Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of…

Randomized trialFrontiers in pharmacology· 2026

Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of aspergillosis: an exploratory randomized pilot study with a cost-consequence analysis.

Jaime Monserrat-Villatoro, David Bueno, Miriam Estébanez … Alberto M Borobia

Pediatric Hematology & OncologyRandomized trialTreatmentQ1IF 5.1

Genotype-guided dosing lowered early subtherapeutic voriconazole levels in pediatric hematology patients.

Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of aspergillosis: an exploratory randomized pilot study with a cost-consequence analysis.

Jaime Monserrat-Villatoro … Alberto M Borobia

Frontiers in pharmacology · 2026

Background

Voriconazole is a first-line therapy for invasive aspergillosis, but its use is complicated by pronounced pharmacokinetic variability, largely driven by CYP2C19 polymorphisms.

Purpose

We aimed to evaluate whether preemptive CYP2C19 genotyping improves early target attainment and clinical management in pediatric hematology patients compared with standard practice.

Methods

We conducted a pragmatic, randomized, single-blind trial (EudraCT: 2019-000376-41; ClinicalTrials.gov: NCT04238884).

n = Thirty-two patients (16 per arm)

Results

genotype-guided dosing cut early subtherapeutic voriconazole levels by more than half

31.3%

Genotype-guided

68.8%

Standard-of-care

More results

An exploratory cost-consequence analysis was associated with a mean difference in direct medical costs of €5,819 per patient in favour of the genotype-guided arm, although this difference was not statistically significant (p = 0.300).

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Conclusion

Preemptive CYP2C19 genotyping was associated with a lower proportion of early subtherapeutic voriconazole exposure compared with standard care, with no signal of increased toxicity.

Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of aspergillosis: an exploratory randomized pilot study with a cost-consequence analysis.

Pre-emptive CYP2C19 genotyping and voriconazole exposure in pediatric hematology patients at risk of aspergillosis: an exploratory randomized pilot study with a cost-consequence analysis.

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