同胞脐血和同供者骨髓联合移植治疗儿童重型β-地中海贫血

Combined Transplantation of Umbilical Cord Blood and Bone Marrow from Same Sibling Donor in Children with β-Thalassemia Major

为了研究同胞脐血和骨髓联合移植治疗儿童重型β-地中海贫血的疗效,用HLA全相合同胞脐血+骨髓移植治疗3例重型β-地中海贫血。供给3例受者的有核细胞细胞数分别为19.5×107/kg、20.8×107/kg和23.3×107/kg。移植的预处理方案:采用马利兰+环磷酰胺+抗胸腺细胞球蛋白方案。结果表明:3例患儿均获得长期稳定植入,中性粒细胞0.5×109/L的时间分别为16、18、17天,血小板50×109/L的时间分别为48、50、49天,造血重建速度较单纯脐血移植快。3例患儿现已脱离了地中海贫血状态生活了1.5、2.0、2.1年,血红蛋白一直维持在正常水平。3例患儿均发生I度急性移植物抗宿主病(aGVHD)。结论:同胞脐血和骨髓联合移植可能作为一种安全和有效的移植方式用于治疗儿童地中海贫血。

The objective of this study was to investigate the curative effect of combined sibling umbilical cord blood and bone marrow transplantation in treatment of β-thalassemia major. Combined umbilical cord blood and bone marrow transplantation from an HLA-identical sibling were performed for 3 patients with β-thalassemia major. The nucleated cells infused into 3 recepients were 19.5 × 10^7/kg, 20. 8 × 10^7/kg and 23.3× 10^7/kg respectively. They accepted the conditioning regimen consisting of busulfan, cyclophosphamide, antithymocyteglobulin, The results showed that three patients gained protracted and stable engraftment. The time to achieve more than 0.5× 10^9/L neutrophils in three patients was 16, 18, and 17 days respectively; the time to achieve more than 50× 10^9/L platelet in three patients was 48, 50, and 49 days respectively, The speed of hematopoietic recovery was faster than that of umbilical cord blood transplantation （UCBT） only, Three patients all suffered from acute graft-versus-host disease （aGVHD） of I grade. They had lived with free-thalassemia for 1,5, 2.0 and 2. 1 years respectively. Their Hb had been maintained at normal level without transfusion. It is concluded that combined UCBT and BMT may be an effective and safe way to treat pediatric β-thalassemia major,