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Published on: October 17, 2025
Pharmacogenomics in pediatric leukemia.
Steven W Paugh1, Gabriele Stocco, William E Evans
1Hematological Malignancies Program and Department of Pharmaceutical Sciences, St Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.
Genetic variations influence how children respond to medications, particularly cancer drugs. Understanding these pharmacogenomic factors can help optimize treatment, improving efficacy and reducing toxicity in pediatric patients.
Area of Science:
- Genomics
- Pediatric Pharmacology
- Cancer Therapeutics
Background:
- Children often have a narrow therapeutic index for medications, increasing toxicity risk.
- Balancing drug toxicity and efficacy is crucial, especially in pediatric cancer chemotherapy.
- Interpatient variability in drug clearance affects treatment outcomes.
Purpose of the Study:
- To review genetic factors influencing drug pharmacokinetics and pharmacodynamics in children.
- To explore how genetic variations impact medication response in pediatric populations.
- To discuss the implications for optimizing childhood cancer treatment.
Main Methods:
- Review of genome-wide association studies in pediatric patient cohorts.
- Analysis of genetic variations affecting drug metabolism and response.
- Exploration of polygenic models for predicting drug response phenotypes.
Main Results:
- Genome-wide studies identify genetic variations impacting disease susceptibility and drug characteristics.
- Multiple genetic factors contribute to pharmacogenomic phenotypes and drug response.
- Findings offer insights into disease mechanisms and potential molecular diagnostics.
Conclusions:
- Advances in genomic technology are key to understanding interpatient drug response differences.
- Systematic deployment of genomics can enhance pediatric drug efficacy.
- Genomic insights promise to reduce drug toxicity in children.
Related Concept Videos
Pharmacogenetic Phenotypes: Alterations in Pharmacokinetics, Drug Targets and Biologic Milieu
Pharmacokinetics in Pediatric Patients: Drug Metabolism
Pharmacogenomics: Identification of New Drug Targets
Pharmacogenetics and Pharmacogenomics: Overview
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase
Pharmacokinetics in Pediatric Patients: Drug Excretion

