异基因造血干细胞移植治疗24例重型再生障碍性贫血  

Allogeneic hematopoietic stem cell transplantation in the treatment of 24 patients with severe aplastic anemia

作　　者：付春梅 张璞[1] 王洋[1] 李晓林[1] 薛燕[1] 付杰[1] 张慈现[1] 杨宇娟[1] 段雅雅[1] 冯凯[1] Fu Chunmei;Zhang Pu;Wang Yang;Li Xiaolin;Xue Yan;Fu Jie;Zhang Cixian;Yang Yujuan;Duan Yaya;Feng Kai(Department of Hematology,Xuzhou Central Hospital,Xuzhou 221009,Jiangsu Province,China)

机构地区：[1]徐州市中心医院血液科,江苏省徐州市221009

出　　处：《中国组织工程研究》2023年第1期15-20,共6页Chinese Journal of Tissue Engineering Research

基　　金：吴阶平医学基金会临床科研专项资助基金(320.6755.15029),项目负责人:冯凯。

摘　　要：背景:重型再生障碍性贫血病情重、病死率高,需快速恢复造血功能,目前异基因造血干细胞移植为一线治疗方案,同胞全相合异基因造血干细胞移植为首选,单倍体相合造血干细胞移植作为替代治疗方案也取得了较好的效果。目的:探讨异基因造血干细胞移植(包括同胞全相合造血干细胞移植及单倍体相合造血干细胞移植)治疗重型再生障碍性贫血的临床疗效。方法:回顾性分析2015年4月至2021年7月于徐州市中心医院接受异基因造血干细胞移植治疗24例重型再生障碍性贫血患者的临床资料,其中接受同胞全相合造血干细胞移植8例,接受单倍体相合造血干细胞移植16例。24例重型再生障碍性贫血患者预处理方案为氟达拉滨、环磷酰胺、抗淋巴细胞球蛋白方案。同胞全相合造血干细胞移植采用环孢素联合短程甲氨蝶呤预防移植物抗宿主病,单倍体相合造血干细胞移植在此基础上增加吗替麦考酚酯。结果与结论:①24例重型再生障碍性贫血患者中有2例患者预处理期间死于严重感染,其余22例均达造血重建;中性粒细胞植入中位时间为12.5(10-18)d,血小板植入中位时间为14.5(10-26)d;②22例植入成功患者发生急性移植物抗宿主病8例(36%),Ⅲ/Ⅳ度急性移植物抗宿主病2例,慢性移植物抗宿主病累计发生4例(18%),Ⅲ/Ⅳ度慢性移植物抗宿主病1例;③18例患者存活,6例患者死亡,5年预计总生存率为74%;④结果表明,异基因造血干细胞移植为重型再生障碍性贫血的有效治疗手段,同胞全合供者作为首选,无同胞全相合供者时可选择单倍体相合供者作为替代。BACKGROUND:Severe aplastic anemia has severe illness and high mortality,which requires rapid recovery of hematopoietic function.At present,hematopoietic stem cell transplantation is the first-line treatment,human leucocyte antigen-matched sibling donor hematopoietic stem cell transplantation is the first choice,and haploidentical hematopoietic stem cell transplantation has also achieved good results as an alternative treatment.OBJECTIVE:To investigate the efficacy of allogeneic hematopoietic stem cell transplantation(including matched sibling donor hematopoietic stem cell transplantation and haploidentical hematopoietic stem cell transplantation) in the treatment of severe aplastic anemia.METHODS:The clinical data of 24 patients with severe aplastic anemia diagnosed from April 2015 to July 2021 undergoing allogeneic hematopoietic stem cell transplantation in Xuzhou Center Hospital were retrospectively analyzed.Among them,8 patients received matched sibling donor hematopoietic stem cell transplantation and 16 patients received haploidentical hematopoietic stem cell transplantation.The pretreatment regimen of 24 patients with severe aplastic anemia was fludarabine,cyclophosphamide and anti-lymphocytic globulin.The prevention of graft versus host disease was cyclosporine combined with shortrange methotrexate in matched sibling donor hematopoietic stem cell transplantation,and in haploid matched hematopoietic stem cell transplantation on the basis of this addition of mycophenolate mofetil.RESULTS AND CONCLUSION:(1) Among the 24 patients,2 severe aplastic anemia patients died of severe infection during pretreatment,and the other 22 patients reached hematopoietic reconstitution.The median time of neutrophil implantation was 12.5(10-18) days,and the median time of platelet implantation was 14.5(10-26) days.(2) Totally 22 patients with successful implantation developed acute graft versus host disease in 8 patients(36%),grade Ⅲ/Ⅳ acute graft versus host disease in 2 patients,chronic graft versus host disease in 4 pati

关 键 词：重型再生障碍性贫血 异基因造血干细胞移植 单倍体移植 同胞全相合 造血重建 移植物抗宿主病 预后 

分 类 号：R457[医药卫生—治疗学] R394.2[医药卫生—临床医学]