摘要
【目的】观察脐带血、新生儿外周血混合的造血干细胞移植治疗 1例重型 β 地中海贫血的疗效和副作用。【方法】采集 1例产前HLA配型相合男性 (XY)胎儿的脐带血 5 3mL ,并于供者娩出后 5h内抽取未作任何药物“动员”的新生儿外周血 2 8mL。测定脐带血、新生儿外周血的各项造血指标。重型 β 地中海贫血女性 (XX)患儿经总量马利兰 2 0mg/kg ,环磷酰胺 2 0 0mg/kg ,马法兰 90mg/m2 和抗胸腺淋巴细胞球蛋白 90mg/kg组成的方案预处理后 ,输入HLA相合同胞的脐带血5 3mL和新生儿外周血 2 8mL ,患儿共获得有核细胞数 5 7× 10 7/kg ,CFU GM 4 93× 10 5/kg ,CD3 4+ CD3 8-细胞 3 1× 10 5/kg。【结果】术后 14d中性粒细胞绝对值 >0 5× 10 9/L ,34d血小板 >2 0× 10 9/L ,聚合酶链—反向点杂交检测患儿术后为 β 6 5 4杂合子 ,性染色体从术前的XX转变为XY。术后 14d起脱离红细胞输注 ,血红蛋白从 86 g/L稳步升至 110g/L以上。现已脱离地中海贫血状态存活 197d。供者于生后 9个月内的追踪观察表明生长发育正常。【结论】新生儿外周血中含有一定量的造血干 /祖细胞 ,对脐血细胞数量不足者 。
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. 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/m 2 and antithymocyte globulin(ATG) 90 mg/kg, the patient received the 53 mL UCB and 28 mL NPB, achieving 5 7×10 7/kg nucleated cells(NC), 93×10 5/kg CFU GM and 3 1×10 5/kg CD 34 +CD 38 - cells from his HLA identical sibling. Absolute nucleated cell(ANC) reached 0 5×10 9/L on 14th day post transplant, and platelets reached 20×10 9/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.
出处
《中山医科大学学报》
CSCD
北大核心
2001年第3期205-208,共4页
Academic Journal of Sun Yat-sen University of Medical Sciences
基金
美国中华医学基金会资助 ( 96 6 30 )
关键词
造血干细胞移植
脐带血
新生儿外周血
Β地中海贫血
hematopoietic stem cell transplantation
umbilical cord blood
neonatal peripheral blood
β-thalassemia
