Key result
Pharmacogenetics guides pediatric cardiac drug dosing and selection despite limited pediatric-specific trial data.
Why the study?
Variability in drug response and medication-related adverse events remain significant challenges in pediatric cardiology due to complex pharmacotherapy with off-label cardiovascular medications.
Integration of pharmacogenetic testing into pediatric cardiology practice may improve therapeutic precision, enhance safety, and reduce preventable adverse drug events for commonly used cardiovascular medications.
May support selective use of CPIC-guided testing in pediatric cardiology; leaves open the need for pediatric outcome trials.
Children with congenital and acquired heart disease frequently require complex and prolonged pharmacotherapy, often involving off-label cardiovascular medications with narrow therapeutic ranges. Consequently, variability in drug response and medication-related adverse events remains a significant clinical challenge in pediatric cardiology. Pharmacogenetics provides a framework to individualize drug selection and dosing by incorporating genetic variability alongside established clinical and developmental factors. This review outlines the current clinical utility and emerging role of pharmacogenetics in pediatric cardiology, emphasizing applications most relevant to day-to-day patient care. Established pharmacogenetic associations with direct implications for pediatric cardiac practice include gene–drug pairs involving tacrolimus ( CYP3A5 ), warfarin ( CYP2C9 , VKORC1, CYP4F2, rs12777823 within the CYP2C) , clopidogrel ( CYP2C19) , flecainide ( CYP2D6) , metoprolol ( CYP2D6) , and statins ( SLCO1B1, ABCG2, CYP2C9 ), many of which are supported by guideline-based recommendations from the Clinical Pharmacogenetics Implementation Consortium. This review also highlights pharmacogenetic considerations for commonly prescribed non-cardiac medications that may influence cardiovascular outcomes in children with heart disease, identifies emerging areas for research, and emphasizes that as pharmacogenetic testing becomes increasingly accessible, its thoughtful integration into pediatric cardiology practice may improve therapeutic precision, enhance safety, and reduce preventable adverse drug events.
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Adenikinju et al. (2026) conducted a review in Pediatric heart disease. Pharmacogenetic-guided dosing vs. Standard dosing was evaluated. Pharmacogenetics provides a framework to individualize drug selection and dosing in pediatric cardiology, with established guidelines for medications like tacrolimus, warfarin, and clopidogrel, despite limited pediatric-specific data.
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