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相关论文: Estimating treatment effects from observational da…

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Researchers are often interested in estimating effects of generalized time-varying treatment strategies on the mean of an outcome at one or more selected follow-up times of interest. For example, the Medications and Weight Gain in PCORnet…

Clinical studies sometimes encounter truncation by death, rendering outcomes undefined. Statistical analysis based solely on observed survivors may give biased results because the characteristics of survivors differ between treatment…

统计方法学 · 统计学 2022-11-23 Yuhao Deng , Yingjun Chang , Xiao-Hua Zhou

We consider the problem of estimating quantile treatment effects without assuming strict overlap , i.e., we do not assume that the propensity score is bounded away from zero. More specifically, we consider an inverse probability weighting…

统计理论 · 数学 2026-02-24 Marco Avella-Medina , Richard Davis , Gennady Samorodnitsky

Medication adherence is essential to ensure treatment effectiveness, but too often in routine care non-adherence compromises the desired outcome. We explore longitudinal causal modelling using observational data to estimate the time-varying…

统计方法学 · 统计学 2026-03-10 Xiaoran Liang , Deniz Türkmen , Jane A H Masoli , Luke C Pilling , Jack Bowden

Intercurrent events, such as treatment switching, rescue medication, dropout, or truncation by death, frequently complicate intention-to-treat analyses in randomized clinical trials. Existing causal inference frameworks typically target…

统计方法学 · 统计学 2026-03-12 Georgi Baklicharov , Kelly Van Lancker , Stijn Vansteelandt

In some randomized clinical trials, patients may die before the measurements of their outcomes. Even though randomization generates comparable treatment and control groups, the remaining survivors often differ significantly in background…

应用统计 · 统计学 2018-03-07 Fan Yang , Peng Ding

While the inverse probability of treatment weighting (IPTW) is a commonly used approach for treatment comparisons in observational data, the resulting estimates may be subject to bias and excessively large variance when there is lack of…

统计方法学 · 统计学 2024-02-13 Zhiqiang Cao , Lama Ghazi , Claudia Mastrogiacomo , Laura Forastiere , F. Perry Wilson , Fan Li

In clinical trials, principal stratification analysis is commonly employed to address the issue of truncation by death, where a subject dies before the outcome can be measured. However, in practice, many survivor outcomes may remain…

统计方法学 · 统计学 2025-07-08 Wei Li , Yuan Liu , Shanshan Luo , Zhi Geng

In a widely cited paper, Xie and Liu (henceforth XL) proposed to use inverse probability of treatment weighting (IPTW) to account for possible confounding in observational studies with survival endpoints subject to right censoring. Their…

统计方法学 · 统计学 2025-11-04 Zhiwei Zhang , Yongwu Shao , Zhishen Ye

In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for…

统计方法学 · 统计学 2023-06-27 Wenjie Hu , Xiaohua Zhou , Peng Wu

Longitudinal observational patient data can be used to investigate the causal effects of time-varying treatments on time-to-event outcomes. Several methods have been developed for controlling for the time-dependent confounding that…

统计方法学 · 统计学 2021-10-08 Ruth H. Keogh , Jon Michael Gran , Shaun R. Seaman , Gwyneth Davies , Stijn Vansteelandt

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…

应用统计 · 统计学 2023-09-06 Qingyan Xiang , Ronald J. Bosch , Judith J. Lok

The analysis of causal effects when the outcome of interest is possibly truncated by death has a long history in statistics and causal inference. The survivor average causal effect is commonly identified with more assumptions than those…

统计方法学 · 统计学 2020-03-24 Jaffer M. Zaidi , Eric J. Tchetgen Tchetgen , Tyler J. VanderWeele

Death among subjects is common in observational studies evaluating the causal effects of interventions among geriatric or severely ill patients. High mortality rates complicate the comparison of the prevalence of adverse events (AEs)…

统计方法学 · 统计学 2024-10-08 Anthony Sisti , Andrew Zullo , Roee Gutman

It is common in medical studies that the outcome of interest is truncated by death, meaning that a subject has died before the outcome could be measured. In this case, restricted analysis among survivors may be subject to selection bias.…

统计方法学 · 统计学 2018-04-25 Linbo Wang , Xiao-Hua Zhou , Thomas S. Richardson

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable…

统计方法学 · 统计学 2025-09-01 Jiaqi Tong , Chao Cheng , Guangyu Tong , Michael O. Harhay , Fan Li

Observational data have been actively used to estimate treatment effect, driven by the growing availability of electronic health records (EHRs). However, EHRs typically consist of longitudinal records, often introducing time-dependent…

机器学习 · 计算机科学 2024-06-14 Junghwan Lee , Simin Ma , Nicoleta Serban , Shihao Yang

Continuous outcome measurements truncated by death present a challenge for the estimation of unbiased treatment effects in randomized controlled trials (RCTs). One way to deal with such situations is to estimate the survivor average causal…

统计方法学 · 统计学 2025-12-02 Stefanie von Felten , Chiara Vanetta , Christoph M. Rüegger , Sven Wellmann , Leonhard Held

Inverse probability of treatment weighting (IPTW) is widely used to estimate causal effects, but guidance is limited for count exposures. It is also unclear how IPTW performs when combined with multiple imputation in this context. In this…

统计方法学 · 统计学 2026-03-26 Martin N. Danka , Jessica K. Bone , George B. Ploubidis , Richard J. Silverwood

In longitudinal observational studies, marginal structural models (MSMs) are a class of causal models used to analyse the effect of an exposure on the (time-to-event) outcome of interest, while accounting for exposure-affected…

统计方法学 · 统计学 2025-11-04 Marta Spreafico
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