mitotan

Mitotane, also known by the brand name Lysodren, is an adrenolytic anticancer medication used primarily for adrenocortical carcinoma (ACC). It is administered orally and typically given in a slow, dose-escalation regimen to reach a target blood concentration. Because of its long half-life and pharmacokinetic properties, dosing is often gradual and may require divided doses or prolonged titration.

Mechanism of action is not fully understood, but mitotane is selectively cytotoxic to adrenal cortical cells and inhibits steroid synthesis, resulting in decreased production of cortisol and other adrenal steroids. It can cause atrophy of the adrenal cortex. The drug is sometimes used to control hypercortisolism in Cushing’s syndrome due to adrenal tumors.

Clinical use centers on unresectable, metastatic, or recurrent ACC, where mitotane may be used as a primary therapy or as adjuvant treatment. It may also be used to suppress cortisol production in patients with ACTH-independent Cushing's syndrome related to adrenal tumors.

Administration and monitoring require regular laboratory and clinical assessment. Therapeutic drug monitoring aims for plasma levels roughly in the 14–20 mg/L range. Patients are monitored for adrenal function, electrolytes, liver enzymes, and neurological status, as mitotane can cause fatigue, dizziness, ataxia, gastrointestinal upset, and rash. Long-term therapy carries risks such as adrenal insufficiency, potential thyroid abnormalities, and hematologic effects. Because mitotane induces and inhibits hepatic enzymes, it can interact with other medications, including anticoagulants and certain antidiabetic drugs.

See also: Adrenocortical carcinoma, Cushing's syndrome, adrenolytic therapy, Lysodren.