Patient Centered Hazard Ratio Estimation Using Principal Stratification Weights: Application to the NORCCAP Randomized Trial of Colorectal Cancer Screening
Patient Centered Hazard Ratio Estimation Using Principal Stratification Weights: Application to the NORCCAP Randomized Trial of Colorectal Cancer Screening
复制标题
使用主分层权重以患者为中心的风险比估计：在 NORCCAP 结直肠癌筛查随机试验中的应用
DOI：
10.1353/obs.2016.0004
复制
发表时间：
2016
期刊：
影响因子：
--
通讯作者：
A. J. O’Malley
中科院分区：
文献类型：
--
作者：
T. Mackenzie;M. Løberg;A. J. O’Malley
摘要：在随机试验中，最常报道的疗效评估方法是意向治疗(ITT)，其次是方案。ITT是首选的，因为它是治疗分配效果的无偏估计器。然而，如果有任何不坚持，ITT是对治疗效果的有偏见的估计，定义为如果治疗的潜在结果与如果不治疗的潜在结果之间的对比。治疗效果与患者最为相关。主要分层是一个评估治疗效果的框架，它结合了潜在的结局和潜在的依从层。它对编者平均因果效应(CACE)产生了一个无偏见的估计器，在全有或全无坚持的情况下，不同的方法或比例不同。本文讨论了在对事件发生的时间进行正确审查的情况下，编写者的因果危险比的估计。我们提出了一种使用权重来实现主体分层的新方法。我们报告了改变依从量和选择偏倚的模拟结果，这些结果表明我们提出的危险比估计器与ITT和每种方案的估计器相比具有最小的偏差。我们使用一项基于人群的随机对照试验对结直肠癌筛查进行了验证，该试验受控于筛查臂中高频率的不依从性。
Abstract:In randomized trials, the most commonly reported method of effect estimation is intention- to-treat (ITT), and to a lesser extent the per-protocol. The ITT is preferred because it is an unbiased estimator of the effect of treatment assignment. However, if there is any non-adherence the ITT is a biased estimate of the treatment effect, defined as the contrast between the potential outcome if treated versus the potential outcome if not treated. The treatment effect is most relevant to patients. Principal stratification is a framework for estimating treatment effects that combines potential outcomes and latent adherence strata. It yields an unbiased estimator of the complier average causal effect (CACE) for a difference in means or proportions, in the setting of all-or-nothing adherence. This paper addresses estimation of the causal hazard ratio for the compliers in a setting of right censoring of a time-to-event. We propose a novel approach to operationalizing principal stratification using weights. We report the results of simulations that vary the amount of adherence and selection bias that show the hazard ratio estimators we propose have minimal bias compared to the ITT, and per-protocol estimators. We demonstrate the approach using a population based randomized controlled trial of colorectal cancer screening subject to a high frequency of nonadherence in the screening arm.
影响因子：
120.7
作者：
Stukel, Therese A.;Fisher, Elliott S.;Vermeulen, Marian J.
通讯作者：
Vermeulen, Marian J.
DOI：
10.1056/nejmoa1414293
发表时间：
2015-05-28
期刊：
The New England journal of medicine
影响因子：
--
作者：
Halpern SD;French B;Small DS;Saulsgiver K;Harhay MO;Audrain-McGovern J;Loewenstein G;Brennan TA;Asch DA;Volpp KG
通讯作者：
Volpp KG
影响因子：
120.7
作者：
Holme, Oyvind;Loberg, Magnus;Kalager, Mette;Bretthauer, Michael;Hernan, Miguel A.;Aas, Eline;Eide, Tor J.;Skovlund, Eva;Schneede, Jorn;Tveit, Kjell Magne;Hoff, Geir
通讯作者：
Hoff, Geir