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Cytometry experiments yield high-dimensional point cloud data that is difficult to interpret manually. Boolean gating techniques coupled with comparisons of relative abundances of cellular subsets is the current standard for cytometry data…

Quantitative Methods · Quantitative Biology 2022-05-04 Soham Mukherjee , Darren Wethington , Tamal K. Dey , Jayajit Das

Clinical trials assessing neurological treatment are challenging due to the diversity of brain function, and the difficulty in quantifying it. Traditional treatment studies in epilepsy use seizure frequency as the primary outcome measure,…

Applications · Statistics 2025-12-02 Ian Miller , Ann Hyslop , Colin Decker

Longitudinal and time-to-event data are often analyzed in biomarker research to study the association between the longitudinal biomarker measurements and the event-time outcome, in which the longitudinal information contributes to the…

Methodology · Statistics 2025-09-09 Minzee Kim , Joel A. Dubin

In randomized controlled trials, ordinal outcomes typically improve statistical efficiency over binary outcomes. The treatment effect on an ordinal outcome is usually described by the odds ratio from a proportional odds model, but this…

Methodology · Statistics 2026-01-01 Lindsey E. Turner , Carolyn T. Bramante , Thomas A. Murray

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

The analysis of survey data is a frequently arising issue in clinical trials, particularly when capturing quantities which are difficult to measure. Typical examples are questionnaires about patient's well-being, pain, or consent to an…

Methodology · Statistics 2024-01-31 Johannes Wieditz , Clemens Miller , Jan Scholand , Marcus Nemeth

Before new clinical measurement methods are implemented in clinical practice, it must be confirmed whether their results are equivalent to those of existing methods. The agreement of the trend between these methods is evaluated using the…

Methodology · Statistics 2021-03-01 Mayu Hiraishi , Kensuke Tanioka , Toshio Shimokawa

To address an important risk classification issue that arises in clinical practice, we propose a new mixture model via latent cure rate markers for survival data with a cure fraction. In the proposed model, the latent cure rate markers are…

Applications · Statistics 2009-10-12 Sungduk Kim , Yingmei Xi , Ming-Hui Chen

Instrumental variables are commonly used to estimate effects of a treatment afflicted by unmeasured confounding, and in practice instruments are often continuous (e.g., measures of distance, or treatment preference). However, available…

Methodology · Statistics 2018-07-05 Edward H. Kennedy , Scott A. Lorch , Dylan S. Small

Causal inference for observational longitudinal studies often requires the accurate estimation of treatment effects on time-to-event outcomes in the presence of time-dependent patient history and time-dependent covariates. To tackle this…

Machine Learning · Statistics 2022-06-17 Jie Zhu , Blanca Gallego

In recent years, medical information technology has made it possible for electronic health record (EHR) to store fairly complete clinical data. This has brought health care into the era of "big data". However, medical data are often sparse…

Machine Learning · Computer Science 2023-06-06 Weizhi Nie , Yuhe Yu , Chen Zhang , Dan Song , Lina Zhao , Yunpeng Bai

Clustered observations are ubiquitous in controlled and observational studies and arise naturally in multi-centre trials or longitudinal surveys. We present a novel model for the analysis of clustered observations where the marginal…

Methodology · Statistics 2022-11-03 Luisa Barbanti , Torsten Hothorn

The g-formula can be used to estimate the treatment effect while accounting for confounding bias in observational studies. With regard to time-to-event endpoints, possibly subject to competing risks, the construction of valid pointwise…

Methodology · Statistics 2024-04-03 Jasmin Rühl , Sarah Friedrich

Survival analysis is essential for studying time-to-event outcomes and providing a dynamic understanding of the probability of an event occurring over time. Various survival analysis techniques, from traditional statistical models to…

Machine Learning · Computer Science 2024-03-13 Ziwen Wang , Jin Wee Lee , Tanujit Chakraborty , Yilin Ning , Mingxuan Liu , Feng Xie , Marcus Eng Hock Ong , Nan Liu

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

Medication adherence is essential to ensure treatment effectiveness, but too often in routine care non-adherence compromises the desired outcome. We explore longitudinal causal modelling using observational data to estimate the time-varying…

Methodology · Statistics 2026-03-10 Xiaoran Liang , Deniz Türkmen , Jane A H Masoli , Luke C Pilling , Jack Bowden

Over the last decades, many prognostic models based on artificial intelligence techniques have been used to provide detailed predictions in healthcare. Unfortunately, the real-world observational data used to train and validate these models…

Machine Learning · Computer Science 2023-11-21 Alice Bernasconi , Alessio Zanga , Peter J. F. Lucas , Marco Scutari , Fabio Stella

We demonstrate a comprehensive semiparametric approach to causal mediation analysis, addressing the complexities inherent in settings with longitudinal and continuous treatments, confounders, and mediators. Our methodology utilizes a…

This paper is motivated by the analysis of a survey study of college student wellbeing before and after the outbreak of the COVID-19 pandemic. A statistical challenge in well-being survey studies lies in that outcome variables are often…

Methodology · Statistics 2023-06-09 Shaobo Li , Zhaohu Fan , Ivy Liu , Philip S. Morrison , Dungang Liu

The current state of evaluation in survival analysis is plagued by the persistent use of evaluation metrics in ways that are misaligned with the stated modeling objective. In addition, many such evaluations are based on censoring…