Haploidentical allogeneic hematopoietic stem cell transplantation for severe aplastic anemia:a retrospective analysis / 中国组织工程研究

ABSTRACT

BACKGROUND:Al ogeneic hematopoietic stem cel transplantation is currently recognized as the first-line therapy for severe aplastic anemia. However, with the popularity of the one-child families, the source of ful y matched hematopoietic stem cel transplantation is limited, so haploidentical hematopoietic stem cel transplantation is favored. OBJECTIVE:To retrospectively compare and analyze the clinical efficacy and safety of haploidentical al ogeneic hematopoietic stem cel transplantation and ful y matched hematopoietic stem cel transplantation for the treatment of severe aplastic anemia. METHODS:Clinical data of 15 patients with severe aplastic anemia (treatment group) who underwent haploidentical al ogeneic hematopoietic stem cel transplantation in the Department of Hematology General Hospital of Beijing Military Region from January 2013 to January 2015 were retrospectively analyzed. Pretreatment regimen was cyclophosphamide, fludarabine, Busulfex, combined with anti-human lymphocyte immune globulin. Donors received granulocyte colony-stimulating factor, and the transplantation method was bone marrow mobilization combined with peripheral blood stem cel transplantation. Combined immunosuppressive agents including cyclosporine A, methotrexate, tacrolimus, were adopted for prevention of graft versus host disease. Another 15 cases of severe aplastic anemia undergoing ful y matched hematopoietic stem cel transplantation served as control group over the same period. Complications and survival of the two groups were statistical y analyzed. RESULTS AND CONCLUSION:By the end of July 2015, the median fol ow-up time of the treatment group was 20.7 months (6-30 months), and hematopoietic reconstruction was achieved in al cases, including four cases of graft versus host disease, five cases of pulmonary infection, three cases of sepsis, and one case died of pulmonary infection, one cases died of sepsis, and two cases died of graft versus host disease. In the control group, the median fol ow-up time was 19.7 months (5-28 months), hematopoietic reconstruction was achieved in al cases. There were three cases of graft versus host disease, four cases of pulmonary infection, one case died of pulmonary infection, and two cases died of graft versus host disease. The total survival rates of the two groups were 73%and 80%respectively, with no significant difference (P=0.67). The haploidentical al ogeneic hematopoietic stem cel transplantation for severe aplastic anemia is safe and effective, and the clinical efficacy is comparable to the ful y matched hematopoietic stem cel transplantation.