Comprehensive Analysis of Mitotane-Related Adverse Events Using the FDA Adverse Event Reporting System.

Objective: Mitotane is currently the only product approved by the FDA for the treatment of adrenocortical cancer. However, there is a lack of comprehensive studies on the adverse events of mitotane.

Methods: Adverse event reports for mitotane in the FDA Adverse Event Reporting System database since 2004 were collected and analysed to identify mitotane as the primary suspect drug. Reporting ratios, multi-item γ Poisson constrictors, proportional reporting ratios, and Bayesian confidence propagation neural networks were used to analyse the disproportionality of mitotane-related adverse events.

Results: A total of 21,433,114 adverse event reports were retrieved from the FAERS database, with 772 cases identified where mitotane was the primary suspected drug. Positive signals were observed for adverse reactions listed on the drug label, such as nausea, diarrhoea, vomiting, dizziness, loss of appetite, and adrenal insufficiency. Additionally, potential adverse reactions not specified on the label were detected, including fatigue, malignant tumour progression, ovarian cysts, chills, amnesia, and QT interval prolongation on the electrocardiogram. These findings highlight the critical need for vigilant monitoring of adverse events, particularly during the first few months of treatment.

Conclusion: This study provides preliminary safety data on the practical application of mitotane, confirming some known adverse reactions and revealing other potential risks. These findings provide critical safety information for clinicians prescribing mitotane for the treatment of adrenocortical cancer.