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Patient-centered outcomes, such as quality of life and length of hospital stay, are the focus in a wide array of clinical studies. However, participants in randomized trials for elderly or critically and severely ill patient populations may…

统计方法学 · 统计学 2024-04-17 Dane Isenberg , Michael Harhay , Nandita Mitra , Fan Li

In semicompeting risks problems, nonterminal time-to-event outcomes such as time to hospital readmission are subject to truncation by death. These settings are often modeled with illness-death models for the hazards of the terminal and…

统计方法学 · 统计学 2019-02-27 Leah Comment , Fabrizia Mealli , Sebastien Haneuse , Corwin Zigler

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

Objective: Randomised controlled trials (RCTs) are widely considered as gold standard for assessing the effectiveness of new health interventions. When treatment non-compliance is present in RCTs, the treatment effect in the subgroup of…

应用统计 · 统计学 2025-03-25 Theodosios Papazoglou , Ed Waddingham , Alastair Young

Even in a carefully designed randomized trial, outcomes for some study participants can be missing, or more precisely, ill-defined, because participants had died prior to date of outcome collection. This problem, known as truncation by…

统计方法学 · 统计学 2022-09-27 Tamir Zehavi , Daniel Nevo

In cluster-randomized crossover (CRXO) trials, groups of individuals are randomly assigned to two or more sequences of alternating treatments. Since clusters serve as their own control, the CRXO design is typically more statistically…

统计方法学 · 统计学 2026-01-30 Dane Isenberg , Michael O. Harhay , Andrew B. Forbes , Paul J. Young , Fan Li , Nandita Mitra

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

Cluster-randomized trials (CRTs) on fragile populations frequently encounter complex attrition problems where the reasons for missing outcomes can be heterogeneous, with participants who are known alive, known to have died, or with unknown…

统计方法学 · 统计学 2025-05-06 Guangyu Tong , Chenxi Li , Eric Velazquez , Michael O. Harhay , Fan Li

We derive new variance formulas for inference on a general class of estimands of causal average treatment effects in a Randomized Control Trial (RCT). We generalize Robins (1988) and show that when the estimand of interest is the Sample…

统计理论 · 数学 2017-10-19 Jasjeet S. Sekhon , Yotam Shem-Tov

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

Defining a causal estimand for a longitudinal outcome truncated by death is challenging, because the outcome may be undefined at the end of follow-up. Although a range of estimands and several estimators have been proposed, guidance on the…

统计方法学 · 统计学 2026-04-30 Juliette Ortholand , Young Lee , Marie-Abele C Bind

Randomised controlled trials in reproductive medicine are often subject to outcome truncation, where study outcomes are only defined in a subset of participants. Examples include birthweight (measurable only in the subgroup of participants…

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

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

机器学习 · 统计学 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

Detecting heterogeneity in treatment response enriches the interpretation of gerontologic trials. In aging research, estimating the effect of the intervention on clinically meaningful outcomes faces analytical challenges when it is…

应用统计 · 统计学 2026-01-08 Changjun Li , Heather Allore , Michael O. Harhay , Fan Li , Guangyu Tong

Competing risk is a common phenomenon when dealing with time-to-event outcomes in biostatistical applications. An attractive estimand in this setting is the "number of life-years lost due to a specific cause of death", Andersen et al.…

统计方法学 · 统计学 2025-02-04 Simon Christoffer Ziersen , Torben Martinussen

One of the major challenges in estimating conditional potential outcomes and conditional average treatment effects (CATE) is the presence of hidden confounders. Since testing for hidden confounders cannot be accomplished only with…

机器学习 · 计算机科学 2025-06-17 Ahmed Aloui , Juncheng Dong , Ali Hasan , Vahid Tarokh

Confounding bias and selection bias bring two significant challenges to the validity of conclusions drawn from applied causal inference. The latter can stem from informative missingness, such as in cases of attrition. We introduce the…

统计方法学 · 统计学 2025-03-25 Johan de Aguas , Johan Pensar , Tomás Varnet Pérez , Guido Biele

Treatment effect estimates are often available from randomized controlled trials as a single average treatment effect for a certain patient population. Estimates of the conditional average treatment effect (CATE) are more useful for…

统计方法学 · 统计学 2023-09-12 Wouter A. C. van Amsterdam , Rajesh Ranganath

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
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