How effective is mitotane?

The main mechanism of action of mitotane is to destroy the mitochondrial function of adrenocortical cells and inhibit the growth of tumor cells by interfering with the activity of the mitochondrial respiratory chain. It also blocks the synthesis and secretion of cortisol and androgens, thereby reducing tumor size. In a clinical study on mitotane in the treatment of adrenocortical cancer, mitotane, as a first-line treatment strategy, significantly improved the patients' progression-free survival (PFS) and response rate. Specifically, the median disease progression-free survival in the mitotane treatment group reached 17.1 months, demonstrating its significant anti-tumor effect.

In addition to its direct anti-tumor effect, mitotane can also effectively relieve various symptoms caused by excessive secretion of hormones in patients with adrenocortical cancer, such as Cushing's syndrome, hypertension, hypokalemia, etc. Alleviation of these symptoms can help improve patients' quality of life and reduce the pain caused by the disease.

Mitotane can further enhance the therapeutic effect when used in combination with other chemotherapy drugs. For example, mitotane combined with EDP chemotherapy (etoposide, doxorubicin, and cisplatin) significantly prolonged progression-free survival from 2.1 months to 5.0 months in patients with advanced adrenocortical cancer. This combination treatment option provides patients with more treatment options and a better chance of survival.

Although mitotane is highly effective, different patients may respond differently to it. Therefore, doctors will develop a personalized treatment plan based on the patient's specific situation and regularly evaluate the treatment effect. At the same time, mitotane may also cause some side effects during use, such as nausea, vomiting, diarrhea and other gastrointestinal reactions. Doctors will pay close attention to the patient's side effects and promptly adjust the dosage or provide corresponding supportive treatment to ensure the patient's safety and comfort.

References:

https://pmc.ncbi.nlm.nih.gov/articles/PMC5599197/