III-15 Julie Bertrand

Population approach in high-throughput pharmacogenetics: challenging the maximum likelihood approaches and exploration of a Bayesian alternative

Julie Bertrand (1), Maria De Iorio (2), David J. Balding (1)

(1) Genetics Institute, University College London, London, UK, (2) Department of Statistical Science, University College London, London, UK

Context: In a previous study, we have shown that an integrated approach, simultaneously estimating the PK model parameters and the genetic size effects, was, on a rich design, just as powerful as the classical stepwise procedure for covariate building to detect the effect of 6 causal variants from a large single nucleotide polymorphisms (SNPs) array [1].

Objectives: To challenge the integrated approach and the classical stepwise procedure on i/ genetic predictors of multiple PK parameters and ii/ a challenging study design. To explore a Bayesian alternative.

Methods: We simulated a two compartments PK model, with parameter values from a real-case study. From the initial scenario with N=300 subjects/n=6 sampling times and the effect of 6 SNPs on the apparent elimination clearance (CL/F) only, we now simulated i/ one scenario with the same design but the effect of 2 pairs of correlated SNPs each affecting CL/F and the central compartment apparent volume of distribution and ii/ one scenario with the same 6 causal variants on CL/F but 700 supplementary subjects having only a trough concentration.

Our integrated approach includes a penalized regression at each iteration of the Stochastic Approximation Expectation Maximization algorithm. Two penalization are considered here; Lasso and its generalization with heavier tails, HyperLasso. The full-Bayesian alternative is implemented in R using the R2jags package. For the SNP effect size we used a double-exponential (DE) prior to mimic Lasso shrinkage. The penalty parameters are set to ensure a 20% family wise error rate using an asymptotic approximation-based formula corrected for the information in the design.

Results: When genetic predictors affect multiple PK parameters, the integrated approach is more powerful than the stepwise procedure with 637 (Lasso) and 639 (HyperLasso) versus 596 true positive (on a maximum of 800).

References:
[1] Penalized regression implementation within the SAEM algorithm to advance high-throughput personalized drug therapy. Bertrand J, Bading DJ, De Iorio M. PAGE 2013.

Reference: PAGE 23 () Abstr 3205 [www.page-meeting.org/?abstract=3205]

Poster: Methodology - Covariate/Variability Models

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