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 grade Ⅱ-Ⅳ acute 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 grade Ⅱ-Ⅳ aGVHD 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.
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 d Ⅱ~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 d Ⅱ~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。.
Keywords: HLA-sibling allogeneic hematopoietic stem cell transplantation; Haploidentical hematopoietic stem-cell transplantation.; Hepatitis related aplastic anemia.