Zhao Ying-Qi, Redman Mary W, LeBlanc Michael L
Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
Stat Med. 2019 Dec 10;38(28):5317-5331. doi: 10.1002/sim.8363. Epub 2019 Sep 9.
The hazard ratio is widely used to measure or to summarize the magnitude of treatment effects, but it is justifiably difficult to interpret in a meaningful way to patients and perhaps for clinicians as well. In addition, it is most meaningful when the hazard functions are approximately proportional over time. We propose a new measure, termed personalized chance of longer survival. The measure, which quantifies the probability of living longer with one treatment over the another, accounts for individualized characteristics to directly address personalized treatment effects. Hence, the measure is patient focused, which can be used to evaluate subgroups easily. We believe it is intuitive to understand and clinically interpretable in the presence of nonproportionality. Furthermore, because it estimates the probability of living longer by some fixed amount of time, it encodes the probabilistic part of treatment effect estimation. We provide nonparametric estimation and inference procedures that can accommodate censored survival outcomes. We conduct extensive simulation studies, which characterize performance of the proposed method, and data from a large randomized Phase III clinical trial (SWOG S0819) are analyzed using the proposed method.
风险比被广泛用于衡量或总结治疗效果的大小,但要以一种对患者有意义的方式进行解释,甚至对临床医生来说可能也很困难。此外,当风险函数随时间大致成比例时,它才最有意义。我们提出了一种新的度量方法,称为个性化长期生存机会。该度量方法量化了接受一种治疗比另一种治疗活得更长的概率,考虑了个体特征以直接解决个性化治疗效果问题。因此,该度量方法以患者为中心,可轻松用于评估亚组。我们认为它直观易懂,在存在不成比例的情况下具有临床可解释性。此外,由于它估计了在一定固定时间内活得更长的概率,它对治疗效果估计中的概率部分进行了编码。我们提供了可以处理删失生存结局的非参数估计和推断程序。我们进行了广泛的模拟研究,以描述所提出方法的性能,并使用所提出的方法分析了来自一项大型随机III期临床试验(SWOG S0819)的数据。