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The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist…

Background: Effective allocation of limited donor lungs in cystic fibrosis (CF) requires accurate survival predictions, so that high-risk patients may be prioritized for transplantation. In practice, decisions about allocation are made…

Applications · Statistics 2017-06-30 Aasthaa Bansal , Nicole Mayer-Hamblett , Christopher H. Goss , Patrick J. Heagerty

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…

Methodology · Statistics 2019-02-27 Leah Comment , Fabrizia Mealli , Sebastien Haneuse , Corwin Zigler

Investigating the causal relationship between exposure and the time-to-event outcome is an important topic in biomedical research. Previous literature has discussed the potential issues of using the hazard ratio as a marginal causal effect…

Methodology · Statistics 2022-05-05 Zihan Lin , Ai Ni , Bo Lu

Chronic diseases are long-lasting conditions that require lifelong medical attention. Using big EMR data, we have developed early disease risk prediction models for five common chronic diseases: diabetes, hypertension, CKD, COPD, and…

Machine Learning · Computer Science 2026-03-13 Shaheer Ahmad Khan , Muhammad Usamah Shahid , Muddassar Farooq

The analysis of case-control studies with several subtypes of cases is increasingly common, e.g. in cancer epidemiology. For matched designs, we show that a natural strategy is based on a stratified conditional logistic regression model.…

Methodology · Statistics 2019-01-23 Nadim Ballout , Cedric Garcia , Vivian Viallon

In early-phase cancer clinical trials, the limited availability of data presents significant challenges in developing a framework to efficiently quantify treatment effectiveness. To address this, we propose a novel utility-based Bayesian…

Methodology · Statistics 2025-06-10 Saurabh Bhandari , Michael J. Daniels , Chenguang Wang

Purpose: To identify a clinically interpretable subset of survival-relevant features in HN cancer using Bayesian Network (BN) and evaluate its prognostic and causal utility. Methods and Materials: We used the RADCURE dataset, consisting of…

Aims: To propose a general sample size framework for developing or updating a clinical prediction model using any statistical or machine learning method, based on drawing samples from anticipated posterior distributions and targeting…

The determination of the sample size required by a crossover trial typically depends on the specification of one or more variance components. Uncertainty about the value of these parameters at the design stage means that there is often a…

Methodology · Statistics 2018-03-28 Michael Grayling , Adrian Mander , James Wason

Surrogate endpoints play an important role in drug development when they can be used to measure treatment effect early compared to the final clinical outcome and to predict clinical benefit or harm. Such endpoints are assessed for their…

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…

Methodology · Statistics 2021-12-21 Shuxi Zeng , Fan Li , Liangyuan Hu , Fan Li

When dealing with right-censored data, where some outcomes are missing due to a limited observation period, survival analysis -- known as time-to-event analysis -- focuses on predicting the time until an event of interest occurs. Multiple…

Machine Learning · Statistics 2024-10-23 Julie Alberge , Vincent Maladière , Olivier Grisel , Judith Abécassis , Gaël Varoquaux

Surrogate models based on machine learning methods have become an important part of modern engineering to replace costly computer simulations. The data used for creating a surrogate model are essential for the model accuracy and often…

Machine Learning · Statistics 2023-10-03 Sven Lämmle , Can Bogoclu , Kevin Cremanns , Dirk Roos

Prediction of survival for cancer patients is an open area of research. However, many of these studies focus on datasets with a large number of patients. We present a novel method that is specifically designed to address the challenge of…

Machine Learning · Computer Science 2015-09-30 Hamid Reza Hassanzadeh , John H. Phan , May D. Wang

Conventional survival analysis approaches estimate risk scores or individualized time-to-event distributions conditioned on covariates. In practice, there is often great population-level phenotypic heterogeneity, resulting from (unknown)…

Machine Learning · Statistics 2020-03-03 Paidamoyo Chapfuwa , Chunyuan Li , Nikhil Mehta , Lawrence Carin , Ricardo Henao

Mid-study design modifications are becoming increasingly accepted in confirmatory clinical trials, so long as appropriate methods are applied such that error rates are controlled. It is therefore unfortunate that the important case of…

Applications · Statistics 2014-05-08 Dominic Magirr , Thomas Jaki , Franz Koenig , Martin Posch

Survival prognosis is crucial for medical informatics. Practitioners often confront small-sized clinical data, especially cancer patient cases, which can be insufficient to induce useful patterns for survival predictions. This study deals…

Machine Learning · Computer Science 2025-01-23 Yonghao Zhao , Changtao Li , Chi Shu , Qingbin Wu , Hong Li , Chuan Xu , Tianrui Li , Ziqiang Wang , Zhipeng Luo , Yazhou He

Gene expression estimation from pathology images has the potential to reduce the RNA sequencing cost. Point-wise loss functions have been widely used to minimize the discrepancy between predicted and absolute gene expression values.…

Computer Vision and Pattern Recognition · Computer Science 2025-12-09 Kazuya Nishimura , Haruka Hirose , Ryoma Bise , Kaito Shiku , Yasuhiro Kojima

Survival prediction with small sets of features is a highly relevant topic for decision-making in clinical practice. I describe a method for predicting survival of amyotrophic lateral sclerosis (ALS) patients that was developed as a…

Applications · Statistics 2016-12-05 Christoph Kurz
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