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As standards of care advance, patients are living longer and once-fatal diseases are becoming manageable. Clinical trials increasingly focus on reducing disease burden, which can be quantified by the timing and occurrence of multiple…

In recent years, cancer clinical trials have increasingly encountered non proportional hazards (NPH) scenarios, particularly with the emergence of immunotherapy. In randomized controlled trials comparing immunotherapy with conventional…

应用统计 · 统计学 2025-02-28 Takuto Iijima , Tomotaka Momozaki , Shuji Ando

What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has…

统计方法学 · 统计学 2023-04-18 José L. Jiménez , Isobel Barrott , Francesca Gasperoni , Dominic Magirr

Analysis of lifetime data from epidemiological studies or destructive testing often involves current status censoring, wherein individuals are examined only once and their event status is recorded only at that specific time point. In…

统计方法学 · 统计学 2024-10-15 Pavithra Hariharan , P. G. Sankaran

Multi-state models provide an extension of the usual survival/event-history analysis setting. In the medical domain, multi-state models give the possibility of further investigating intermediate events such as relapse and remission. In this…

统计方法学 · 统计学 2021-06-24 D. Manevski , H. Putter , M. Pohar Perme , E. F. Bonneville , J. Schetelig , L. C. de Wreede

In population-based cancer survival analysis, the net survival is important for government to assess health care programs. For decades, it is observed that the net survival reaches a plateau after long-term follow-up, this is so called…

统计方法学 · 统计学 2020-09-29 Yueh Wang , Hung Hung

Background: Randomized controlled trials (RCTs) are costly, time-consuming, and often infeasible, while treatment-effect estimation from observational data is limited by unobserved confounding. Methods: We developed a three-step framework…

Restricted mean survival time (RMST) models have gained popularity when analyzing time-to-event outcomes because RMST models offer more straightforward interpretations of treatment effects with fewer assumptions than hazard ratios commonly…

统计方法学 · 统计学 2023-10-23 Kaiyuan Hua , Xiaofei Wang , Hwanhee Hong

We propose a novel methodology to quantify the effect of stochastic interventions on non-terminal time-to-events that lie on the pathway between an exposure and a terminal time-to-event outcome. Investigating these effects is particularly…

统计方法学 · 统计学 2024-07-17 Linda Valeri , Cécile Proust-Lima , Weijia Fan , Jarvis T. Chen , Hélène Jacqmin-Gadda

Analysing subgroups defined by biomarkers is of increasing importance in clinical research. In some situations the biomarker is subject to misclassification error, meaning the true subgroups are identified with imperfect sensitivity and…

统计方法学 · 统计学 2018-01-16 Fang Wan , Andrew C. Titman , Thomas F. Jaki

In the absence of data from a randomized trial, researchers often aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the…

统计方法学 · 统计学 2021-06-15 Ted Westling , Alex Luedtke , Peter Gilbert , Marco Carone

The progression-free survival ratio (PFSr) is a widely used measure in personalized oncology trials. It evaluates the effectiveness of treatment by comparing two consecutive event times - one under standard therapy and one under an…

统计方法学 · 统计学 2025-12-23 Merle Munko , Simon Mack , Marc Ditzhaus , Stefan Fröhling , Dennis Dobler , Dominic Edelmann

In medicine, survival analysis studies the time duration to events of interest such as mortality. One major challenge is how to deal with multiple competing events (e.g., multiple disease diagnoses). In this work, we propose a…

机器学习 · 计算机科学 2022-06-29 Zifeng Wang , Jimeng Sun

We consider survival data in the presence of a cure fraction, meaning that some subjects will never experience the event of interest. We assume a mixture cure model consisting of two sub-models: one for the probability of being uncured…

统计方法学 · 统计学 2023-03-17 Eni Musta , Tsz Pang Yuen

To address an important risk classification issue that arises in clinical practice, we propose a new mixture model via latent cure rate markers for survival data with a cure fraction. In the proposed model, the latent cure rate markers are…

应用统计 · 统计学 2009-10-12 Sungduk Kim , Yingmei Xi , Ming-Hui Chen

Balanced representation learning methods have been applied successfully to counterfactual inference from observational data. However, approaches that account for survival outcomes are relatively limited. Survival data are frequently…

The restricted mean survival time (RMST) model has been garnering attention as a way to provide a clinically intuitive measure: the mean survival time. RMST models, which use methods based on pseudo time-to-event values and inverse…

统计方法学 · 统计学 2024-06-11 Keisuke Hanada , Masahiro Kojima

Mixture cure models have been widely used to analyze survival data with a cure fraction. They assume that a subgroup of the individuals under study will never experience the event (cured subjects). So, the goal is twofold: to study both the…

统计方法学 · 统计学 2024-02-01 Ana López-Cheda , M. Amalia Jácome , Ignacio López-de-Ullibarri

In clinical follow-up studies with a time-to-event end point, the difference in the restricted mean survival time (RMST) is a suitable substitute for the hazard ratio (HR). However, the RMST only measures the survival of patients over a…

统计方法学 · 统计学 2023-06-27 Zijing Yang , Chengfeng Zhang , Yawen Hou , Zheng Chen

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