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Meta-analysis, by synthesizing effect estimates from multiple studies conducted in diverse settings, stands at the top of the evidence hierarchy in clinical research. Yet, conventional approaches based on fixed- or random-effects models…

The focus of a survival study is partly on the distribution of survival times, and partly on the health or quality of life of patients while they live. Health varies over time, and survival is the most basic aspect of health, so the two…

Methodology · Statistics 2016-01-20 Walter Dempsey , Peter McCullagh

In biomedical studies it is of substantial interest to develop risk prediction scores using high-dimensional data such as gene expression data for clinical endpoints that are subject to censoring. In the presence of well-established…

Applications · Statistics 2011-11-24 Qi Long , Matthias Chung , Carlos S. Moreno , Brent A. Johnson

The participants in randomized trials and other studies used for causal inference are often not representative of the populations seen by clinical decision-makers. To account for differences between populations, researchers may consider…

Methodology · Statistics 2022-07-12 Anders Huitfeldt , Sonja A. Swanson , Mats Julius Stensrud , Etsuji Suzuki

Researchers often use linear regression to analyse randomized experiments to improve treatment effect estimation by adjusting for imbalances of covariates in the treatment and control groups. Our work offers a randomization-based inference…

Statistics Theory · Mathematics 2022-07-08 Hanzhong Liu , Yuehan Yang

Gun violence is a major source of injury and death in the United States. However, relatively little is known about the effects of firearm injuries on survivors and their family members and how these effects vary across subpopulations. To…

Applications · Statistics 2024-06-04 Eric R. Cohn , Zirui Song , Jose R. Zubizarreta

Background Survival extrapolation is essential in the cost-effectiveness analysis to quantify the lifetime survival benefit associated with a new intervention, due to the restricted duration of randomized controlled trials (RCTs). Current…

Methodology · Statistics 2022-06-02 Zhaojing Che , Nathan Green , Gianluca Baio

With an increasing focus on precision medicine in medical research, numerous studies have been conducted in recent years to clarify the relationship between treatment effects and patient characteristics. The treatment effects for patients…

Methodology · Statistics 2023-09-22 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

In many applications of survival data analysis, the individuals are treated in different medical centres or belong to different clusters defined by geographical or administrative regions. The analysis of such data requires accounting for…

Methodology · Statistics 2022-05-03 Rubio , F. J. , Drikvandi , R

Mediation analysis for survival outcomes is challenging. Most existing methods quantify the treatment effect using the hazard ratio (HR) and attempt to decompose the HR into the direct effect of treatment plus an indirect, or mediated,…

The most popular approach for analyzing survival data is the Cox regression model. The Cox model may, however, be misspecified, and its proportionality assumption may not always be fulfilled. An alternative approach for survival prediction…

Machine Learning · Statistics 2018-05-17 Marvin N. Wright , Theresa Dankowski , Andreas Ziegler

The restricted mean survival time (RMST) has become a popular measure to summarize event times in longitudinal studies. Defined as the area under the survival function up to a time horizon $\tau$ > 0, the RMST can be interpreted as the life…

Methodology · Statistics 2024-11-05 Alina Schenk , Vanessa Basten , Matthias Schmid

Factorial analyses offer a powerful nonparametric means to detect main or interaction effects among multiple treatments. For survival outcomes, e.g. from clinical trials, such techniques can be adopted for comparing reasonable…

Methodology · Statistics 2023-02-06 Takeshi Emura , Marc Ditzhaus , Dennis Dobler , Kenta Murotani

Methods for estimating heterogeneous treatment effects (HTE) from observational data have largely focused on continuous or binary outcomes, with less attention paid to survival outcomes and almost none to settings with competing risks. In…

Methodology · Statistics 2024-09-30 Shenbo Xu , Raluca Cobzaru , Stan N. Finkelstein , Roy E. Welsch , Kenney Ng , Zach Shahn

Forest-based methods have recently gained in popularity for non-parametric treatment effect estimation. Building on this line of work, we introduce causal survival forests, which can be used to estimate heterogeneous treatment effects in a…

Methodology · Statistics 2023-03-01 Yifan Cui , Michael R. Kosorok , Erik Sverdrup , Stefan Wager , Ruoqing Zhu

Predicting patient survival probabilities based on observed covariates is an important assessment in clinical practice. These patient-specific covariates are often measured over multiple follow-up appointments. It is then of interest to…

Methodology · Statistics 2021-11-11 Annabel L. Davies , Anthony C. C. Coolen , Tobias Galla

Motivation: Identification of genomic, molecular and clinical markers prognostic of patient survival is important for developing personalized disease prevention, diagnostic and treatment approaches. Modern omics technologies have made it…

Applications · Statistics 2024-03-05 Zhi Zhao , John Zobolas , Manuela Zucknick , Tero Aittokallio

Clustered observations are ubiquitous in controlled and observational studies and arise naturally in multi-centre trials or longitudinal surveys. We present a novel model for the analysis of clustered observations where the marginal…

Methodology · Statistics 2022-11-03 Luisa Barbanti , Torsten Hothorn

Survival analysis is a challenging variation of regression modeling because of the presence of censoring, where the outcome measurement is only partially known, due to, for example, loss to follow up. Such problems come up frequently in…

Machine Learning · Computer Science 2022-06-28 Chirag Nagpal , Steve Yadlowsky , Negar Rostamzadeh , Katherine Heller

Determining the extent to which a patient is benefiting from cancer therapy is challenging. Criteria for quantifying the extent of "tumor response" observed within a few cycles of treatment have been established for various types of solid…

Methodology · Statistics 2020-09-18 Jie Zhou , Xun Jiang , H. Amy Xia , Peng Wei , Brian P. Hobbs