Increased immunosuppressive treatment combined with unrelated umbilical cord blood infusion in children with severe aplastic anemia

A combination treatment of unrelated umbilical cord blood (UCB) and increased immunosuppressive treatment (IST) were investigated to reveal the potentially curative therapy for the severe aplastic anemia (SAA). A total of 36 children (2–17 ages) with SAA who received UCB infusion after an IST were analyzed. The treatment consisted of 100 mg/kg cyclophosphamide, 12.5–15 mg/kg antithymocyte globulin and 3 mg/kg cyclosporine. After 3 months, the hematologic complete response (CR) rate was 22.2% and partial response (PR) rate was 38.9%. After 6 months, the CR rate and PR rate was 50.4% and 26.3%, respectively. The probability of 3-year survival was 83.3%. There was no difference in the survival rate either between the horse-ATG and rabbit-ATG or between the SAA and VSAA. The results indicated that the increased IST combined with unrelated UCB infusion has an effective therapeutic potential for children with SAA who lack of compatible donor for transplantation.