Evidence map›Paper›PMID 33407760›Full record

Trial reportTrials2021

A comparison of approaches for combining predictive markers for personalised treatment recommendations.

Matthias Pierce, Richard Emsley

Open access · goldAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Trials, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.3field-weighted citation impact, top 46% of its field
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, 4 citations in OpenAlex.

  1. Article
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

2 authors at 2 institutions in 1 country.

Matthias PierceCentre for Biostatistics, School of Health Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester Academic Health Science Centre, 1st Floor, Jean McFarlane Building, Oxford Road, Manchester, M13 9PL, UK. matthias.pierce@manchester.ac.uk.ORCID http://orcid.org/0000-0003-2182-0369
Richard EmsleyDepartment of Biostatistics and Health Informatics, Institute of Psychiatry, Psychology and Neuroscience, King's College London, London, UK.
King's College London · GBManchester Academic Health Science Centre · GB

Funding

Medical Research Council MR/K025635/1
6 · The paper itself

Abstract

backgroundIn the presence of heterogeneous treatment effects, it is desirable to divide patients into subgroups based on their expected response to treatment. This is formalised via a personalised treatment recommendation: an algorithm that uses biomarker measurements to select treatments. It could be that multiple, rather than single, biomarkers better predict these subgroups. However, finding the optimal combination of multiple biomarkers can be a difficult prediction problem.

methodsWe described three parametric methods for finding the optimal combination of biomarkers in a personalised treatment recommendation, using randomised trial data: a regression approach that models outcome using treatment by biomarker interactions; an approach proposed by Kraemer that forms a combined measure from individual biomarker weights, calculated on all treated and control pairs; and a novel modification of Kraemer's approach that utilises a prognostic score to sample matched treated and control subjects. Using Monte Carlo simulations under multiple data-generating models, we compare these approaches and draw conclusions based on a measure of improvement under a personalised treatment recommendation compared to a standard treatment. The three methods are applied to data from a randomised trial of home-delivered pragmatic rehabilitation versus treatment as usual for patients with chronic fatigue syndrome (the FINE trial). Prior analysis of this data indicated some treatment effect heterogeneity from multiple, correlated biomarkers.

resultsThe regression approach outperformed Kraemer's approach across all data-generating scenarios. The modification of Kraemer's approach leads to improved treatment recommendations, except in the case where there was a strong unobserved prognostic biomarker. In the FINE example, the regression method indicated a weak improvement under its personalised treatment recommendation algorithm.

conclusionsThe method proposed by Kraemer does not perform better than a regression approach for combining multiple biomarkers. All methods are sensitive to misspecification of the parametric models.

Indexed as

Fatigue Syndrome, ChronicPrecision MedicineBiomarkersHumansResearch DesignBiomarkersModeratorsPersonalised medicinePersonalised treatment recommendationsPrecision medicinePredictive biomarkersStratified medicine

Identifiers

PMID33407760
PMCPMC7788953
OpenAlexW3120073847

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.