异基因造血干细胞移植治疗肝炎相关性再生障碍性贫血28例疗效及安全性

Efficacy and safety of allogeneic hematopoietic stem cell transplantation in the treatment of 28 patients with hepatitis-related aplastic anemia

目的研究异基因造血干细胞移植(allo-HSCT)治疗肝炎相关性再生障碍性贫血(HRAA)的疗效及安全性。方法对2012年1月至2022年6月在中国医学科学院血液病医院干细胞移植中心接受allo-HSCT的HRAA患者进行回顾性分析。随访时间至2022年10月30日。结果①共纳入接受allo-HSCT的HRAA患者28例,男18例(64.3%),女10例(35.7%),中位年龄25.5(9~44)岁。重型再生障碍性贫血(SAA)17例,极重型再生障碍性贫血(VSAA)10例,输血依赖型非重型再生障碍性贫血(TD-NSAA)1例。28例患者中单倍体造血干细胞移植(haplo-HSCT)15例,同胞全相合造血干细胞移植(MSD-HSCT)13例。②移植后100 dⅡ~Ⅳ度急性GVHD累积发生率为25.0%(95%CI 12.8%~45.4%),2年慢性GVHD累积发生率为4.2%(95%CI 0.6%~25.4%)。③移植后2年总生存(OS)率为81.4%(95%CI 10.5%~20.6%),无失败生存(FFS)率为81.4%(95%CI 10.5%~20.6%),移植相关死亡率(TRM)为14.6%(95%CI 5.7%~34.3%),所有患者移植后均未发生显著肝损伤。④haplo-HSCT组移植后巨细胞病毒(CMV)血症发生率高于MSD-HSCT组[60.0%(95%CI 35.2%~84.8%)对7.7%(95%CI 0~22.2%),P=0.004],两组EB病毒血症发生率、2年OS率、2年FFS率、2年TRM、移植后100 dⅡ~Ⅳ度急性GVHD累积发生率及2年慢性GVHD累积发生率差异均无统计学意义。结论allo-HSCT治疗HRAA安全有效。在无法获得HLA全相合同胞供者时,haplo-HSCT可作为替代选择。

Objective To evaluate the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation(allo-HSCT)for hepatitis-related aplastic anemia(HRAA)patients.Methods Retrospective analysis was performed on hepatitis-associated aplastic anemia patients who received haplo-HSCT at our center between January 2012 and June 2022.October 30,2022 was the final date of follow-up.Results This study included 28 HRAA patients receiving allo-HSCT,including 18 males(64.3%)and 10 females(35.7%),with a median age of 25.5(9-44)years.About 17 cases of severe aplastic anemia(SAA),10 cases of very severe aplastic anemia(VSAA),and 1 case of transfusion-dependent aplastic anemia(TD-NSAA)were identified.Among 28 patients,15 patients received haplo-HSCT,and 13 received MSD-HSCT.The 2-year overall survival(OS)rate,the 2-year failure-free survival(FFS)rate,the 2-year transplant-related mortality(TRM)rate,the 100-day gradeⅡ-Ⅳacute graft-versus-host disease(aGVHD)cumulative incidence rate,and the 2-year chronic graft-versus-host disease(cGVHD)cumulative incidence rate were 81.4%,81.4%(95%CI 10.5%-20.6%),14.6%(95%CI 5.7%-34.3%),25.0%(95%CI 12.8%-45.4%),and 4.2%(95%CI 0.6%-25.4%),respectively.After transplantation,all patients had no significant liver function damage.Compared with the MSD-HSCT group,only the incidence of cytomegaloviremia was significantly higher in the haplo-HSCT group[60.0%(95%CI 35.2%-84.8%)vs 7.7%(95%CI 0-22.2%),P=0.004].No statistically significant difference in the Epstein-Barr virus was found in the 2-year OS,2-year FFS,2-year TRM,and 100-day gradeⅡ-ⅣaGVHD cumulative incidence rates and 2-year cGVHD cumulative incidence rate.Conclusion Allo-HSCT is safe and effective for HRAA,and haplo-HSCT can be used as a safe and effective alternative for newly diagnosed HRAA patients who cannot obtain HLA-matched sibling donors.