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To draw real-world evidence about the comparative effectiveness of multiple time-varying treatments on patient survival, we develop a joint marginal structural survival model and a novel weighting strategy to account for time-varying…

统计方法学 · 统计学 2023-08-08 Liangyuan Hu , Jiayi Ji , Himanshu Joshi , Erick Scott , Fan Li

Restricted mean survival time (RMST) is gaining attention as a measure to quantify the treatment effect on survival outcomes in randomized clinical trials. Several methods to determine sample size based on the RMST-based tests have been…

统计方法学 · 统计学 2022-12-19 Satoshi Hattori , Hajime Uno

Medical advances have increased cancer survival rates and the possibility of finding a cure. Hence, it is crucial to evaluate the impact of treatments both in terms of cure and prolongation of survival. To achieve this, we may use a Cox…

统计方法学 · 统计学 2024-12-31 Marta Cipriani , Marta Fiocco , Marco Alfò , Maria Quelhas , Eni Musta

Population-adjusted indirect comparisons estimate treatment effects when access to individual patient data is limited and there are cross-trial differences in effect modifiers. Popular methods include matching-adjusted indirect comparison…

应用统计 · 统计学 2021-11-05 Antonio Remiro-Azócar , Anna Heath , Gianluca Baio

In this study, we consider sequences drawn from time-homogeneous Markov chains and introduce a novel approach for estimating first hitting-time distributions to specified terminal states. Our method- ology is based on the…

统计理论 · 数学 2026-05-19 Mikael Escobar-Bach , Alexandre Popier , Malo Sahin

The log-rank test and the Cox proportional hazards model are commonly used to compare time-to-event data in clinical trials, as they are most powerful under proportional hazards. But there is a loss of power if this assumption is violated,…

统计方法学 · 统计学 2024-02-14 Jonas Brugger , Tim Friede , Florian Klinglmüller , Martin Posch , Robin Ristl , Franz König

In observational studies of survival time featuring a binary time-dependent treatment, the hazard ratio (an instantaneous measure) is often used to represent the treatment effect. However, investigators are often more interested in the…

统计方法学 · 统计学 2020-05-21 Yun Li , Douglas E. Schaubel , Kevin He

Researchers are often interested in treatment effects on outcomes that are only defined conditional on a post-treatment event status. For example, in a study of the effect of different cancer treatments on quality of life at end of…

We consider the problem of estimating the distribution of time-to-event data that are subject to censoring and for which the event of interest might never occur, i.e., some subjects are cured. To model this kind of data in the presence of…

统计理论 · 数学 2018-06-05 François Portier , Ingrid Van Keilegom , Anouar El Ghouch

To use historical controls for indirect comparison with single-arm trials, the population difference between data sources should be adjusted to reduce confounding bias. The adjustment is more difficult for time-to-event data with a cure…

统计方法学 · 统计学 2023-08-23 Jixian Wang , Hongtao Zhang , Ram Tiwari

In this work, we aim to predict the survival time (ST) of glioblastoma (GBM) patients undergoing different treatments based on preoperative magnetic resonance (MR) scans. The personalized and precise treatment planning can be achieved by…

计算机视觉与模式识别 · 计算机科学 2024-02-13 Xiaofeng Liu , Nadya Shusharina , Helen A Shih , C. -C. Jay Kuo , Georges El Fakhri , Jonghye Woo

Modeling clustered/correlated failure time data has been becoming increasingly important in clinical trials and epidemiology studies. In this paper, we consider a semiparametric marginal promotion time cure model for clustered…

统计方法学 · 统计学 2025-05-15 Fei Xiao , Yingwei Peng , Dipankar Bandyopadhyayd , Yi Niu

Effectiveness of immune-oncology chemotherapies has been presented in recent clinical trials. The Kaplan-Meier estimates of the survival functions of the immune therapy and the control often suggested the presence of the lag-time until the…

应用统计 · 统计学 2024-07-26 Hisato Sunami , Satoshi Hattori

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…

统计方法学 · 统计学 2022-11-03 Luisa Barbanti , Torsten Hothorn

Meta-analyses of survival studies aim to reveal the variation of an effect measure of interest over different studies and present a meaningful summary. They must address between study heterogeneity in several dimensions and eliminate…

统计方法学 · 统计学 2021-11-29 Joris Hautekiet , Elena-Sophie Prigge , Theresa Obermueller , Marc Arbyn , Els Goetghebeur

In a clinical trial with a survival outcome, an interim analysis is often performed to allow for early stopping for efficacy. If the interim analysis is early in the trial, one might conclude that a new treatment is more effective (compared…

统计方法学 · 统计学 2023-05-09 Marianne A Jonker , Steven Teerenstra

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes…

统计方法学 · 统计学 2021-12-21 Shuxi Zeng , Fan Li , Liangyuan Hu , Fan Li

An aspect of interest in surveillance of diseases is whether the survival time distribution changes over time. By following data in health registries over time, this can be monitored, either in real time or retrospectively. With relevant…

应用统计 · 统计学 2025-03-10 Jimmy Huy Tran , Jan Terje Kvaløy , Hartwig Kørner

Inferring treatment effects on a survival time outcome based on data from an observational study is challenging due to the presence of censoring and possible confounding. An additional challenge occurs when a unit's treatment affects the…

统计方法学 · 统计学 2025-08-13 Chanhwa Lee , Donglin Zeng , Michael Emch , John D. Clemens , Michael G. Hudgens

For measuring the strength of visually-observed subpopulation differences, the Population Difference Criterion is proposed to assess the statistical significance of visually observed subpopulation differences. It addresses the following…

统计方法学 · 统计学 2023-09-21 Xi Yang , Jan Hannig , Katherine A. Hoadley , Iain Carmichael , J. S. Marron