Qualifying drug dosing regimens in pediatrics using Gaussian processes

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A1 Alkuperäisartikkeli tieteellisessä aikakauslehdessä

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en

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18

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STATISTICS IN MEDICINE, Volume 40, issue 10, pp. 2355-2372

Abstract

Drug development commonly studies an adult population first and then the pediatric population. The knowledge from the adult population is taken advantage of for the design of the pediatric trials. Adjusted drug doses for these are often derived from adult pharmacokinetic (PK) models which are extrapolated to patients with smaller body size. This extrapolation is based on scaling physiologic model parameters with a body size measure accounting for organ size differences. The inherent assumption is that children are merely small adults. However, children can be subject to additional effects such as organ maturation. These effects are not present in the adult population and are possibly overlooked at the design stage of the pediatric trials. It is thus crucial to qualify the extrapolation assumptions once the pediatric trial data are available. In this work, we propose a model based on a non-parametric regression method called Gaussian process (GP) to detect deviations from the made extrapolation assumptions. We introduce the theoretical background of this model and compare its performance to a parametric expansion of the adult model. The comparison includes simulations and a clinical study data example. The results show that the GP approach can reliably detect maturation trends from sparse pediatric data.

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Siivola, E, Weber, S & Vehtari, A 2021, 'Qualifying drug dosing regimens in pediatrics using Gaussian processes', STATISTICS IN MEDICINE, vol. 40, no. 10, pp. 2355-2372. https://doi.org/10.1002/sim.8907