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The ISCHEMIA Trial randomly assigned patients with ischemic heart disease to an invasive treatment strategy centered on revascularization with a control group assigned non-invasive medical therapy. As is common in such ``strategy trials,''…

Econometrics · Economics 2025-01-06 Joshua D. Angrist , Bruno Ferman , Carol Gao , Peter Hull , Otavio L. Tecchio , Robert W. Yeh

Purpose. Patients with advanced cancer may undergo multiple lines of treatment, switching therapies as their disease progresses. Motivated by a study of metastatic prostate cancer, we develop a microsimulation framework to study therapy…

Distinguishing causal relationships from statistical correlations remains a fundamental challenge in clinical research, limiting the translation of observational findings into interventional treatment guidelines. Here we apply causal…

There is growing interest in developing causal inference methods for multi-valued treatments with a focus on pairwise average treatment effects. Here we focus on a clinically important, yet less-studied estimand: causal drug-drug…

Methodology · Statistics 2022-09-20 Di Shu , Peisong Han , Sean Hennessy , Todd A Miano

The primary objective of phase I oncology studies is to establish the safety profile of a new treatment and determine the maximum tolerated dose (MTD). This is motivated by the development of cytotoxic agents based on the underlying…

Applications · Statistics 2023-02-10 Yiding Zhang , Zhixing Xu , Hui Quan , Ji Lin

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

Analyzing outcomes in long-term cancer survivor studies can be complex. The effects of predictors on the failure process may be difficult to assess over longer periods of time, as the commonly used assumption of proportionality of hazards…

Applications · Statistics 2015-09-07 Yolanda Hagar , James J. Dignam , Vanja Dukic

To promote precision medicine, individualized treatment regimes (ITRs) are crucial for optimizing the expected clinical outcome based on patient-specific characteristics. However, existing ITR research has primarily focused on scenarios…

Methodology · Statistics 2024-02-20 Chang Wang , Lu Wang

Analyzing and effectively communicating the efficacy and toxicity of treatment is the basis of risk benefit analysis (RBA). More efficient and objective tools are needed. We apply Chauhan Weighted Trajectory Analysis (CWTA) to perform RBA…

Methodology · Statistics 2024-09-24 Utkarsh Chauhan , Daylen Mackey , John R Mackey

In the study of therapeutic strategies for the treatment of cancer, eco-evolutionary dynamics are of particular interest, since characteristics of the tumour population, interaction with the environment and effects of the treatment,…

Populations and Evolution · Quantitative Biology 2023-07-19 Giulia Chiari , Giada Fiandaca , Marcello Edoardo Delitala

With the advancement of treatment modalities in radiation therapy for cancer patients, outcomes have improved, but at the cost of increased treatment plan complexity and planning time. The accurate prediction of dose distributions would…

Medical Physics · Physics 2018-12-03 Dan Nguyen , Troy Long , Xun Jia , Weiguo Lu , Xuejun Gu , Zohaib Iqbal , Steve Jiang

In cancer treatment, chemotherapy is administered according a constant schedule. The chronotherapy approach, considering chronobiological drug delivery, adapts the chemotherapy profile to the circadian rhythms of the human organism. This…

Quantitative Methods · Quantitative Biology 2016-04-12 Benjamin Schott , Johannes Stegmaier , Alexandre Arbaud , Markus Reischl , Ralf Mikut , Francis Lévi

Delayed treatment effects on time-to-event outcomes have often been observed in randomized controlled studies of cancer immunotherapies. In the case of delayed onset of treatment effect, the conventional test/estimation approach using the…

Methodology · Statistics 2024-03-19 Miki Horiguchi , Lu Tian , Kenneth L. Kehl , Hajime Uno

In carbon-ion radiotherapy, single-beam delivery each day in alternate directions has been commonly practiced for operational efficiency, taking advantage of the Bragg peak and the relative biological effectiveness (RBE) for uniform dose…

Medical Physics · Physics 2018-01-24 Nobuyuki Kanematsu , Taku Inaniwa

Covariate balance is crucial in obtaining unbiased estimates of treatment effects in observational studies. Methods based on inverse probability weights have been widely used to estimate treatment effects with observational data. Machine…

Methodology · Statistics 2021-04-08 Michele Santacatterina

The authors propose robust adaptive strategies based on stochastic minimax optimization for a series of simulated treatments on a one-dimensional patient phantom. The plan applied during the first fractions should be able to handle…

Optimization and Control · Mathematics 2018-10-01 Michelle Böck , Anders Forsgren , Kjell Eriksson , Björn Hårdemark

Dynamic Treatment Regimes (DTRs) provide a systematic framework for optimizing sequential decision-making in chronic disease management, where therapies must adapt to patients' evolving clinical profiles. Inverse probability weighting (IPW)…

Methodology · Statistics 2026-03-26 Chloe Si , David A. Stephens , Erica E. M. Moodie

Estimating individualized treatment rules (ITRs) is crucial for tailoring interventions in precision medicine. Typical ITR estimation methods rely on conditional average treatment effects (CATEs) to guide treatment assignments. However,…

Methodology · Statistics 2025-10-20 Peng Wu , Qing Jiang , Shanshan Luo , Zhi Geng

The purpose of this study was to use various dosimetrical indices to determine the best IMRT modality technique for treating patients with prostate cancer. Ten patients with prostate cancer were included in this study. Intensity modulated…

Randomized discontinuation design (RDD) is an enrichment strategy commonly used to address limitations of traditional placebo-controlled trials, particularly the ethical concern of prolonged placebo exposure. RDD consists of two phases: an…

Methodology · Statistics 2025-06-03 Ayon Mukherjee , Oleksandr Sverdlov , Ngoc-Thuy Ha , Yu Deng