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

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.