Haploidentical peripheral blood hematopoietic stem cell transplantation for treatment of severe aplastic anemia

Objective To investigate the efficacy and safety of modified busulfan/cyclophosphamide+antithymocyte globulin(Bu/Cy+ATG) conditioning regimen to treat severe aplastic anemia(SAA).Methods Retrospective analysis was performed on 3 patients with SAA treated by modified Bu/Cy+ATG conditioning regimen before related haploidentical hematopoietic stem cell transplantation.Donors were their mothers,with 3/6 HLA matches in 1 patient and 4/6 matches in the other 2 patients.Bu/Cy+ATG conditioning regimen contained busulfan 0.8 mg/kg,4 times per day for 2 d,cyclophosphamide 50 mg·kg·d-1 for 4 d,and antithymocyte globulin 2.5 mg·kg·d-1 for 4 d.Cyclosporine,methotrexate,and mycophenolate were used to prevent graft-versus-host disease(GVHD).Results All 3 patients achieved full donor type engraftment.Two suffered from grade Ⅱ-Ⅲ acute GVHD.One suffered from limited chronic GVHD.Three patients were infected with CMV,but the condition was effectively controlled by antiviral treatments.With 5-25 month follow-up,all 3 patients survived.Conclusion Preliminary results showed that the modified Bu/Cy+ATG conditioning regimen used to treat SAA with haploidentical hematopoietic stem cell transplantation was safe and effective.