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

This is the dataset of a pragmatic, randomized, single-blind trial (EudraCT: 2019-000376-41; ClinicalTrials.gov: NCT04238884), including pediatric hematology patients at high risk of aspergillosis. The patients were randomized (1:1) to receive either standard weight-based dosing (Control) or preemptive CYP2C19 (*1, *2, *3, *17 alleles) genotype-guided dosing (Experimental). The primary endpoint was the proportion of patients achieving a therapeutic voriconazole trough concentration (Cmin: 1.0–5.0 mg/L) at Day 5 (±1). The analysis was conducted using a modified intention-to-treat approach.