Text Box: UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT) IN CHILDREN WITH HEREDITARY HAEMOLYTIC ANEMIA Huang S-L, Fang J-P, Chen C, Zhou D-H, Bao R, Wu Y-F Hematopoietic Stem Cell Transplantation Center, Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University of Medical Sciences, Guangzhou, China Objective: To evaluate the efficacy of UCBT in hereditary hemolytic anemia. Methods: Eight patients with hereditary haemolytic anemia were treated by Umbilical cord blood transplantation (UCBT) from related donors, and one patient was treated cord blood from unrelated donor. The patients received the median UCB nucleated cells(NC) 6.70×107/kg, body weight [range (4.7-12.7)×107/kg] and CD34＋CD38－ cells 2.95×105/kg.[range (0.6-11.7) ×105/kg] and CFU-GM 1.09×105/kg [range (0.24-230) ×105/kg]. The Conditioning regimen was consisted of busalphan 16-20mg/kg, cyclophosphamide 180-200mg/kg, horse antithymocyte globulin (ATG) 90mg/kg, or rabbit anti-lymphocyte globulin (ALG) 25mg/kg. Melphalan 90mg/m2 was added at six cases from the sibling donors. Thitepea 6 mg/kg and fludarabine 150 mg/m2 were added at one case from the unrelated cord blood. Cyclosporine A (CsA), alone or combinated with methotrexate or prednisone, was given as prophylaxis against acute graft-verses-host disease (GVHD). Results: 7 cases were engrafted, but one rejected and recured thalassemia state at days 60 after UCBT. Neutrophils achieving 0.5×109/L on day 18.5 (10-22), plalelets>20×109/L on day 35 (19-106). 5 cases had aGVHD of grade I, II and IV respectively. One had cGVHD. 5 patients were survival with ex-thalassemia state. The median follow-up was 20.6 months(8-39), the probability of survival was 88.88％. Conclusion: Cord blood is a feasible alternative source of allogeneic hematopoietic stem cells for pediatric patients with heriditary diseases.

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