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目的 探讨脐血移植(UCBT)治疗重型β 地中海贫血 (简称β 地贫 )的疗效。方法 用人类白细胞抗原(HLA)全相合或不全相合UCBT治疗重型β 地贫患儿 12例。供受者的有核细胞(3.63~16.0)×107 /kg,CD34+细胞(0 11~1 03)×106 /kg,粒 巨噬细胞集落形成单位 (0.17~1.18)×105 /kg。移植的预处理方案:HLA全相合的患儿采用马利兰+环磷酰胺+抗胸腺细胞球蛋白方案;HLA2个位点不全相合者采用高剂量输血+连续静脉滴注去铁胺+羟基脲+氟达拉宾+马利兰+环磷酰胺+抗胸腺细胞球蛋白方案。结果 10例患儿获得植入,其中 7例为长期稳定植入, 3例植入后发生排斥; 2例未能植入。获得植入的 10例患儿均发生急性移植物抗宿主病 (aGVHD),其中Ⅰ度aGVHD7例,Ⅱ度aGVHD3例。脱离地贫状态生存 7例,血红蛋白始终维持正常。3例恢复地贫状态。2例未获植入的患儿 1例发生移植后再生障碍性贫血, 1例死于严重感染。结论 UCBT是目前β 地贫最有效的治疗手段。
Objective To investigate the efficacy of cord blood transplantation (UCBT) in the treatment of severe β-thalassemia (β thalassemia). Methods Twelve patients with severe β-thalassemia were treated with HLA-identical or incompletely matched UCBT. The numbers of nucleated cells (3.63 ~ 16.0) × 107 / kg, CD34 + cells (0 11 ~ 1 03) × 106 / kg and granulocyte macrophage colony forming units (0.17 ~ 1.18) × 105 / kg were Transplanted preconditioning regimens: HLA-matched children with Malilan + cyclophosphamide + anti-thymocyte globulin program; HLA2 incomplete combination of sites by high-dose transfusion + continuous intravenous deferoxamine + hydroxyurea + fluorine Dalabin + marilan + cyclophosphamide + anti-thymocyte globulin program. Results Ten patients were implanted. Seven of them were implanted stably for a long time, three patients were rejected after implanted. Two patients were implanted. Acute graft versus host disease (aGVHD) occurred in all of the 10 patients who received implantation, of whom 7 were grade Ⅰ aGVHD and 3 were grade Ⅱ aGVHD. 7 cases were out of thalassemia, hemoglobin remained normal all the time. 3 cases of return to thalamic status. Two cases of non-implanted children had aplastic anemia after transplantation and one case died of severe infection. Conclusion UCBT is currently the most effective treatment for β-thalassemia.