Evidence map›Paper›PMID 36305571›Full record

ArticleStatistics in medicine2022

Sample size determination for the association between longitudinal and time-to-event outcomes using the joint modeling time-dependent slopes parameterization.

Jessica LeClair, Joseph Massaro, Oleksandr Sverdlov, Leslie Gordon, Yorghos Tripodis

Abstract read
In one paragraph

Article in Statistics in medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
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

5 authors.

Jessica LeClairDepartment of Biostatistics, Boston University School of Public Health, Boston, Massachusetts, USA.ORCID 0000-0001-7874-4913
Joseph MassaroDepartment of Biostatistics, Boston University School of Public Health, Boston, Massachusetts, USA.
Oleksandr SverdlovEarly Development Analytics, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.ORCID 0000-0002-1626-2588
Leslie GordonDivision of Genetics, Department of Pediatrics, Hasbro Children's Hospital and Warren Alpert Medical School of Brown University, Providence, Rhode Island, USA.
Yorghos TripodisDepartment of Biostatistics, Boston University School of Public Health, Boston, Massachusetts, USA.ORCID 0000-0003-2190-7608

Funding

Neuropathology CoreP30AG013846 · NIA · BOSTON UNIVERSITY MEDICAL CAMPUS · PI KOWALL, NEIL W. · 1996 to 2020
$29.1M
RL5: Research Education CoreP30AG072978 · NIA · BOSTON UNIVERSITY MEDICAL CAMPUS · PI Ann C. McKee · 2021 to 2026
$25.6M
Interdisciplinary Training for BiostatisticiansT32GM074905 · NIGMS · BOSTON UNIVERSITY MEDICAL CAMPUS · PI WHITE, LAURA FORSBERG · 2005 to 2019
$3.0M
BUSPH Interdisciplinary Training Program for BiostatisticiansT32GM140972 · NIGMS · BOSTON UNIVERSITY MEDICAL CAMPUS · PI Kathryn L Lunetta, Yorghos Tripodis · 2021 to 2026
$1.9M
NIA NIH HHS P30 AG013846NIA NIH HHS P30 AG072978NIGMS NIH HHS T32 GM074905NIGMS NIH HHS T32 GM140972
6 · The paper itself

Abstract

Given their improvements in bias reduction and efficiency, joint models (JMs) for longitudinal and time-to-event data offer great potential to clinical trials. However, for JM to become more widely used, there is a need for additional development of design considerations. To this end, Chen et al previously developed two closed-form sample size formulas in the JM setting. In this current work, we expand upon this framework by utilizing the time-dependent slopes parameterization, where the change in the longitudinal outcome influences the hazard, in addition to the current value of the longitudinal process. Our extended formula for the required number of events can be used when testing significance of the association between the longitudinal and time-to-event outcomes. We find that if the data indeed are generated such that not only the current value, but also the slope of the longitudinal outcome influence the hazard of the time-to-event process, it is advisable to use the current formula developed utilizing the time-dependent slopes parameterization. In this setting, our proposed formula will provide a more accurate estimate of power compared to the method by Chen et al. To illustrate our proposed method, we present power calculations of a biomarker qualification study for Hutchinson-Gilford progeria syndrome, an ultra-rare premature aging disease.

Indexed as

ProgeriaHumansLongitudinal StudiesSample Sizejoint modelinglongitudinal dataprogeriasample sizescore testsurvival analysis

Identifiers

PMID36305571
PMCPMC9771931

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Registered trials

None linked

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.