Adrenocortical carcinoma (ACC) is characterized as a rare but aggressive malignancy, associated with high postoperative recurrence rates and poor prognosis.
Mitotane is the sole approved agent for ACC. Its antitumor effects are attributed to the disruption of mitochondrial integrity, inhibition of steroidogenic enzymes, and interference with cholesterol metabolism.
Clinical evidence indicates that maintaining plasma concentrations of mitotane between 14–20 mg/L optimizes efficacy while minimizing toxicity.
This review details mitotane's mechanisms of action, its clinical utility in both adjuvant and advanced treatment settings, recommended dosage strategies, therapeutic drug monitoring methods, and approaches for managing common adverse effects.
The review also explores developing combination therapies involving chemotherapy and immunotherapy, emphasizing the need for personalized treatment plans and further investigation into new therapeutic combinations.