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

Abstract

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。

Translated abstract

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.