Adrenocortical carcinoma (ACC) is a rare but aggressive malignancy with high postoperative recurrence rates and poor prognosis.

Published in LabMed Discovery.

Mitotane remains the only approved agent for ACC, exerting antitumor effects by disrupting mitochondrial integrity, inhibiting steroidogenic enzymes, and interfering with cholesterol metabolism.

Clinical evidence supports maintaining plasma concentrations between 14–20 mg/L to maximise efficacy while minimising toxicity.

This comprehensive review outlines mitotane’s mechanisms of action, clinical applications in adjuvant and advanced settings, dosage strategies, therapeutic drug monitoring approaches, and management of frequent adverse effects.

It also discusses emerging combination strategies with chemotherapy and immunotherapy, highlighting the need for personalised treatment and further research into novel therapeutic combinations.

Source: Shanghai Jiao Tong University Journal Center