Evidence map›Paper›PMID 34464990›Full record

Observational studyStatistics in medicine2021

Penalized regression calibration: A method for the prediction of survival outcomes using complex longitudinal and high-dimensional data.

Mirko Signorelli, Pietro Spitali, Cristina Al-Khalili Szigyarto, MARK-MD Consortium, Roula Tsonaka

Abstract readObservational Study
In one paragraph

Observational study in Statistics in medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

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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

5 authors.

Mirko SignorelliMathematical Institute, Leiden University, Leiden, The Netherlands.ORCID 0000-0002-8102-3356
Pietro SpitaliDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.ORCID 0000-0003-2783-688X
Cristina Al-Khalili SzigyartoDepartment of Protein Science, KTH-Royal Institute of Technology, Stockholm, Sweden.ORCID 0000-0001-6990-1905
MARK-MD Consortium
Roula TsonakaDepartment of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Longitudinal and high-dimensional measurements have become increasingly common in biomedical research. However, methods to predict survival outcomes using covariates that are both longitudinal and high-dimensional are currently missing. In this article, we propose penalized regression calibration (PRC), a method that can be employed to predict survival in such situations. PRC comprises three modeling steps: First, the trajectories described by the longitudinal predictors are flexibly modeled through the specification of multivariate mixed effects models. Second, subject-specific summaries of the longitudinal trajectories are derived from the fitted mixed models. Third, the time to event outcome is predicted using the subject-specific summaries as covariates in a penalized Cox model. To ensure a proper internal validation of the fitted PRC models, we furthermore develop a cluster bootstrap optimism correction procedure that allows to correct for the optimistic bias of apparent measures of predictiveness. PRC and the CBOCP are implemented in the R package pencal, available from CRAN. After studying the behavior of PRC via simulations, we conclude by illustrating an application of PRC to data from an observational study that involved patients affected by Duchenne muscular dystrophy, where the goal is predict time to loss of ambulation using longitudinal blood biomarkers.

Indexed as

CalibrationBiasBiomarkersHumansLongitudinal StudiesProportional Hazards ModelsBiomarkersDuchenne muscular dystrophyhigh-dimensionalitylongitudinal data analysisoptimism correctionpenalized regression calibrationrisk prediction modelingsurvival analysis

Identifiers

PMID34464990
PMCPMC9293191

What OpenQuestion holds

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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.