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Missing data is a pervasive problem in epidemiology, with multiple imputation (MI) a commonly used analysis method. MI is valid when data are missing at random (MAR). However, definitions of MAR with multiple incomplete variables are not…

Methodology · Statistics 2025-04-14 Paul Madley-Dowd , Rachael A. Hughes , Maya B. Mathur , Jon Heron , Kate Tilling

In several clinical areas, traditional clinical trials often use a responder outcome, a composite endpoint that involves dichotomising a continuous measure. An augmented binary method that improves power whilst retaining the original…

Methodology · Statistics 2024-08-19 Svetlana Cherlin , James M S Wason

We give two prediction intervals (PI) for Generalized Linear Models that take model selection uncertainty into account. The first is a straightforward extension of asymptotic normality results and the second includes an extra optimization…

Methodology · Statistics 2023-05-26 Dean Dustin , Bertrand Clarke

Multiple imputation (MI) inference handles missing data by imputing the missing values $m$ times, and then combining the results from the $m$ complete-data analyses. However, the existing method for combining likelihood ratio tests (LRTs)…

Statistics Theory · Mathematics 2022-01-03 Kin Wai Chan , Xiao-Li Meng

As meta-analysis of multiple diagnostic tests impacts clinical decision making and patient health, there is growing interest in statistical models that synthesize evidence from studies comparing multiple diagnostic tests. To compare the…

Methodology · Statistics 2026-05-19 Aristidis K. Nikoloulopoulos

Widely used methods for analyzing missing data can be biased in small samples. To understand these biases, we evaluate in detail the situation where a small univariate normal sample, with values missing at random, is analyzed using either…

Statistics Theory · Mathematics 2017-03-27 Paul T. von Hippel

We propose a novel semiparametric model for the joint distribution of a continuous longitudinal outcome and the baseline covariates using an enriched Dirichlet process (EDP) prior. This joint model decomposes into a linear mixed model for…

Methodology · Statistics 2018-06-08 Bret Zeldow , James Flory , Alisa Stephens-Shields , Marsha Raebel , Jason Roy

The standard regression tree method applied to observations within clusters poses both methodological and implementation challenges. Effectively leveraging these data requires methods that account for both individual-level and sample-level…

Methodology · Statistics 2025-03-05 Jeremiah Allis , Xin Jin , Riddhi Ghosh

Clinical trials often involve the assessment of multiple endpoints to comprehensively evaluate the efficacy and safety of interventions. In the work, we consider a global nonparametric testing procedure based on multivariate rank for the…

Methodology · Statistics 2023-06-29 Kexuan Li , Lingli Yang , Shaofei Zhao , Susie Sinks , Luan Lin , Peng Sun

The recently published ICH E9 addendum on estimands in clinical trials provides a framework for precisely defining the treatment effect that is to be estimated, but says little about estimation methods. Here we report analyses of a clinical…

Applications · Statistics 2023-09-25 Camila Olarte Parra , Rhian M. Daniel , David Wright , Jonathan W. Bartlett

Sparse regularized regression methods are now widely used in genome-wide association studies (GWAS) to address the multiple testing burden that limits discovery of potentially important predictors. Linear mixed models (LMMs) have become an…

Methodology · Statistics 2022-06-27 Julien St-Pierre , Karim Oualkacha , Sahir Rai Bhatnagar

A recent paper proposed an extended trivariate generalized linear mixed model (TGLMM) for synthesis of diagnostic test accuracy studies in the presence of non-evaluable index test results. Inspired by the aforementioned model we propose an…

Applications · Statistics 2020-01-01 Aristidis K. Nikoloulopoulos

Sequential multiple assignment randomized trials (SMARTs) are used to construct data-driven optimal intervention strategies for subjects based on their intervention and covariate histories in different branches of health and behavioral…

Methodology · Statistics 2022-04-28 Palash Ghosh , Xiaoxi Yan , Bibhas Chakraborty

Multivariate data occurs in a wide range of fields, with ever more flexible model specifications being proposed, often within a multivariate generalised linear mixed effects (MGLME) framework. In this article, we describe an extended…

Methodology · Statistics 2017-10-09 Michael J. Crowther

In observational studies, weighting methods that directly optimize the balance between treatment and covariates have received much attention lately; however these have mainly focused on binary treatments. Inspired by domain adaptation, we…

Statistics Theory · Mathematics 2020-02-27 Guillaume Martinet

Background: Existing guidelines for handling missing data are generally not consistent with the goals of prediction modelling, where missing data can occur at any stage of the model pipeline. Multiple imputation (MI), often heralded as the…

Methodology · Statistics 2022-06-27 Rose Sisk , Matthew Sperrin , Niels Peek , Maarten van Smeden , Glen P. Martin

Introduction In analysis of time-to-event outcomes, a mixture cure (MC) model is preferred over a standard survival model when the sample includes individuals who will never experience the event of interest. Motivated by a cohort study of…

Applications · Statistics 2026-04-02 Changchang Xu , Laurent Briollais , Irene L Andrulis , Shelley B Bull

With the rapid development of new anti-cancer agents which are cytostatic, new endpoints are needed to better measure treatment efficacy in phase II trials. For this purpose, Von Hoff (1998) proposed the growth modulation index (GMI), i.e.…

Methodology · Statistics 2021-11-01 Li Chen , Mark Burkard , Jianrong Wu , Jill M. Kolesar , Chi Wang

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

Methodology · Statistics 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

The primary outcome of Randomized clinical Trials (RCTs) are typically dichotomous, continuous, multivariate continuous, or time-to-event. However, what if this outcome is unstructured, e.g., a list of variables of mixed types, longitudinal…