Allogeneic hematopoietic stem cell transplantation with low-dose bone marrow and PBSCs in 18 patients with severe aplastic anemia
Objective:We analyzed the outcome of patients with severe aplastic anemia(SAA)undergoing allogeneic hematopoietic stem cell transplantation who received both low-dose bone marrow and peripheral blood stem cells(PBSC),and assessed the efficacy and safety of this stem cell source.Methods:In this retrospective cohort study,we included patients with newly diagnosed SAA who received hematopoietic stem cell transplantation with low-dose bone marrow(median 200 mL,range 80-280 mL)and PBSC.The analysis focused on their hematopoietic reconstruction,incidence of graft-versus-host disease(GVHD),occurrence of infections,overall survival rate,and other relevant indicators.Results:The median age of the 18 patients was 27 years(range,13-52).There were 11 males and 11 females.There were 15 cases of severe aplastic anemia type Ⅰ(SAA-Ⅰ)and 3 cases of type Ⅱ(SAA-Ⅱ).There were 15 cases of haploidentical donors and 3 cases of matched sibling donors.The conditioning regimen consisted of the following:Fludarabine/cyclophosphamide/anti thymo-cyte globulin regimen(n=2),Fludarabine/cyclophosphamide and post-transplant cyclophosphamide regimen(n=8),Fludarabine/cyclophosphamide/anti thymocyte globulin and post-transplant cyclophosphamide regimen(n=8).Cyclospo-rine or tacrolimus in combination with methotrexate and mycophenolate mofetil was used to GVHD prophylaxis.All patients achieved durable engraftment,but one patient experienced secondary poor graft function.A total of 10(55.6％)individual patients experienced infections after transplant.Of a total of 19 documented infection events,10 were attributed to CMV or EBV reactivation,and 6 to bacterial infections.The incidence of acute GVHD was 16.7％,including one case of grade Ⅰ and two cases of grade Ⅱ,and that of mild chronic GVHD was 16.7％.The two-year overall survival rate was 72.2％(95％CI:51.4-93.0).The mortality of 5 patients was attributed to infectious complications,including 4 cases of pulmonary infections and one case of intracranial infection.Conclusion:The combination of low-dose bone marrow and peripheral blood appears to be a potentially effective and feasible graft source for hematopoietic stem cell transplantation in SAA patients.
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