Evidence map›Paper›PMID 37086186›Full record

Observational studyStatistics in medicine2023

Causal inference in survival analysis using longitudinal observational data: Sequential trials and marginal structural models.

Ruth H Keogh, Jon Michael Gran, Shaun R Seaman, Gwyneth Davies, Stijn Vansteelandt

Abstract readObservational Study
In one paragraph

Observational study in Statistics in medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.

0numbers the graph read from it
0cells of the map it votes in
27citing 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

27 citing papers in PubMed.

  1. Article
  2. Article
  3. Causal graph neural networks for healthcare.Nature biomedical engineering · 2026
    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

5 authors.

Ruth H KeoghDepartment of Medical Statistics and Centre for Statistical Methodology, London School of Hygiene and Tropical Medicine, Keppel Street, London, WC1E 7HT, UK.ORCID 0000-0001-6504-3253
Jon Michael GranOslo Centre for Biostatistics and Epidemiology, Department of Biostatistics, Institute of Basic Medical Sciences, University of Oslo, P.O. Box 1122 Blindern, Oslo, 0317, Norway.
Shaun R SeamanMRC Biostatistics Unit, University of Cambridge, East Forvie Building, Forvie Site, Robinson Way, Cambridge, CB2 0SR, UK.ORCID 0000-0003-3726-5937
Gwyneth DaviesPopulation, Policy and Practice Research and Teaching Department, UCL Great Ormond Street Institute of Child Health, University College London, WC1N 1EH, London, UK.ORCID 0000-0001-7937-2728
Stijn VansteelandtDepartment of Medical Statistics and Centre for Statistical Methodology, London School of Hygiene and Tropical Medicine, Keppel Street, London, WC1E 7HT, UK.ORCID 0000-0002-4207-8733

Funding

Medical Research Council MC_UU_00002/10Medical Research Council MR/S017968/1Medical Research Council MR/T041285/1
6 · The paper itself

Abstract

Longitudinal observational data on patients can be used to investigate causal effects of time-varying treatments on time-to-event outcomes. Several methods have been developed for estimating such effects by controlling for the time-dependent confounding that typically occurs. The most commonly used is marginal structural models (MSM) estimated using inverse probability of treatment weights (IPTW) (MSM-IPTW). An alternative, the sequential trials approach, is increasingly popular, and involves creating a sequence of "trials" from new time origins and comparing treatment initiators and non-initiators. Individuals are censored when they deviate from their treatment assignment at the start of each "trial" (initiator or noninitiator), which is accounted for using inverse probability of censoring weights. The analysis uses data combined across trials. We show that the sequential trials approach can estimate the parameters of a particular MSM. The causal estimand that we focus on is the marginal risk difference between the sustained treatment strategies of "always treat" vs "never treat." We compare how the sequential trials approach and MSM-IPTW estimate this estimand, and discuss their assumptions and how data are used differently. The performance of the two approaches is compared in a simulation study. The sequential trials approach, which tends to involve less extreme weights than MSM-IPTW, results in greater efficiency for estimating the marginal risk difference at most follow-up times, but this can, in certain scenarios, be reversed at later time points and relies on modelling assumptions. We apply the methods to longitudinal observational data from the UK Cystic Fibrosis Registry to estimate the effect of dornase alfa on survival.

Indexed as

Models, StatisticalCausalityHumansLongitudinal StudiesModels, StructuralProbabilitySurvival AnalysisTreatment Outcomecystic fibrosisinverse probability weightingmarginal structural modelregistriessequential trialssurvivaltarget trialstime-dependent confounding

Identifiers

PMID37086186
PMCPMC7614580

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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.