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Multiple outcomes, both continuous and discrete, are routinely gathered on subjects in longitudinal studies and during routine clinical follow-up in general. To motivate our work, we consider a longitudinal study on patients with primary…

Applications · Statistics 2013-04-17 Arnošt Komárek , Lenka Komárková

A randomized trial allows estimation of the causal effect of an intervention compared to a control in the overall population and in subpopulations defined by baseline characteristics. Often, however, clinical questions also arise regarding…

Subgroup analysis evaluates treatment effects across multiple sub-populations. When subgroups are defined by latent memberships inferred from imperfect measurements, the analysis typically involves two inter-connected models, a latent class…

Methodology · Statistics 2026-01-05 Yuanhui Luo , Xinzhou Guo , Yuqi Gu

We consider the problem of identifying promising subpopulations in terms of treatment effectiveness or treatment effect heterogeneity, from a Bayesian decision theoretic perspective. We first show that a straight-forward application of…

Methodology · Statistics 2025-09-09 Entejar Alam , Poorbita Kundu , Antonio R. Linero

Background: Clinical trials are designed to prove the efficacy of an intervention by means of model-based approaches involving parametric hypothesis testing. Issues arise when no effect is observed in the study population. Indeed, an effect…

While studying response trajectory, often the population of interest may be diverse enough to exist distinct subgroups within it and the longitudinal change in response may not be uniform in these subgroups. That is, the timeslope and/or…

Methodology · Statistics 2020-06-30 Madan Gopal Kundu , Jaroslaw Harezlak

Micro-randomized trials (MRTs) are widely used to assess the marginal and moderated effect of mobile health (mHealth) treatments delivered via mobile devices. In many applications, the mHealth treatments are categorical with multiple levels…

Methodology · Statistics 2025-04-23 Jeremy Lin , Tianchen Qian

In the analysis of cluster data, the regression coefficients are frequently assumed to be the same across all clusters. This hampers the ability to study the varying impacts of factors on each cluster. In this paper, a semiparametric model…

Statistics Theory · Mathematics 2009-08-25 Wenyang Zhang , Jianqing Fan , Yan Sun

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

MicroRNAs play important roles in many biological processes. Their aberrant expression can have oncogenic or tumor suppressor function directly participating to carcinogenesis, malignant transformation, invasiveness and metastasis. Indeed,…

This work presents an unsupervised deep discriminant analysis for clustering. The method is based on deep neural networks and aims to minimize the intra-cluster discrepancy and maximize the inter-cluster discrepancy in an unsupervised…

Machine Learning · Computer Science 2022-06-13 Jinyu Cai , Wenzhong Guo , Jicong Fan

Identifying relationships between molecular variations and their clinical presentations has been challenged by the heterogeneous causes of a disease. It is imperative to unveil the relationship between the high dimensional molecular…

Methodology · Statistics 2021-09-02 Wennan Chang , Changlin Wan , Yong Zang , Chi Zhang , Sha Cao

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Adaptive interventions, aka dynamic treatment regimens, are sequences of pre-specified decision rules that guide the provision of treatment for an individual given information about their baseline and evolving needs, including in response…

Methodology · Statistics 2024-05-02 Wenchu Pan , Daniel Almirall , Amy M. Kilbourne , Andrew Quanbeck , Lu Wang

Discovering subgroups with the maximum average treatment effect is crucial for targeted decision making in domains such as precision medicine, public policy, and education. While most prior work is formulated in the potential outcome…

Machine Learning · Computer Science 2025-11-26 Lincen Yang , Zhong Li , Matthijs van Leeuwen , Saber Salehkaleybar

Understanding treatment heterogeneity is essential to the development of precision medicine, which seeks to tailor medical treatments to subgroups of patients with similar characteristics. One of the challenges to achieve this goal is that…

Methodology · Statistics 2019-08-21 Shujie Ma , Jian Huang , Zhiwei Zhang , Mingming Liu

Interference occurs when the potential outcomes of a unit depend on the treatment of others. Interference can be highly heterogeneous, where treating certain individuals might have a larger effect on the population's overall outcome. A…

Methodology · Statistics 2025-04-11 Samantha G Dean , Georgia Papadogeorgou , Laura Forastiere

The correct identification of clusters is crucial for an accurate monitoring of the spread of a disease and also in many other natural, social and physical phenomena which exhibit an epidemic structure. Nevertheless, even when an accurate…

Physics and Society · Physics 2021-04-12 Eugenio Lippiello , Polytzois Bountzis

Cluster analysis is a popular unsupervised learning tool used in many disciplines to identify heterogeneous sub-populations within a sample. However, validating cluster analysis results and determining the number of clusters in a data set…

Machine Learning · Statistics 2024-04-26 Ali Turfah , Xiaoquan Wen

In microbiome studies, it is often of great interest to identify clusters or partitions of microbiome profiles within a study population and to characterize the distinctive attributes of each resulting microbial community. While raw counts…

Methodology · Statistics 2025-08-18 Zhongmao Liu , Xiaohui Yin , Yanjiao Zhou , Gen Li , Kun Chen
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