Efficacy of haploidentical allogeneic hematopoietic stem cell transplantation for severe aplastic anemia: a report of 40 cases

Objective To explore the efficacy and prognosis of haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) for severe aplastic anemia (SAA). Methods The clinical data were retrospectively analyzed for 40 SAA cases undergoing haplo-HSCT from September 2013 to February 2018. The conditioning regimen contained cyclophosphamide, fludarabine and antithymocyte globulin with or without busulfan or low-dose total body irradiation. Cyclosporin A, short-term methotrexate and mycophenolate mofetil were dosed for preventing graft versus host disease (GVHD). The median counts of mononuclear cell and CD34+ stem cell were 5.3(2.0~13.5)×108/kg and 5.6(1.6~15.9)×106/kg respectively. Results Among them, hematopoietic reconstitution was achieved (n=36, 90.0 %). The median times for myeloid engraftment and platelet engraftment were 15(10-25) and 17(10~58) days respectively. The incidence of acute graft-versus-host disease(aGVHD)was (35.0±6.8) %. The incidence of chronic GVHD (cGVHD) was (23.0±7.4) %. And 28 SAA cases (70.0 %) survived during a median follow-up period of 353(30~1226) days, The cumulative overall survival (OS) was (67.8±7.8) %, the average survival time (883±82)days and transplantation-related death (TRM) within 100 days (10.0±3.1) %. Conclusions Haplo-HSCT is an effective treatment for SAA patients. And a larger number of cases are required for enhancing OS. Key words: Allogeneic hematopoietic stem cell transplantation; Aplastic anemia; Human leukocyte antigen