Evidence map›Paper›PMID 42144739›Full record

ArticleStatistics in medicine2026

A Bayesian Location-Scale Joint Model for Time-To-Event and Multivariate Longitudinal Data With Association Based on Within-Individual Variability.

Marco Palma, Omar El Makkaoui, Ruth H Keogh, Siobhán B Carr, Rhonda Szczesniak, David Taylor-Robinson, Angela M Wood, Graciela Muniz-Terrera, Jessica K Barrett

Abstract read
In one paragraph

Article in Statistics in medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Marco PalmaMRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Omar El MakkaouiMRC Biostatistics Unit, University of Cambridge, Cambridge, UK.
Ruth H KeoghDepartment of Medical Statistics, London School of Hygiene & Tropical Medicine, London, UK.ORCID https://orcid.org/0000-0001-6504-3253
Siobhán B CarrRoyal Brompton Hospital, Guy's and St Thomas' NHS Foundation Trust, London, UK.
Rhonda SzczesniakDivisions of Biostatistics and Epidemiology and Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.ORCID https://orcid.org/0000-0003-0705-715X
David Taylor-RobinsonDepartment of Public Health, Policy and Systems, University of Liverpool, Liverpool, UK.
Angela M WoodBritish Heart Foundation Cardiovascular Epidemiology Unit, Department of Public Health and Primary Care, University of Cambridge, Cambridge, UK.
Graciela Muniz-TerreraOhio University Heritage College of Osteopathic Medicine, Athens, Ohio, USA.
Jessica K BarrettMRC Biostatistics Unit, University of Cambridge, Cambridge, UK.

Funding

R01- Mapping environmental contributions to rapid lung disease progression in cystic fibrosisR01HL141286 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI SZCZESNIAK, RHONDA · 2019 to 2023
$2.3M
British Heart Foundation CH/12/2/29428British Heart Foundation HDRUK2023.0239British Heart Foundation RE/24/130011British Heart Foundation RG/F/23/110103Cystic Fibrosis Foundation Naren19R0Cystic Fibrosis Foundation SZCZES22AB0Health Data Research UK Big Data for Complex Disease-HDR-23012Health Data Research UK HDRUK2023.0028Medical Research Council MC_UU_00002/5Medical Research Council MC_UU_00040/02Medical Research Council MR/V020595/1National Institute for Health and Care Research NIHR 203312National Institute for Health and Care Research NIHR 302438National Institute for Health and Care Research NIHR 303137NHLBI NIH HHS R01 HL141286NIH HHS R01 HL141286Osteopathic Heritage FoundationUK Research and Innovation MR/X015017/1Ulverscroft Foundation
6 · The paper itself

Abstract

Within-individual variability of health indicators measured over time is becoming commonly used to inform about disease progression. Simple summary statistics (e.g., the standard deviation for each individual) are often used but they are not suited to account for time changes. In addition, when these summary statistics are used as covariates in a regression model for time-to-event outcomes, the estimates of the hazard ratios are subject to regression dilution. To overcome these issues, a joint model is built where the association between the time-to-event outcome and multivariate longitudinal markers is specified in terms of the within-individual variability of the latter. A mixed-effect location-scale model is used to analyze the longitudinal biomarkers, their within-individual variability and their correlation. The time to event is modeled using a proportional hazard regression model, with a flexible specification of the baseline hazard, and the information from the longitudinal biomarkers is shared as a function of the random effects. The model can be used to quantify within-individual variability for the longitudinal markers and their association with the time-to-event outcome. We show through a simulation study the performance of the model in comparison with standard joint models with constant variance. The model is applied on a dataset of adult women from the UK cystic fibrosis registry, to evaluate the association between lung function, malnutrition and mortality.

Indexed as

Models, StatisticalAdultBayes TheoremBiomarkersComputer SimulationCystic FibrosisDisease ProgressionFemaleHumansLongitudinal StudiesMultivariate AnalysisProportional Hazards ModelsBiomarkerscystic fibrosisjoint modelmixed‐effects location‐scale model (MELSM)within‐individual variability

Identifiers

PMID42144739
PMCPMC13181161

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.