Evidence map›Paper›PMID 30073700›Full record

ArticleStatistics in medicine2018

Using marginal structural models to adjust for treatment drop-in when developing clinical prediction models.

Matthew Sperrin, Glen P Martin, Alexander Pate, Tjeerd Van Staa, Niels Peek, Iain Buchan

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Article in Statistics in medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed, 2 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

32 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
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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

6 authors.

Matthew SperrinFarr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.ORCID 0000-0002-5351-9960
Glen P MartinFarr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.ORCID 0000-0002-3410-9472
Alexander PateFarr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.
Tjeerd Van StaaFarr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.
Niels PeekFarr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.
Iain BuchanFarr Institute, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, Manchester, UK.

Funding

Medical Research Council MC_PC_13042Medical Research Council MR/K006665/1
6 · The paper itself

Abstract

Clinical prediction models (CPMs) can inform decision making about treatment initiation, which requires predicted risks assuming no treatment is given. However, this is challenging since CPMs are usually derived using data sets where patients received treatment, often initiated postbaseline as "treatment drop-ins." This study proposes the use of marginal structural models (MSMs) to adjust for treatment drop-in. We illustrate the use of MSMs in the CPM framework through simulation studies that represent randomized controlled trials and real-world observational data and the example of statin initiation for cardiovascular disease prevention. The simulations include a binary treatment and a covariate, each recorded at two timepoints and having a prognostic effect on a binary outcome. The bias in predicted risk was examined in a model ignoring treatment, a model fitted on treatment-naïve patients (at baseline), a model including baseline treatment, and the MSM. In all simulation scenarios, all models except the MSM underestimated the risk of outcome given absence of treatment. These results were supported in the statin initiation example, which showed that ignoring statin initiation postbaseline resulted in models that significantly underestimated the risk of a cardiovascular disease event occurring within 10 years. Consequently, CPMs that do not acknowledge treatment drop-in can lead to underallocation of treatment. In conclusion, when developing CPMs to predict treatment-naïve risk, researchers should consider using MSMs to adjust for treatment drop-in, and also seek to exploit the ability of MSMs to allow estimation of individual treatment effects.

Indexed as

Decision Support TechniquesCardiovascular DiseasesHumansHydroxymethylglutaryl-CoA Reductase InhibitorsModels, StatisticalPrognosisRandomized Controlled Trials as TopicTreatment OutcomeHydroxymethylglutaryl-CoA Reductase Inhibitorsclinical prediction modelscounterfactual causal inferencelongitudinal datamarginal structural modelstreatment drop-invalidation

Identifiers

PMID30073700
PMCPMC6282523

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LicenceCC BY
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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.