Department of Data Science, Dana Farber Cancer Institute, Harvard School of Public Health, Boston, Massachusetts.
Center for International Blood and Marrow Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
Transplant Cell Ther. 2021 Aug;27(8):650-657. doi: 10.1016/j.jtct.2021.05.005. Epub 2021 May 15.
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. © 2021 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.
随着用于评估异基因造血细胞移植 (alloHCT) 结果的移植特异性综合终点的最新发展，这些新终点的使用正在迅速增加。这些综合终点将多个终点结合到一个单一终点中，看起来很简单，可以作为干预措施总体有效性的综合衡量标准。然而，并非所有的终点都具有同等的临床意义，而且干预措施可能不会以相同的比例在综合终点的所有组成部分中发挥作用。如果不同组成部分的终点有相反的效果，这可能会使结果的解释变得复杂。我们评估了最近 alloHCT 研究中使用的各种综合终点的优缺点，并提出了使用和解释这些终点的指南。
2021 年美国血液和骨髓移植学会。版权所有。
