无进展生存期和无病生存期等综合结果指标越来越多地用作肿瘤学研究中的替代终点，经常作为构成 FDA 和 EMA 批准基础的关键试验的主要终点。此类结果测量将两个或多个不同事件（例如，肿瘤（再）生长、新病变和/或死亡）组合成单个事件发生时间终点。使用复合终点可以提高临床试验的统计功效，并缩短证明疗效所需的随访时间，从而降低成本；然而，这些终点有许多限制。综合结果的定义通常很模糊，不同研究之间的定义差异很大，使得试验结果的比较变得复杂。改变复合结果中事件的构成可能会改变研究结论，包括治疗效果是否具有统计显着性。此外，复合结果中包含的事件通常具有不同的临床意义，反映不同的生物学途径和/或受到治疗的不同影响。因此，了解组成事件的精确细分对于准确解释试验结果和衡量干预的真正益处至关重要。然而，在肿瘤学临床试验中，很少提供此类信息。在本视角中，我们通过回顾发表在五种顶级肿瘤学期刊上的 50 项以无进展生存为结果的研究来强调这一缺陷，讨论使用复合终点的优点和挑战，并强调透明报告该组成部分的必要性events.© 2023。这是美国政府作品，在美国不受版权保护；外国版权保护可能适用。

Composite outcome measures such as progression-free survival and disease-free survival are increasingly used as surrogate end points in oncology research, frequently serving as the primary end point of pivotal trials that form the basis for FDA and EMA approvals. Such outcome measures combine two or more distinct events (for example, tumour (re)growth, new lesions and/or death) into a single, time-to-event end point. The use of a composite end point can increase the statistical power of a clinical trial and decrease the follow-up period required to demonstrate efficacy, thus lowering costs; however, these end points have a number of limitations. Composite outcomes are often vaguely defined, with definitions that vary greatly between studies, complicating comparisons of results across trials. Altering the makeup of events included in a composite outcome can alter study conclusions, including whether treatment effects are statistically significant. Moreover, the events included in a composite outcome often vary in clinical significance, reflect distinct biological pathways and/or are affected differently by treatment. Therefore, knowing the precise breakdown of the component events is essential to accurately interpret trial results and gauge the true benefit of an intervention. In oncology clinical trials, however, such information is rarely provided. In this Perspective, we emphasize this deficiency through a review of 50 studies with progression-free survival as an outcome published in five top oncology journals, discuss the advantages and challenges of using composite end points, and highlight the need for transparent reporting of the component events.© 2023. This is a U.S. Government work and not under copyright protection in the US; foreign copyright protection may apply.