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arXiv 2401.03084stat.ME
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右删失结果下最优治疗规则估计量的有限样本性能

Finite sample performance of optimal treatment rule estimators with right-censored outcomes

Michael Jetsupphasuk, Michael G. Hudgens, Jessie K. Edwards, Stephen R. Cole

AI总结:

本文通过模拟比较右删失观测生存数据下多种最优治疗规则及期望结果估计方法,发现灵活建模结果的方法在非线性规则等情景中表现更优。

摘要:

当存在治疗效应异质性时,根据患者特征推荐治疗方案可能改善患者护理。近来,大量关注集中于估计使期望结果最大化的最优治疗规则。然而,对于结果存在右删失的情形,特别是在估计量的实际使用方面,所受关注相对较少。本研究开展模拟,以评估最优治疗规则估计量以及治疗规则下期望结果估计量的有限样本性能。这些模拟受到生物医学和公共卫生研究中常见情形的推动:数据为观测性数据,生存时间可能被右删失,并且研究者有兴趣估计基线治疗决策以最大化生存概率。研究在一系列模拟情景下,比较了用于最优治疗规则估计的多种结果回归和直接搜索估计方法。能够灵活建模结果的方法表现相对较好,包括在治疗规则为非线性的情景中。用于复现本研究结果的 R 代码已在 Github 上提供。

英文摘要:

Patient care may be improved by recommending treatments based on patient characteristics when there is treatment effect heterogeneity. Recently, there has been a great deal of attention focused on the estimation of optimal treatment rules that maximize expected outcomes. However, there has been comparatively less attention given to settings where the outcome is right-censored, especially with regard to the practical use of estimators. In this study, simulations were undertaken to assess the finite-sample performance of estimators for optimal treatment rules and estimators for the expected outcome under treatment rules. The simulations were motivated by the common setting in biomedical and public health research where the data is observational, survival times may be right-censored, and there is interest in estimating baseline treatment decisions to maximize survival probability. A variety of outcome regression and direct search estimation methods were compared for optimal treatment rule estimation across a range of simulation scenarios. Methods that flexibly model the outcome performed comparatively well, including in settings where the treatment rule was non-linear. R code to reproduce this study's results are available on Github.

机构、总结、中文摘要均由AI分析而来,可能存在错误,请以论文正文为准。

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