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免疫抑制治疗联合无关脐带血输注治疗儿童重型再生障碍性贫血。

Increased immunosuppressive treatment combined with unrelated umbilical cord blood infusion in children with severe aplastic anemia.

机构信息

Department of Hematology, The General Hospital of Jinan Military District, Jinan, China.

Department of Hematology, The General Hospital of Jinan Military District, Jinan, China.

出版信息

Cell Immunol. 2014 May-Jun;289(1-2):150-4. doi: 10.1016/j.cellimm.2014.03.014. Epub 2014 Apr 3.

DOI:10.1016/j.cellimm.2014.03.014
PMID:24838091
Abstract

A combination treatment of unrelated umbilical cord blood (UCB) and increased immunosuppressive treatment (IST) were investigated to reveal the potentially curative therapy for the severe aplastic anemia (SAA). A total of 36 children (2-17 ages) with SAA who received UCB infusion after an IST were analyzed. The treatment consisted of 100mg/kg cyclophosphamide, 12.5-15 mg/kg antithymocyte globulin and 3mg/kg cyclosporine. After 3 months, the hematologic complete response (CR) rate was 22.2% and partial response (PR) rate was 38.9%. After 6 months, the CR rate and PR rate was 50.4% and 26.3%, respectively. The probability of 3-year survival was 83.3%. There was no difference in the survival rate either between the horse-ATG and rabbit-ATG or between the SAA and VSAA. The results indicated that the increased IST combined with unrelated UCB infusion has an effective therapeutic potential for children with SAA who lack of compatible donor for transplantation.

摘要

本研究旨在探讨无关脐血(UCB)联合强化免疫抑制治疗(IST)的联合治疗方案,为严重再生障碍性贫血(SAA)提供潜在的治愈疗法。对 36 例接受 IST 后 UCB 输注的 SAA 患儿进行分析。治疗方案包括 100mg/kg 环磷酰胺、12.5-15mg/kg 抗胸腺细胞球蛋白和 3mg/kg 环孢素。3 个月后,完全血液学缓解(CR)率为 22.2%,部分缓解(PR)率为 38.9%。6 个月后,CR 率和 PR 率分别为 50.4%和 26.3%。3 年生存率为 83.3%。马抗胸腺细胞球蛋白和兔抗胸腺细胞球蛋白、SAA 和 VSAA 之间的生存率无差异。结果表明,强化 IST 联合无关 UCB 输注对缺乏移植相容供体的 SAA 患儿具有有效的治疗潜力。

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