Transplantation with Combination of Umbilical Cord Blood and Neonatal Peripheral Blood in the Treatment of A Patient with β-thalassemia Major / 中山大学学报(医学科学版)

ABSTRACT

【Objective】To observe the efficacy and side effects of hematopietic stem cell transplantation with combination of umbilical cord blood(UCB) and neonatal peripheral blood(NPB) in the treatment of β-thalassemia major.【Methods】28 mL NPB was drawn from a HLA identical neonate within 5 hours after his birth to complement stem cell of the UCB he donated for transplantation to his sibling with β-thalassemia major.Various items of hematopoiesis reconstruction were detected in UCB and NPB respectively.After conditioning with chemotherapy by using busulfan 20 mg/kg,cyclophosphamide 200 mg/kg,melphalan 90 mg/m2 and antithymocyte globulin(ATG) 90 mg/kg,the patient received the 53 mL UCB and 28 mL NPB,achieving 5.7×107/kg nucleated cells(NC),93×105/kg CFU-GM and 3.1×105/kg CD34+CD38- cells from his HLA-identical sibling.【Results】Absolute nucleated cell(ANC) reached 0.5×109/L on 14th day post transplant,and platelets reached 20×109/L on 34th day after transplant.The heterozygosity of β-654 mutation point was detected by the PCR-RDB.The sexual chromosome changed from XX pretransplant to XY posttransplant.The patient was free red blood cell transfusion from 14th day post transplant.Her hemoglobin rose progressively from 86 g/L to 110 g/L.The patient survived for 197 days free from disease after transplantation.Following up for 9 months, the donor grew and developed normally.【Conclusion】The NPB contains a lot of stem cells.The transplantation with combination of suitable NPB and UCB is an effective tactics when the UCB cells are deficient.