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Paediatric reduced intensity conditioning: analysis of centre strategies on regimens and definitions by the EBMT Paediatric Diseases and Complications and Quality of Life WP

机译:儿科降低强度调节:EBMT儿科疾病和并发症及生活质量WP的治疗方案和定义的中心策略分析

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The aim of this analysis was to explore the diversity of reduced intensity conditioning (RIC) in paediatric allo-SCT in daily practice across Europe. Data from the European Group for Blood and Marrow Transplantation (EBMT) Promise database from 1994 to 2008 were supplemented by a survey of EBMT centres performing paediatric allo-SCT on the current policy asking for the underlying diseases and for the drug combinations. Records from 161 centres from 30 countries were analysed and 139 various RIC regimens were reported. More centres applied RIC for malignant rather than for non-malignant diseases. In general, fludarabine (FLU)-based regimens predominated except for BU-based regimens in myeloid malignancies and haemoglobinopathies. Treosulfan (TREO) was mainly applied for unspecified malignant diseases and for haemophagocytic diseases. FLU-based regimens revealed the greatest number of different combinations. Correlating the number of regimens with the number of treating centres revealed the lowest variety in FLU and the highest variety in TBI and TREO. FLU/melphalane and FLU/CY were the most frequent combinations. This extreme heterogeneity in RIC may influence both the efficacy and the safety of the procedures, which requires further investigation. Optimization and standardization of RIC is the final goal to provide a platform for future prospective studies.
机译:这项分析的目的是探讨整个欧洲在日常实践中小儿同种异体SCT的减低强度调节(RIC)的多样性。 1994年至2008年,来自欧洲血液和骨髓移植组织(EBMT)Promise数据库的数据得到了对EBMT中心进行调查的补充,这些中心进行了有关儿科同种异体SCT的现行政策,询问潜在疾病和药物组合。分析了来自30个国家/地区的161个中心的记录,并报告了139种RIC方案。更多的中心将RIC用于恶性而非非恶性疾病。通常,在髓样恶性肿瘤和血红蛋白病中,基于氟达拉滨(FLU)的治疗方案占优势,除了基于BU的治疗方案。 Treosulfan(TREO)主要用于未指明的恶性疾病和噬血细胞疾病。基于FLU的方案显示出最多数量的不同组合。将治疗方案的数量与治疗中心的数量相关联,发现FLU的种类最少,而TBI和TREO的种类最多。 FLU / melphalane和FLU / CY是最常见的组合。 RIC的这种极端异质性可能会影响程序的有效性和安全性,这需要进一步研究。 RIC的优化和标准化是为未来的前瞻性研究提供平台的最终目标。

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