Neutropenia and infectious events during off-label treatment with venetoclax in children with malignant disease: a pharmacovigilance analysis of FDA adverse event reporting system reports

Venetoclax, a BCL-2 inhibitor, is increasingly used off-label in pediatric malignancies, but safety data remain limited. This pharmacovigilance study analyzed 488 pediatric reports from the FDA Adverse Event Reporting System (FAERS) involving neutropenia and infection with venetoclax use. Disproportionately reported events included sepsis, leukopenia/agranulocytosis, and pseudomembranous colitis. Most cases involved acute myeloid leukemia or myelodysplastic syndromes and combination therapy. Network analysis revealed co-reporting of hematologic and infectious toxicities. These findings emphasize the need for vigilant monitoring and dedicated pediatric studies to guide venetoclax use and improve supportive care strategies in this vulnerable population receiving intensive or multimodal treatments.