Comparison of frontline immunosuppressive therapy and hematopoietic stem cell transplantation for severe hepatitis-associated aplastic anemia
Objective:To compare the clinical efficacy between frontline immunosuppressive therapy(IST)and hematopoietic stem cell transplantation(HSCT)for patients with severe hepatitis-associated aplastic anemia(HAAA).Methods:A total of 41 patients who received frontline IST(12 cases)or HSCT(29 cases)between Jan-uary 1,2013 and August 31,2022 were retrospectively analyzed.Among 29 patients undergoing HSCT,there were 13 cases of matched-sibling donor hematopoietic stem cell transplantation(MSD-HSCT)and 16 cases of hap-lo-identical donor hematopoietic stem cell transplantation(HID-HSCT).Results:Except for death events,the me-dian follow-up time for all patients after transplantation was 67(16-129)months.There was no significant differ-ence in hematopoietic reconstruction time,the infection rate,the incidences of grade Ⅱ-Ⅳ acute graft-versus-host disease and chronic graft-versus-host disease between the MSD-HSCT group and the HID-HSCT group.The inci-dence of treatment failure in the IST group was higher than that in the HSCT group(41.7％vs 10.3％,P=0.034).The estimated 5-year overall survival(OS)and failure-free survival(FFS)were 85.4％and 80.5％for all of the 41 patients.The estimated 5-year OS in the MSD-HSCT group,HID-HSCT group and IST group were 92.3％,87.5％and 75.0％,respectively.The estimated 5-year FFS in the MSD-HSCT group,HID-HSCT group and IST group were 92.3％,87.5％and 58.3％,respectively.There was no significant difference in the estimated 5-year OS between the HSCT group and the IST group,but the estimated 5-year FFS in the MSD-HSCT group was significantly higher than that in the IST group(P=0.039).There was no significant difference in the expected 5-year OS and FFS between the MSD-HSCT group and the HID-HSCT group.Conclusion:HSCT is more suitable for patients with severe HAAA than IST.MSD-HSCT remains the first-line option for HAAA,and HID-HSCT can replace IST as the first-line option for younger patients without a MSD donor.The frontline haplo-HSCT was an effective and safe approach for the treatment of patients with SAA who lack a HLA-matched sibling donor.
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