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首页> 外文期刊>Pediatric blood & cancer >Fludarabine‐based reduced toxicity yet myeloablative conditioning is effective and safe particularly in patients with high‐risk thalassemia undergoing allogeneic transplantation
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Fludarabine‐based reduced toxicity yet myeloablative conditioning is effective and safe particularly in patients with high‐risk thalassemia undergoing allogeneic transplantation

机译:基于Fludarabine的降低的毒性且肌肉性化条件是有效和安全的,特别是在高风险的患者中进行同种异体移植症

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摘要

Abstract Introduction Thalassemia major (TM) is an inherited disorder caused by ineffective erythropoiesis. At the present time, allogeneic stem cell transplantation (allo‐SCT) is a curative option. Conventional busulfan and cyclophosphamide based myeloablative conditioning regimens are limited by increased toxicity, especially in high‐risk patients. Replacement of cyclophosphamide with fludarabine has reduced toxicity and nonrelapse mortality (NRM), thus improving outcomes. We analyzed long‐term data of our fludarabine‐based myeloablative, reduced toxicity protocol, specifically in high‐risk patients. Methods We retrospectively analyzed a cohort of 47 consecutive patients with TM undergoing allo‐SCT from matched donors, using the fludarabine‐based regimen (reduced toxicity regimen). The median age of the cohort was 10?years. Thirty‐eight patients (80%) were in the high‐risk and nine patients (20%) were in the low‐risk category. The primary aim of this analysis was thalassemia‐free survival (TFS). Results The rejection rate was 11% within high‐risk patients with NRM of 2%. With a median follow‐up period of 7?years (1–15?years), the 10‐year TFS in the entire cohort was 87%, and the overall survival (OS) was 97%. The 10‐year TFS and OS among the low‐risk and high‐risk groups were 90% versus 84%, respectively ( P ?=?0.45) and 100% versus 96%, respectively ( P ?=?0.5), and both subsets of patients did equally well. Conclusion In conclusion, replacement of high‐dose cyclophosphamide with fludarabine is well tolerated with minimal regimen‐related toxicity and acceptable rejection rates, especially in high‐risk patients.
机译:摘要介绍秋季海组织主要(TM)是由无效的促红细胞生成引起的遗传障碍。目前,同种异体干细胞移植(Allo-SCT)是一种治疗方法。常规的伯普兰和基于环磷酰胺的髓鞘调节方案受到毒性增加的限制,特别是在高风险患者中。用氟咔啉替代环磷酰胺,具有降低的毒性和非筛选死亡率(NRM),从而改善结果。我们分析了基于氟氮胺的髓系的长期数据,细微的毒性方案,特别是在高风险患者中。方法采用氟氮滨滨的方案(减少毒性方案),回顾性分析了从匹配的供体从匹配的供体中接受血液谐波的47个连续患者的队列。队列中位年龄为10年代。三十八名患者(80%)在高风险中,九个患者(20%)是低风险的类别。该分析的主要目的是自由贫血生存率(TFS)。结果抑制率为11%,高风险患者为2%。随着7岁的中位随访时间(1-15?年),整个队列的10年TFS为87%,总生存(OS)为97%。低风险和高风险群体中的10年TFS和Os分别为80%,分别为84%(p?= 0.45)和100%,分别为96%(p?= 0.5),两者均患者的子集同样良好。结论结论是,用氟氮杂的高剂量环磷酰胺替代耐受性耐受性毒性最小的毒性和可接受的抑制率,特别是在高危患者中。

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  • 来源
    《Pediatric blood & cancer》 |2018年第11期|共6页
  • 作者单位

    Bone Marrow Transplantation and Cancer Immunotherapy DepartmentHadassah Ein KeremJerusalem Israel;

    Bone Marrow Transplantation and Cancer Immunotherapy DepartmentHadassah Ein KeremJerusalem Israel;

    Bone Marrow Transplantation and Cancer Immunotherapy DepartmentHadassah Ein KeremJerusalem Israel;

    Bone Marrow Transplantation and Cancer Immunotherapy DepartmentHadassah Ein KeremJerusalem Israel;

    Department of bone marrow transplant and cancer immunotherapyHadassah Medical UniversityJerusalem;

    Bone Marrow Transplantation and Cancer Immunotherapy DepartmentHadassah Ein KeremJerusalem Israel;

    Bone Marrow Transplantation and Cancer Immunotherapy DepartmentHadassah Ein KeremJerusalem Israel;

  • 收录信息
  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类 儿科学;
  • 关键词

    fludarabine conditioning; thalassemia; transplantation;

    机译:Fludarabine调理;地中海贫血;移植;

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