Novel Composite Endpoints after Allogeneic Hematopoietic Cell Transplantation.

Novel Composite Endpoints after Allogeneic Hematopoietic Cell Transplantation.
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DOI:
10.1016/j.jtct.2021.05.005
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发表时间:
2021-08
影响因子:
3.2
通讯作者:
Weisdorf DJ
Weisdorf DJ
中科院分区:
医学2区
文献类型:
--
作者:
Kim HT;Logan B;Weisdorf DJ

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随着近年来用于评价异基因造血细胞移植(alloHCT)结局的移植特异性复合终点的发展,这些新终点的使用正在迅速增长。将多个终点组合成一个终点,这些复合终点看起来很简单,可以用作干预措施总体有效性的汇总指标。然而,所有组分终点可能不具有相同的临床意义,并且干预可能不会在复合终点的所有组分的相同方向上成比例地起作用。这可能会使结果的解释复杂化,特别是如果不同组分终点存在相反的影响。我们评估了最近alloHCT研究中使用的各种复合终点的益处和局限性,并提出了其使用和解释的指南。
With the recent development of transplant-specific composite endpoints for evaluation of allogeneic hematopoietic cell transplantation (alloHCT) outcomes, the use of these novel endpoints is growing rapidly. Combining multiple endpoints into a single endpoint, these composite endpoints appear simple and can be used as a summary measure for overall effectiveness of an intervention. However, all component endpoints may not have equal clinical significance and an intervention may not work proportionally in the same direction for all components of a composite endpoint. This may complicate the interpretation of results, particularly if there are opposing effects of differing component endpoints. We assess the benefits and limitations of various composite endpoints used in alloHCT studies recently and propose guidelines for their use and interpretation.
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