Voriconazole in the treatment of pediatric patients with hematologic malignancies and invasive fungal infections: a real-world study.

Purpose: To evaluate the factors influencing voriconazole (VRC) administration, clinical efficacy, and safety in children with hematological malignancies (HM) and invasive fungal infection (IFD).

Methods: This is a single-center, retrospective real-world study conducted between July 2018 and December 2023. Univariate and multivariate logistic regression analysis were used to analyze the affecting factors.

Results: A total of 167 patients were included in this study. Among them, 13.77% (23/167) of children under 2 years old received off-label use of VRC, with an initial maintenance dose higher than that of other groups (P < 0.001). There were 8 cases (4.79%) of cured, 148 cases (88.62%) of improved, and 11 cases (6.59%) of ineffective. Thirty-eight cases experienced adverse drug reactions (ADR), with a highest incidence (10.2%) of hepatotoxicity. The concomitant proton pump inhibitors (PPIs), days of central venous catheterization and coagulopathy were independent influencing factors of ADR. Sixty-six patients underwent therapeutic drug monitoring (TDM), which increased the probability of achieving the target plasma trough concentration (C). Among children < 2 years old underwent TDM, 88.89% (8/9) achieved therapeutic concentration, and the probability was higher than that of the older groups. Days of VRC treatment had a positive but not statistically significant effect on achievement of target C. Hypoalbuminemia and days of antimicrobials treatment were independent influencing factors of C distribution.

Conclusion: Attention to the off-label use of VRC in children < 2 years old, hypoalbuminemia and coagulopathy correction, potential drug interactions with VRC, and ADR monitoring is crucial for clinical efficacy and safety.