Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles

Mendelian randomisation is an accessible and valuable epidemiological approach to provide insight into the causal nature of relationships between risk factor exposures and disease outcomes. However, if performed without critical thought, we may simply have replaced one set of implausible assumptions...

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Main Authors: Stephen Burgess, Héléne Toinét Cronjé
Format: Article
Language:English
Published: BMJ Publishing Group 2024-02-01
Series:eGastroenterology
Online Access:https://egastroenterology.bmj.com/content/2/1/e100042.full
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author Stephen Burgess
Héléne Toinét Cronjé
author_facet Stephen Burgess
Héléne Toinét Cronjé
author_sort Stephen Burgess
collection DOAJ
description Mendelian randomisation is an accessible and valuable epidemiological approach to provide insight into the causal nature of relationships between risk factor exposures and disease outcomes. However, if performed without critical thought, we may simply have replaced one set of implausible assumptions (no unmeasured confounding or reverse causation) with another set of implausible assumptions (no pleiotropy or other instrument invalidity). The most critical decision to avoid pleiotropy is which genetic variants to use as instrumental variables. Two broad strategies for instrument selection are a biologically motivated strategy and a genome-wide strategy; in general, a biologically motivated strategy is preferred. In this review, we discuss various ways of implementing a biologically motivated selection strategy: using variants in a coding gene region for the exposure or a gene region that encodes a regulator of exposure levels, using a positive control variable and using a biomarker as the exposure rather than its behavioural proxy. In some cases, a genome-wide analysis can provide important complementary evidence, even when its reliability is questionable. In other cases, a biologically-motivated analysis may not be possible. The choice of genetic variants must be informed by biological and functional considerations where possible, requiring collaboration to combine biological and clinical insights with appropriate statistical methodology.
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spelling doaj-art-3aa5fc423ed54ed2afd1022499a63af62024-12-25T15:50:13ZengBMJ Publishing GroupeGastroenterology2766-01252976-72962024-02-012110.1136/egastro-2023-100042Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principlesStephen Burgess0Héléne Toinét Cronjé12 Cardiovascular Epidemiology Unit, Department of Public Health and Primary Care, University of Cambridge, Cambridge, UK3 Health Analytics, Lane Clark & Peacock LLP, London, UKMendelian randomisation is an accessible and valuable epidemiological approach to provide insight into the causal nature of relationships between risk factor exposures and disease outcomes. However, if performed without critical thought, we may simply have replaced one set of implausible assumptions (no unmeasured confounding or reverse causation) with another set of implausible assumptions (no pleiotropy or other instrument invalidity). The most critical decision to avoid pleiotropy is which genetic variants to use as instrumental variables. Two broad strategies for instrument selection are a biologically motivated strategy and a genome-wide strategy; in general, a biologically motivated strategy is preferred. In this review, we discuss various ways of implementing a biologically motivated selection strategy: using variants in a coding gene region for the exposure or a gene region that encodes a regulator of exposure levels, using a positive control variable and using a biomarker as the exposure rather than its behavioural proxy. In some cases, a genome-wide analysis can provide important complementary evidence, even when its reliability is questionable. In other cases, a biologically-motivated analysis may not be possible. The choice of genetic variants must be informed by biological and functional considerations where possible, requiring collaboration to combine biological and clinical insights with appropriate statistical methodology.https://egastroenterology.bmj.com/content/2/1/e100042.full
spellingShingle Stephen Burgess
Héléne Toinét Cronjé
Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles
eGastroenterology
title Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles
title_full Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles
title_fullStr Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles
title_full_unstemmed Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles
title_short Incorporating biological and clinical insights into variant choice for Mendelian randomisation: examples and principles
title_sort incorporating biological and clinical insights into variant choice for mendelian randomisation examples and principles
url https://egastroenterology.bmj.com/content/2/1/e100042.full
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