MR employs genetic variants as instrumental variables (Z) to estimate the causal effect of an exposure (X) on an outcome (Y) using observational data, even in the presence of unmeasured confounding (C).
For univariable MR to provide valid causal estimates of the effect of the exposure on the outcome, there are three assumptions that must be satisfied:
When these assumptions are satisfied, univariable MR can test for a causal effect of an exposure on an outcome without bias from unobserved confounding. With an additional assumption, univariable MR will give an estimate of the size of the causal effect of the exposure on the outcome.
The “exclusion restriction” assumption requires no pleiotropy, i.e., no other pathways from Z to Y except through X.
In practice, however, many variants are pleiotropic. For example, SNPs for lipid fractions.
Multivariable MR is an extension of univariableMR that allows us to estimate the causal effects of multiple exposures on an outcome. Multivariable MR estimates the “direct” causal effects of each exposure included in the estimation on the outcome, conditional on the other exposures.
There are also three assumptions:
\[ \begin{bmatrix} Z1 \\ Z2 \end{bmatrix} \sim N (\begin{bmatrix} 0 \\ 0 \end{bmatrix},\,\begin{bmatrix} 1 ,0.5\\ 0.5 ,1 \end{bmatrix})\, \]
\[ C \sim N (0,1) \]
\[ X1 = 0.4\times Z_1 + 0.3\times Z_1 \times Z_2 + 0.1\times Z_2 + 0.5 \times C + \epsilon_1 \\ X2 = 0.1 \times Z_1 + 0.5\times Z_2 + 0.3\times Z_2^2 + 0.5 \times C + \epsilon_2 \\ \epsilon_1 {\perp \!\!\! \perp} \epsilon_2 \sim N(0,1) \]
\[ Y = -0.3\times X1 + 0.5\times X2 - 0.8 \times C + \epsilon, \,\,\, \epsilon \sim N(0,1) \]
\[ Y\sim X_1+X_2 \]
\[ Y\sim X_1+X_2 + C \]
\[ X_1 \sim Z_1 + Z_2 \\ X_2 \sim Z_1 + Z_2 \\ Y \sim {\hat{X}}_1 + {\hat X}_2 \]
Limitations:
Several previous studies using multivariable MR to disentangle lipids effects or effects on lipids:
Richardson TG, Sanderson E, Palmer TM, Ala-Korpela M, Ference BA, Davey Smith G, Holmes MV. Evaluating the relationship between circulating lipoprotein lipids and apolipoproteins with risk of coronary heart disease: A multivariable Mendelian randomisation analysis. PLoS medicine. 2020 Mar 23;17(3):e1003062.
Nazarzadeh M, Pinho-Gomes AC, Bidel Z, Dehghan A, Canoy D, Hassaine A, Ayala Solares JR, Salimi-Khorshidi G, Smith GD, Otto CM, Rahimi K. Plasma lipids and risk of aortic valve stenosis: a Mendelian randomization study. European heart journal. 2020 Oct 21;41(40):3913-20.
Allara E, Morani G, Carter P, Gkatzionis A, Zuber V, Foley CN, Rees JM, Mason AM, Bell S, Gill D, Lindström S. Genetic determinants of lipids and cardiovascular disease outcomes: a wide-angled Mendelian randomization investigation. Circulation: Genomic and Precision Medicine. 2019 Dec;12(12):e002711.
Hindy G, Engström G, Larsson SC, Traylor M, Markus HS, Melander O, Orho-Melander M. Role of blood lipids in the development of ischemic stroke and its subtypes: a Mendelian randomization study. Stroke. 2018 Apr;49(4):820-7.
Ioannidou A, Watts EL, Perez-Cornago A, Platz EA, Mills IG, Key TJ, Travis RC, PRACTICAL consortium, CRUK, BPC3, CAPS, PEGASUS, Tsilidis KK, Zuber V. The relationship between lipoprotein A and other lipids with prostate cancer risk: A multivariable Mendelian randomisation study. PLoS medicine. 2022 Jan 27;19(1):e1003859.
Bell JA, Richardson TG, Wang Q, Sanderson E, Palmer T, Walker V, O’Keeffe LM, Timpson NJ, Cichonska A, Julkunen H, Würtz P. Effects of general and central adiposity on circulating lipoprotein, lipid, and metabolite levels in UK Biobank: a multivariable Mendelian randomization study. The Lancet Regional Health–Europe. 2022 Oct 1;21.