包含生存的中位数与幸存者或始终幸存者的中位数:我们在测量什么?为什么?
The survival-incorporated median vs the median in the survivors or in the always-survivors: What are we measuring? and Why?
发表日期:2023 Oct 12
作者:
Qingyan Xiang, Ronald J Bosch, Judith J Lok
来源:
STATISTICS IN MEDICINE
摘要:
许多临床研究根据生存率和其他连续/顺序临床结果(例如生活质量评分)来评估治疗的益处。在这些研究中,当受试者在随访评估之前死亡时,临床结果变得不确定并因死亡而被缩短。将结果视为因死亡而“缺失”或“审查”可能会误导治疗效果评估。我们表明,如果我们使用幸存者或永远幸存者的中位数作为估计值来总结临床结果,我们可以得出结论,生存概率和良好临床结果之间存在权衡,即使在概率和良好临床结果之间存在权衡的情况下也是如此。一种治疗的生存率和任何良好临床结果的可能性都更好。因此,我们主张不要总是将死亡视为临床结果缺失的一种机制,而应将其作为结果衡量的一部分。为了说明生存状态,我们将生存合并中位数描述为死亡情况下结果的替代汇总衡量标准。生存合并中位数是一个阈值,50% 的人口存活且结果高于该阈值。通过概念性示例和前列腺癌治疗研究的应用,我们表明,生存合并中位数提供了一种简单而有用的汇总测量方法,可为临床实践提供信息。© 2023 John Wiley
Many clinical studies evaluate the benefit of a treatment based on both survival and other continuous/ordinal clinical outcomes, such as quality of life scores. In these studies, when subjects die before the follow-up assessment, the clinical outcomes become undefined and are truncated by death. Treating outcomes as "missing" or "censored" due to death can be misleading for treatment effect evaluation. We show that if we use the median in the survivors or in the always-survivors as estimands to summarize clinical outcomes, we may conclude that a trade-off exists between the probability of survival and good clinical outcomes, even in settings where both the probability of survival and the probability of any good clinical outcome are better for one treatment. Therefore, we advocate not always treating death as a mechanism through which clinical outcomes are missing, but rather as part of the outcome measure. To account for the survival status, we describe the survival-incorporated median as an alternative summary measure for outcomes in the presence of death. The survival-incorporated median is the threshold such that 50% of the population is alive with an outcome above that threshold. Through conceptual examples and an application to a prostate cancer treatment study, we show that the survival-incorporated median provides a simple and useful summary measure to inform clinical practice.© 2023 John Wiley & Sons Ltd.