Evidence map›Paper›PMID 35346783›Full record

ArticleJournal of clinical epidemiology2022

Joint modeling of endpoints can be used to answer various research questions in randomized clinical trials.

Ruben P A van Eijk, Kit C B Roes, Leonard H van den Berg, Ying Lu

Abstract read
In one paragraph

Article in Journal of clinical epidemiology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–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

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Trial
  3. Trial
  4. Article
  5. Article
  6. Review
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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

4 authors.

Ruben P A van EijkDepartment of Biomedical Data Science and Center for Innovative Study Design, School of Medicine, Stanford University, Stanford, CA, USA; Department of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Utrecht, The Netherlands. Electronic address: r.p.a.vaneijk-2@umcutrecht.nl.
Kit C B RoesDepartment of Health Evidence, Section Biostatistics, Radboud Medical Center Nijmegen, Nijmegen, The Netherlands.
Leonard H van den BergDepartment of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Utrecht, The Netherlands.
Ying LuDepartment of Biomedical Data Science and Center for Innovative Study Design, School of Medicine, Stanford University, Stanford, CA, USA.

Funding

Translational Oncology Research Program (Project-005)P30CA124435 · NCI · STANFORD UNIVERSITY · PI Vittorio Sebastiano · 2007 to 2026
$71.4M
Stanford Center for Clinical & Translational Education and Research (Spectrum)UL1TR003142 · NCATS · STANFORD UNIVERSITY · PI O'HARA, RUTH M · 2019 to 2023
$45.0M
Statistical Methods for Optimizing Personalized Treatment SelectionR01HL089778 · NHLBI · STANFORD UNIVERSITY · PI LU TIAN · 2008 to 2026
$4.8M
NCATS NIH HHS UL1 TR003142NCI NIH HHS P30 CA124435NHLBI NIH HHS R01 HL089778
6 · The paper itself

Abstract

objectivesCorrelated longitudinal and time-to-event outcomes, such as the rate of cognitive decline and the onset of Alzheimer's disease, are frequent (co-)primary and key secondary endpoints in randomized clinical trials (RCTs). Despite their biological associations, these types of data are often analyzed separately, leading to loss of information and increases in bias. In this paper, we set out how joint modeling of longitudinal and time-to-event endpoints can be used in RCTs to answer various research questions. STUDY DESIGN AND

settingThe key concepts of joint models are introduced and illustrated for a completed trial in amyotrophic lateral sclerosis.

resultsThe output of a joint model can be used to answer different clinically relevant research questions, where the interpretation of effect estimates and those obtained from conventional methods are similar. Albeit joint models have the potential to overcome the limitations of commonly used alternatives, they require additional assumptions regarding the distributions, as well as the associations between two endpoints.

conclusionImproving the uptake of joint models in RCTs may start by outlining the exact research question one seeks to answer, thereby determining how best to prespecify the model and defining the parameter that should be of primary interest.

Indexed as

Amyotrophic Lateral SclerosisBiasHumansRandomized Controlled Trials as TopicAmyotrophic lateral sclerosisClinical trialsInformative censoringJoint modelingLongitudinalTime-to-event

Identifiers

PMID35346783
PMCPMC10266556

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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.