同胞脐血移植治疗重型β-地中海贫血的临床研究

Study About Related Umbilical Cord Blood Transplantation in Patients With β-thalassemia Major.

【目的】 探讨同胞脐血移植治疗重型 β 地中海贫血 (以下简称地贫 )的疗效。 【方法】 用HLA全相合或不全相合同胞脐血移植治疗重型 β 地中海贫血患儿 7例。供给受者的有核细胞 ( 4 .5 5～ 16.0 )× 10 7/kg ,CD3 4 + 细胞 ( 0 .11～ 1.0 3 )× 10 6/kg ,粒 巨噬细胞集落形成单位 ( 0 .3 1～ 1.18)× 10 5/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 ;HLA 2个位点不全相合者采用高剂量输血 +连续静注去铁胺 +羟基脲 +氟哒拉膑 +马利兰 +环磷酰胺 +抗胸腺细胞球蛋白方案。 【结果】 7例患儿均获得植入 ,其中 6例患儿为长期稳定植入 ,1例植入后发生排斥。 7例患儿均发生急性移植物抗宿主病 ,其中Ⅰ度 5例 ,Ⅱ度 2例。脱离地贫状态生存 6例 ,血红蛋白一直维持正常 ,1例恢复地贫状态。 【结论】 同胞脐血移植可根治本病。

To examine the curative effect on β thalassemia major with related umbilical cord blood transplantation (RUCBT). RUCBT were performed on 7 patients with β thalassemia major by the HLA identical siblings'cord blood or 2 locus mismatch siblings'umbilical cord blood. The harvest stem cell contain nucleated cells(4.55～16.0)×10 7/kg, CD 34 +cells(0.11～1.03)×10 6/kg, colony forming unit granulocytemacrophages(0.31～1.18)×10 5/kg.the HLA identical patiens accepted the conditioning regimen consisting of busulfan, cyclophosphamide, antithymocyteglobulin; the HLA 2 locus mismatch patient accepted the conditioning regimen consisting of hypertransfusions , continuous i.v. desferrioxamine , hydroxyurea, fludarabine, busulfan, cyclophosphamide , antithymocyteglobulin. All patients were engrafted and accomplished with the acute graft versus host disease(aGVHD).Six patients were survival with ex anemia; their Hb levels had been maintaining normality without transfusion. One patient had rejection and autologous hematopoitic reconstitution.[Conclusions] UCBT is an effective way to treat β thalassemia major.