:Haplo-HSCT is an effective method for the treatment of SAA,low-dose ATG combined with PT/Cy can lighten the economic burden on patients,it would be a feasible treatment plan for SAA with light side effect.

关 键 词：重型再生障碍性贫血 单倍体异基因造血干细胞移植 疗效 

分 类 号：R457.7[医药卫生—治疗学] R556.5[医药卫生—临床医学]