Allogeneic hematopoietic stem cell transplantation for severe aplastic anemia

Xi Rui; Bai Hai; Wang Cun-bang

doi:10.3969/j.issn.2095-4344

Abstract：BACKGROUND:In recent years, with the development of hematopoietic stem cel transplantation technique and the use of immunosuppressants, the therapeutic effects on severe aplastic anemia have been improved significantly. Especialy relative HLA-identical matching alogeneic hematopoietic stem cel transplantation has achieved a high cure rate. OBJECTIVE:To explore the feasibility of alogeneic hematopoietic stem cel transplantation for the treatment of severe aplastic anemia. METHODS: From 2009 to 2011, 20 patients with severe aplastic anemia received fludarabine, cyclophosphamide and antithymocyte globilin as preconditioning before alogeneic hematopoietic stem cel transplantation from 12 matched and 8 mismatched donors. Except one case undergoing unrelated peripheral blood stem cel transplantation, grafts were from mobilized peripheral blood and bone marrow in al cases. Graft versus host disease prophylaxis consisted of cyclosporine-A and short-course methotrexate in the HLA-identical patients as wel as cyclosporine, methotrexate, and short-course mycophenolate mofetil in HLA-mismatched patients. RESULTS AND CONCLUSION:After transplantation, the time to recover the neutrophil count above > 0.5×109/L was median 12.5 days, and the time to recover the platelet count above > 20×109/L was median 18 days. Twenty patients were folowed up for 24-60 months. The overal survival rate was 75% (n=15), the event-free survival rate was 70% (n=14). Five cases died. The event-free survival rate were 83% (10/12 patients) for HLA-identical patients and 50% (4/8 patients) for HLA-mismatched patients. Acute and chronic graft versus host disease was found in 5 of 20 patients, severe bacterial infection in 4 of 20 patients and fungal infections in 3 of 20 patients. <br> Alogeneic stem cel transplantation is effective in the treatment of severe aplastic anemia, especialy for HLA-identical patients.