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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

Time to an event of interest over a lifetime is a central measure of the clinical benefit of an intervention used in a health technology assessment (HTA). Within the same trial, multiple end-points may also be considered. For example,…

Applications · Statistics 2026-01-13 Nathan Green , Murat Kurt , Andriy Moshyk , James Larkin , Gianluca Baio

Survival models are used in various fields, such as the development of cancer treatment protocols. Although many statistical and machine learning models have been proposed to achieve accurate survival predictions, little attention has been…

Machine Learning · Computer Science 2020-03-26 Hrushikesh Loya , Pranav Poduval , Deepak Anand , Neeraj Kumar , Amit Sethi

This paper primarily addresses a dataset relating to cellular, chemical and physical conditions of patients gathered at the time they are operated upon to remove colorectal tumours. This data provides a unique insight into the biochemical…

Machine Learning · Computer Science 2016-11-17 Christopher Roadknight , Durga Suryanarayanan , Uwe Aickelin , John Scholefield , Lindy Durrant

Predicting cancer dynamics under treatment is challenging due to high inter-patient heterogeneity, lack of predictive biomarkers, and sparse and noisy longitudinal data. Mathematical models can summarize cancer dynamics by a few…

Quantitative Methods · Quantitative Biology 2024-05-24 Even Moa Myklebust , Arnoldo Frigessi , Fredrik Schjesvold , Jasmine Foo , Kevin Leder , Alvaro Köhn-Luque

Heterogeneous treatment effect estimation is critical in oncology, particularly in multi-arm trials with overlapping therapeutic components and long-term survivors. These shared mechanisms pose a central challenge to identifying causal…

Methodology · Statistics 2025-10-29 Peter Chang , John Kairalla , Arkaprava Roy

Complex data features, such as unmodelled censored event times and variables with time-dependent effects, are common in cancer recurrence studies and pose challenges for Bayesian survival modelling. Current methodologies for predictive…

Methodology · Statistics 2026-01-12 Saku Suorsa , Aki Vehtari

Patient-level health economic data collected alongside clinical trials are an important component of the process of technology appraisal, with a view to informing resource allocation decisions. For end of life treatments, such as cancer…

Applications · Statistics 2020-11-24 Andrea Gabrio

Using a novel modeling approach based on the so-called environmental stress level (ESL), we develop a mathematical model to describe systematically the collective influence of oxygen concentration and stiffness of the extracellular matrix…

Cell Behavior · Quantitative Biology 2023-10-31 Sabrina Schönfeld , Laura Scarabosio , Alican Ozkan , Marissa Nichole Rylander , Christina Kuttler

Many phase II clinical trials have used survival outcomes as the primary endpoints in recent decades. Suppose the radiotherapy is evaluated in a phase II trial using survival outcomes. In that case, the competing risk issue often arises…

Applications · Statistics 2022-03-15 Jina Park , Wenjing Hu , Ick Hoon Jin , Hao Liu , Yong Zang

Many clinical studies evaluate the benefit of a treatment based on both survival and other continuous/ordinal clinical outcomes, such as Quality of Life scores. In these studies, when subjects die before the follow-up assessment, the…

Applications · Statistics 2023-09-06 Qingyan Xiang , Ronald J. Bosch , Judith J. Lok

Progress in immunotherapy revolutionized the treatment landscape for advanced lung cancer, raising survival expectations beyond those that were historically anticipated with this disease. In the present study, we describe the methods for…

Applications · Statistics 2019-11-25 Lizet Sanchez , Patricia Lorenzo-Luaces , Claudia Fonte , Agustin Lage

Mediation analysis seeks to identify and quantify the paths by which an exposure affects an outcome. Intermediate variables which are effected by the exposure and which effect the outcome are known as mediators. There exists extensive work…

Methodology · Statistics 2020-11-13 James P. Long , Ehsan Irajizad , James D. Doecke , Kim-Anh Do , Min Jin Ha

We built a novel Bayesian hierarchical survival model based on the somatic mutation profile of patients across 50 genes and 27 cancer types. The pan-cancer quality allows for the model to "borrow" information across cancer types, motivated…

Quantitative Methods · Quantitative Biology 2019-10-09 Sarah Samorodnitsky , Katherine A. Hoadley , Eric F. Lock

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

Methodology · Statistics 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

We overview Bayesian estimation, hypothesis testing, and model-averaging and illustrate how they benefit parametric survival analysis. We contrast the Bayesian framework to the currently dominant frequentist approach and highlight…

Methodology · Statistics 2022-09-13 František Bartoš , Frederik Aust , Julia M. Haaf

In meta-analytic modeling, the functional relationship between a primary and surrogate endpoint is estimated using summary data from a set of completed clinical trials. Parameters in the meta-analytic model are used to assess the quality of…

Methodology · Statistics 2025-08-07 James P. Long , Abhishikta Roy , Ehsan Irajizad , Kim-Anh Do , Yu Shen

Surrogate endpoint (SE) for overall survival in cancer patients is essential to improving the efficiency of oncology drug development. In practice, we may discover a new patient level association with survival, based on one or more clinical…

Applications · Statistics 2022-11-08 Wei Zou

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…

Objectives To investigate the use of a Bayesian joint modelling approach to predict overall survival (OS) from immature clinical trial data using an intermediate biomarker. To compare the results with a typical parametric approach of…

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