Pharmacovigilance of carmustine: distinct neurotoxic and systemic safety signals identified in FAERS

Carmustine (BCNU), a nitrosourea alkylating agent, is used in malignant gliomas, lymphomas, and hematopoietic stem cell transplantation. Although its major toxicities are recognized, comprehensive postmarketing evidence on the broader spectrum of carmustine related adverse event signals remains limited. This study evaluated carmustine related reporting signals using real world pharmacovigilance data. Adverse drug reaction (ADR) reports identifying BCNU as the primary suspect were extracted from the FDA Adverse Event Reporting System (FAERS, Q1 2004–Q1 2024). Disproportionality analyses using reporting odds ratio (ROR), proportional reporting ratio (PRR), information component (IC), and empirical Bayesian geometric mean (EBGM) were performed at both system organ class (SOC) and preferred term (PT) levels, with stratification by sex. In total, 718 BCNU reports with 3,091 preferred terms were analyzed. Significant signals emerged for nervous system disorders (ROR = 2.74), blood and lymphatic system disorders (ROR = 2.95), and infections (ROR = 2.15). At the PT level, eosinophilic meningitis, pneumocephalus, and intracranial hypotension showed strong disproportional reporting signals among carmustine related reports. Descriptive sex stratified reporting patterns were observed, including intracranial hypotension in female reports and pseudomeningocele in male reports. Carmustine related FAERS reports showed distinct neurotoxic, hematologic, and procedural reporting patterns, some with descriptive sex specific differences. These findings emphasize the need for continued post-marketing surveillance to inform clinical practice and optimize risk mitigation. This study highlights that BCNU is linked to significant neurotoxic and hematologic complications, including nervous system disorders and blood-related issues, as well as infections. Some adverse events, such as intracranial hypotension in female reports and pseudomeningocele in male reports, showed descriptive sex stratified reporting patterns, which warrant cautious interpretation and further validation. The findings underscore the importance of ongoing post-marketing surveillance to better manage the risks associated with BCNU in clinical settings.