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The estimation of treatment effects is a pervasive problem in medicine. Existing methods for estimating treatment effects from longitudinal observational data assume that there are no hidden confounders, an assumption that is not testable…

机器学习 · 计算机科学 2020-09-21 Ioana Bica , Ahmed M. Alaa , Mihaela van der Schaar

We consider the design of a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome. We detail Bayesian inference for the analysis of the trial using a linear mixed-effects model. The treatment is compared to…

统计方法学 · 统计学 2022-08-29 Kevin J Wilson

Estimating heterogeneous treatment effects across individuals has attracted growing attention as a statistical tool for performing critical decision-making. We propose a Bayesian inference framework that quantifies the uncertainty in…

统计方法学 · 统计学 2023-12-19 Shunsuke Horii , Yoichi Chikahara

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

统计方法学 · 统计学 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

The primary analysis of randomized screening trials for cancer typically adheres to the intention-to-screen principle, measuring cancer-specific mortality reductions between screening and control arms. These mortality reductions result from…

统计方法学 · 统计学 2021-07-30 Sudipta Saha , Zhihui Liu , Olli Saarela

We address the common problem of calculating intervals in the presence of systematic uncertainties. We aim to investigate several approaches, but here describe just a Bayesian technique for setting upper limits. The particular example we…

数据分析、统计与概率 · 物理学 2007-05-23 Joel Heinrich , Craig Blocker , John Conway , Luc Demortier , Louis Lyons , Giovanni Punzi , Pekka K. Sinervo

Determining the extent to which a patient is benefiting from cancer therapy is challenging. Criteria for quantifying the extent of "tumor response" observed within a few cycles of treatment have been established for various types of solid…

统计方法学 · 统计学 2020-09-18 Jie Zhou , Xun Jiang , H. Amy Xia , Peng Wei , Brian P. Hobbs

In high energy physics, a widely used method to treat systematic uncertainties in confidence interval calculations is based on combining a frequentist construction of confidence belts with a Bayesian treatment of systematic uncertainties.…

数据分析、统计与概率 · 物理学 2009-11-10 Fredrik Tegenfeldt , Jan Conrad

Large variability between cell lines brings a difficult optimization problem of drug selection for cancer therapy. Standard approaches use prediction of value for this purpose, corresponding e.g. to expected value of their distribution.…

定量方法 · 定量生物学 2022-09-15 Jarek Duda

In this note we present studies of coverage and power for confidence intervals for a Poisson process with known background calculated using the Likelihood ratio (aka Feldman & Cousins) ordering with Bayesian treatment of uncertainties in…

数据分析、统计与概率 · 物理学 2017-08-23 Jan Conrad , Fredrik Tegenfeldt

Cancer development is driven by mutations and selective forces, including the action of the immune system and interspecific competition. When administered to patients, anti-cancer therapies affect the development and dynamics of tumours,…

动力系统 · 数学 2021-09-30 Elena Piretto , Marcello Delitala , Peter S. Kim , Federico Frascoli

This paper proposes a general framework for probabilistic certification of cancer therapies. The certification is defined in terms of two key issues which are the tumor contraction and the lower admissible bound on the circulating…

系统与控制 · 计算机科学 2015-02-24 Mazen Alamir

Cancer is one of the most common diseases worldwide, posing a serious threat to human health and leading to the deaths of a large number of people. It was observed during the drug administration in chemotherapy that immune cells, cancer…

定量方法 · 定量生物学 2020-08-20 SeyedMehdi Abtahi , Mojtaba Sharifi

Survival time is the primary endpoint of many randomized controlled trials, and a treatment effect is typically quantified by the hazard ratio under the assumption of proportional hazards. Awareness is increasing that in many settings this…

统计方法学 · 统计学 2023-10-04 Robin Ristl , Heiko Götte , Armin Schüler , Martin Posch , Franz König

The development of a new diagnostic test ideally follows a sequence of stages which, amongst other aims, evaluate technical performance. This includes an analytical validity study, a diagnostic accuracy study and an interventional clinical…

We formulate and analyze a mathematical model describing immune response to avascular tumor under the influence of immunotherapy and chemotherapy and their combinations as well as vaccine treatments. The effect of vaccine therapy is…

细胞行为 · 定量生物学 2008-10-15 O. G. Isaeva , V. A. Osipov

Delays in biological systems may be used to model events for which the underlying dynamics cannot be precisely observed. Mathematical modeling of biological systems with delays is usually based on Delay Differential Equations (DDEs), a kind…

定量方法 · 定量生物学 2009-10-08 Roberto Barbuti , Giulio Caravagna , Paolo Milazzo , Andrea Maggiolo-Schettini

The paper considers two different designs; a two-arm superiority cluster randomised controlled trial (RCT) with a continuous outcome, and a twoarm superiority cluster RCT with a binary outcome. From a Bayesian perspective, for the analysis…

统计方法学 · 统计学 2025-11-11 Abdullah Aloufi , Kevin Wilson , Nina Wilson , Lisa Shaw , Christopher Price

The reproducibility crisis has led to an increasing number of replication studies being conducted. Sample sizes for replication studies are often calculated using conditional power based on the effect estimate from the original study.…

统计方法学 · 统计学 2022-10-19 Charlotte Micheloud , Leonhard Held

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

统计方法学 · 统计学 2021-08-31 Elja Arjas , Dario Gasbarra