Jaing Tang-Her, Hung Iou-Jih, Yang Chao-Ping, Lin Tzou-Yien, Chow Robert, Hsieh Suh-Ing
Divisions of Hematology and Oncology, Department of Pediatrics, Chang Gung Children's Hospital, Taiwan.
J Trop Pediatr. 2005 Apr;51(2):122-4. doi: 10.1093/tropej/fmh108.
We report here the first successful transplant of unrelated umbilical cord blood (UCB) for a child with beta-thalassemia major in Taiwan. A total of 2.48 x 10(5)/kg CD34 cells were infused into a 3(1/2)-year-old girl following conditioning with a pre-transplant cytoreductive/immunosuppressive regimen of busulfan and cyclophosphamide. The absolute neutrophil count first exceeded 0.5 x 10(9)/l on day 17, and the patient became red cell- and platelet-independent on days 34 and 49, respectively. Engraftment of donor hematopoietic populations was documented by cytogenetic analysis, hemoglobin electrophoresis, Southern blot analysis for thalassemia markers, and molecular analysis of short tandem repeats sequences. The patient was ex-thalassemic for more than 15 months post transplant. From this experience, it is postulated that UCB will indeed allow us to expand the available donor pool for recipients with an HLA DRB1 mismatched at one locus and reduce the risk of graft-versus-host disease.
我们在此报告台湾首例成功为一名重型β地中海贫血患儿进行非亲属脐带血移植的案例。在采用白消安和环磷酰胺的移植前细胞减灭/免疫抑制方案进行预处理后，将总共2.48 x 10(5)/kg的CD34细胞输注到一名3岁半的女孩体内。绝对中性粒细胞计数在第17天首次超过0.5 x 10(9)/l，患者分别在第34天和第49天不再依赖红细胞和血小板。通过细胞遗传学分析、血红蛋白电泳、地中海贫血标志物的Southern印迹分析以及短串联重复序列的分子分析记录了供体造血群体的植入情况。移植后超过15个月，患者摆脱了地中海贫血。基于这一经验，推测脐带血确实将使我们能够为一个位点HLA DRB1不匹配的受者扩大可用供体库，并降低移植物抗宿主病的风险。
