Mitotane in adrenocortical carcinoma: a profile of its use

Abstract

AbstractThe adrenal cytotoxic agent mitotane (Lysodren®) has a central role in the systemic treatment of adrenocortical carcinoma (ACC), a rare and aggressive cancer of the adrenal glands. Although its precise mechanism of action remains unclear, mitotane has been evaluated and used for more than 60 years and, to date, is the only drug specifically approved for the treatment of ACC. Although ACC continues to be associated with a poor prognosis, mitotane has been shown to provide clinically significant benefit in a good proportion of ACC patients treated with the drug, both in the advanced (unresectable/metastatic) disease and adjuvant therapy settings. While mitotane has generally manageable tolerability with most adverse events (including neurotoxicity) being reversible with dose reduction or treatment interruption, regular monitoring of drug plasma concentrations during treatment is important to help ensure optimal use of mitotane while minimising the impact of drug toxicity.