Evaluating antifungal prophylaxis strategies in pediatric acute myeloid leukemia: a retrospective, single-center analysis of amphotericin B, itraconazole, and voriconazole

Summary Purpose Invasive fungal disease (IFD) represents a major cause of morbidity and mortality in pediatric patients with acute myeloid leukemia (AML) undergoing intensive chemotherapy treatment. While primary antifungal prophylaxis is known to reduce the incidence of IFD, data on pediatric AML patients are sparse. This study assessed the usage and outcomes of antifungal prophylaxis in this high-risk group, contributing to the limited data available on pediatric AML. Material and methods We conducted a retrospective analysis of 394 cycles of antifungal prophylaxis in 92 pediatric patients with de novo AML at our center. Prophylaxis included amphotericin B derivatives ( n = 139), itraconazole ( n = 107), or voriconazole ( n = 148), reflecting varied clinical choices over the study period. Results At least one adverse event was observed in 93% of cycles with antifungal prophylaxis. Most patients experienced only low-grade toxicity, and there was no life-threatening adverse event. Creatinine increase, potassium loss, and episodes with vomiting were significantly more frequent with amphotericin B prophylaxis. Discontinuation of antifungal prophylaxis due to an adverse event was necessary in 3% of cycles. The observed incidence of IFD was 7% across the patient cohort, with no significant difference between drugs. No IFD-related death was reported. Conclusion Our analysis highlights a reasonable balance between tolerability and efficacy of antifungal prophylaxis in pediatric AML patients. While the incidence of IFD aligns with previous reports, our cohort demonstrated notably lower mortality. This retrospective audit supports the continued use of voriconazole for its lower associated toxicity, providing a valuable reference for antifungal management in pediatric AML settings.