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A model for cross-over designs with repeated measures within each period was developed. It is obtained using an extension of generalized estimating equations that includes a parametric component to model treatment effects and a…

Methodology · Statistics 2023-03-21 N. A. Cruz , O. O. Melo , C. A. Martinez

Breast cancer remains a significant global health challenge, with prognosis and treatment decisions largely dependent on clinical characteristics. Accurate prediction of patient outcomes is crucial for personalized treatment strategies.…

This work bridges the gap between staggered adoption designs and survival analysis to estimate causal effects in settings with time-varying treatments, addressing a fundamental challenge in medical research exemplified by the Stanford Heart…

Methodology · Statistics 2025-03-04 Xiang Meng , Iavor Bojinov

Combination drug therapies hold significant promise for enhancing treatment efficacy, particularly in fields such as oncology, immunotherapy, and infectious diseases. However, designing clinical trials for these regimens poses unique…

Methodology · Statistics 2025-06-04 Nan Miles Xi , Man Mandy Jin , Lin Wang , Xin Huang

Given the prominence of targeted therapy and immunotherapy in cancer treatment, it becomes imperative to consider heterogeneity in patients' responses to treatments, which contributes greatly to the widely used proportional hazard…

Methodology · Statistics 2024-09-01 Powei Chen , Siying Hu , Haojin Zhou

In Bayesian semi-parametric analyses of time-to-event data, non-parametric process priors are adopted for the baseline hazard function or the cumulative baseline hazard function for a given finite partition of the time axis. However, it…

Methodology · Statistics 2020-08-06 Yi Li , Sumi Seo , Kyu Ha Lee

The inverse probability weighting approach is popular for evaluating treatment effects in observational studies, but extreme propensity scores could bias the estimator and induce excessive variance. Recently, the overlap weighting approach…

Methodology · Statistics 2022-06-22 Chao Cheng , Fan Li , Laine Thomas , Fan Li

We develop a Bayesian semi-parametric model for the estimating the impact of dynamic treatment rules on survival among patients diagnosed with pediatric acute myeloid leukemia (AML). The data consist of a subset of patients enrolled in the…

Methodology · Statistics 2023-12-12 Arman Oganisian , Kelly D. Getz , Todd A. Alonzo , Richard Aplenc , Jason A. Roy

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

When choosing estimands and estimators in randomized clinical trials, caution is warranted as intercurrent events, such as - due to patients who switch treatment after disease progression, are often extreme. Statistical analyses may then…

Applications · Statistics 2023-03-13 Hege Michiels , An Vandebosch , Stijn Vansteelandt

Pragmatic trials increasingly define outcomes using real-world data such as electronic health records, where assessments are collected during routine care rather than at fixed timepoints. Consequently, these uncontrolled assessments may be…

Proximal causal inference provides a framework for estimating the average treatment effect (ATE) in the presence of unmeasured confounding by leveraging outcome and treatment proxies. Identification in this framework relies on the existence…

Methodology · Statistics 2025-12-29 Chunrong Ai , Jiawei Shan

Causal identification of treatment effects for infectious disease outcomes in interconnected populations is challenging because infection outcomes may be transmissible to others, and treatment given to one individual may affect others'…

Methodology · Statistics 2021-05-11 Xiaoxuan Cai , Eben Kenah , Forrest W. Crawford

No unmeasured confounding is often assumed in estimating treatment effects in observational data when using approaches such as propensity scores and inverse probability weighting. However, in many such studies due to the limitation of the…

Applications · Statistics 2019-08-06 Rong Huang , Ronghui Xu , Parambir S. Dulai

In recent years, research interest in personalised treatments has been growing. However, treatment effect heterogeneity and possibly time-varying treatment effects are still often overlooked in clinical studies. Statistical tools are needed…

Methodology · Statistics 2023-10-27 Caterina Gregorio , Giovanni Baj , Giulia Barbati , Francesca Ieva

The treatment effects of the same therapy observed from multiple clinical trials can often be very different. Yet the patient characteristics accounting for these differences may not be identifiable in real world practice. There needs to be…

Statistics Theory · Mathematics 2015-03-20 Tinghui Yu , Yabing Mai , Sherry Liu , Xiaofei Hu

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

We develop new semiparametric methods for estimating treatment effects. We focus on settings where the outcome distributions may be thick tailed, where treatment effects may be small, where sample sizes are large and where assignment is…

Methodology · Statistics 2023-08-24 Susan Athey , Peter J. Bickel , Aiyou Chen , Guido W. Imbens , Michael Pollmann

Real-world clinical decision making is a complex process that involves balancing the risks and benefits of treatments. Quality-adjusted lifetime is a composite outcome that combines patient quantity and quality of life, making it an…

Statistics Theory · Mathematics 2024-12-09 Hao Sun , Ashkan Ertefaie , Luke Duttweiler , Brent A. Johnson

Given the long follow-up periods that are often required for treatment or intervention studies, the potential to use surrogate markers to decrease the required follow-up time is a very attractive goal. However, previous studies have shown…

Methodology · Statistics 2016-08-12 Layla Parast , Tianxi Cai , Lu Tian
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