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An assurance calculation is a Bayesian alternative to a power calculation. One may be performed to aid the planning of a clinical trial, specifically setting the sample size or to support decisions about whether or not to perform a study.…

Applications · Statistics 2024-03-29 James Salsbury , Jeremy Oakley , Steven Julious , Lisa Hampson

Tumor response, a binary variable, has historically been the main measure of antitumor activity for many cancer phase II single-arm trials. Simon two-stage designs are often used. Sargent et al. proposed a three-outcome trial design in this…

Methodology · Statistics 2025-03-20 Minghua Shan

In cluster-randomized crossover (CRXO) trials, groups of individuals are randomly assigned to two or more sequences of alternating treatments. Since clusters serve as their own control, the CRXO design is typically more statistically…

Methodology · Statistics 2026-01-30 Dane Isenberg , Michael O. Harhay , Andrew B. Forbes , Paul J. Young , Fan Li , Nandita Mitra

The long-term efficacy of targeted therapeutics for cancer treatment can be significantly limited by the type of therapy and development of drug resistance, inter alia. Experimental studies indicate that the factors enhancing acquisition of…

Tissues and Organs · Quantitative Biology 2021-02-16 Joseph Malinzi , Kevin Bosire Basita , Sara Padidar , Henry A. Adeola

Metastasis, the spread of cancer cells from a primary tumor to secondary location(s) in the human organism, is the ultimate cause of death for the majority of cancer patients. That is why, it is crucial to understand metastases evolution in…

Populations and Evolution · Quantitative Biology 2020-06-24 Maroussia Slavtchova-Bojkova , Kaloyan Vitanov

In this article, we present a Bayesian hierarchical model for predicting a latent health state from longitudinal clinical measurements. Model development is motivated by the need to integrate multiple sources of data to improve clinical…

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…

Methodology · Statistics 2024-10-15 Pavithra Hariharan , P. G. Sankaran

When planning an oncology clinical trial, the usual approach is to assume proportional hazards and even an exponential distribution for time-to-event endpoints. Often, besides the gold-standard endpoint overall survival (OS),…

Applications · Statistics 2023-12-19 Alexandra Erdmann , Jan Beyersmann , Kaspar Rufibach

In oncology clinical trials, characterizing the long-term overall survival (OS) benefit for an experimental drug or treatment regimen (experimental group) is often unobservable if some patients in the control group switch to drugs in the…

Applications · Statistics 2019-08-05 Yuqing Xu , Meijing Wu , Weili He , Qiming Liao , Yabing Mai

Treatment-related lymphopenia is a frequent and clinically significant consequence of cancer therapy that can compromise immune-mediated tumor control and worsen patient outcomes. Despite its importance, no mechanistic framework exists to…

Medical Physics · Physics 2026-05-04 Vladislav Sandul , Marco Durante , Thomas Friedrich

Radiotherapy is a commonly used treatment for cancer and is usually given in varying doses. At low radiation doses relatively few cells die as a direct response to radiation but secondary radiation effects such as DNA mutation or bystander…

Quantitative Methods · Quantitative Biology 2016-06-16 Gibin G Powathil , Alastair J Munro , Mark AJ Chaplain , Maciej Swat

Motivation: Epigenetic heterogeneity within a tumour can play an important role in tumour evolution and the emergence of resistance to treatment. It is increasingly recognised that the study of DNA methylation (DNAm) patterns along the…

Quantitative Methods · Quantitative Biology 2017-02-21 James E. Barrett , Andrew Feber , Javier Herrero , Miljana Tanic , Gareth Wilson , Charles Swanton , Stephan Beck

Graphical models are commonly used to discover associations within gene or protein networks for complex diseases such as cancer. Most existing methods estimate a single graph for a population, while in many cases, researchers are interested…

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…

Methodology · Statistics 2022-11-23 Yuhao Deng , Yingjun Chang , Xiao-Hua Zhou

For predicting cancer survival outcomes, standard approaches in clinical research are often based on two main modalities: pathology images for observing cell morphology features, and genomic (e.g., bulk RNA-seq) for quantifying gene…

Computer Vision and Pattern Recognition · Computer Science 2024-03-19 Hongxiao Wang , Yang Yang , Zhuo Zhao , Pengfei Gu , Nishchal Sapkota , Danny Z. Chen

In recent years new cancer treatments improved survival in multiple histologies. Some of these therapeutics, and in particular treatment combinations, are often associated with severe treatment-related adverse events (AEs). It is therefore…

Applications · Statistics 2022-03-10 Steffen Ventz , Lorenzo Trippa

Clustering is commonly performed as an initial analysis step for uncovering structure in 'omics datasets, e.g. to discover molecular subtypes of disease. The high-throughput, high-dimensional nature of these datasets means that they provide…

Methodology · Statistics 2023-03-02 Paul D. W. Kirk , Filippo Pagani , Sylvia Richardson

Tumor development is characterized by a compromised balance between cell life and death decision mechanisms, which are tighly regulated in normal cells. Understanding this process provides insights for developing new treatments for fighting…

Molecular Networks · Quantitative Biology 2013-01-14 Andrei Zinovyev , Simon Fourquet , Laurent Tournier , Laurence Calzone , Emmanuel Barillot

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as…

Methodology · Statistics 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast

We propose a multi-metric flexible Bayesian framework to support efficient interim decision-making in multi-arm multi-stage phase II clinical trials. Multi-arm multi-stage phase II studies increase the efficiency of drug development, but…

Applications · Statistics 2023-12-15 Suzanne M. Dufault , Angela M. Crook , Katie Rolfe , Patrick P. J. Phillips