A medicine for cancer of the adrenal gland

Mitotane

A specialist medicine used to treat cancer of the adrenal gland, which also suppresses the gland's normal hormone production.

What is Mitotane?

Mitotane is a specialist medicine used to treat adrenocortical carcinoma, a rare cancer of the outer part of the adrenal gland. It works by acting directly on adrenal cortex cells, both attacking the cancer and reducing the gland's production of cortisol. Because it suppresses normal cortisol, almost everyone taking it needs steroid replacement, and they must learn 'sick day rules' to increase cover during illness or stress. It can also cause nervous-system side effects, builds up slowly in the body, and is harmful in pregnancy, so reliable contraception is essential. It is given only under close specialist supervision with blood-level monitoring.

Education and reference only. This is a plain-language guide to Mitotane — it deliberately contains no doses. Doses depend on the person, the brand and the reason for treatment, and belong with your prescriber. Always check the BNF, the product labelling (SmPC) and follow medical advice.

Brands: Lysodren
Mitotane (Adrenal cytotoxic agent (adrenocortical carcinoma)) — Meds Global Health reference card with 2D molecular structure
Mitotane — Adrenal cytotoxic agent (adrenocortical carcinoma). The image shows the active ingredient's 2D molecular structure.

What it is

Mitotane is a medicine used to treat adrenocortical carcinoma, a rare and serious cancer of the cortex (outer layer) of the adrenal gland. It is taken by mouth and acts specifically on adrenal cortex cells, both damaging the cancer and reducing the gland's ability to make hormones, particularly cortisol. Because of this, it is used together with steroid replacement to make up for the cortisol the body can no longer produce. It builds up slowly in the body's fat and stays around for a long time after stopping. It is a powerful, specialist treatment used only under close supervision by an expert cancer and endocrine team.

How it works

Mitotane targets the cells of the adrenal cortex, the part of the adrenal gland affected by adrenocortical carcinoma. It damages and reduces the activity of these cells, which both works against the cancer and lowers the gland's production of steroid hormones, especially cortisol. Because it knocks out so much normal cortisol production, the body is left short, which is why steroid replacement is given alongside. The medicine dissolves into body fat and accumulates slowly, so it can take time to build up to an effective level and a long time to clear, which is why blood levels are measured to guide treatment.

Company & origin

Originated / developed by: Specialist manufacturer.

A specialist medicine used in the UK to treat cancer of the adrenal cortex (adrenocortical carcinoma).

Practical use

How to take Mitotane

General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.

  • Take it by mouth as prescribed; taking it with food, especially fatty food, helps it be absorbed.
  • Always take your steroid replacement alongside it and never stop the steroid; carry a steroid emergency card.
  • Follow your 'sick day rules': increase your steroid during illness, injury or stress, and seek urgent help if you cannot keep medicines down.
  • Use reliable contraception during treatment and for a long time afterwards, as it is harmful in pregnancy and lingers in the body.
  • Report drowsiness, confusion, unsteadiness or slurred speech, as these can mean the level is too high.

Weighing it up

Advantages & disadvantages of Mitotane

Advantages

  • One of the main specific treatments available for adrenocortical carcinoma, a rare adrenal cancer.
  • Acts directly on adrenal cortex cells, targeting the affected tissue.
  • Taken by mouth, with blood-level monitoring to guide treatment.

Disadvantages

  • Suppresses the body's own cortisol, so steroid replacement and 'sick day rules' are essential to avoid an adrenal crisis.
  • Commonly causes nervous-system effects such as drowsiness, confusion and unsteadiness.
  • Harmful in pregnancy and lingers in the body for a long time, so reliable contraception is needed during and after treatment.

Practical use

Good to know

Several things make mitotane unusually important to understand. First, because it suppresses the body's own cortisol, almost everyone needs steroid replacement and must follow 'sick day rules', increasing their steroid during illness, injury, infection or stress, and seeking urgent help if they cannot keep medicines down, to avoid a life-threatening adrenal crisis; carrying a steroid emergency card is advised. Second, it commonly causes nervous-system effects such as drowsiness, confusion, unsteadiness and slurred speech, which can be a sign of too much in the blood, so levels are monitored. Third, it is harmful to a developing baby, so reliable contraception is essential during treatment and for a long time afterwards because it lingers in the body. Stomach upset is very common, and it can affect cholesterol, the thyroid and the liver, all of which are watched.

Who should not take it / use with caution

  • People who have had a serious allergic reaction to mitotane should not take it.
  • It must not be used in pregnancy, and it should be avoided when breastfeeding, because it is harmful to the baby.
  • It is used with particular caution in people with significant liver problems, under close specialist supervision.

Monitoring

  • Blood levels of mitotane to keep it in an effective but not toxic range.
  • Regular checks of cortisol need and steroid replacement, along with watching for adrenal crisis.
  • Monitoring of liver function, thyroid function, cholesterol and the nervous system.

Side effects

  • Nausea, vomiting, diarrhoea or loss of appetite, which are very common.
  • Drowsiness, confusion, unsteadiness or slurred speech, which can signal that the level is too high.
  • Signs of too little cortisol or an adrenal crisis, such as severe tiredness, dizziness, vomiting and feeling very unwell, which need urgent treatment.
  • Changes in cholesterol, thyroid function and liver blood tests, which are monitored.

Key interactions

  • It speeds up the breakdown of many other medicines, including some steroids, so doses often need adjusting.
  • The steroid replacement dose usually needs to be higher than normal because mitotane increases how fast it is cleared.
  • It can interact with blood thinners such as warfarin, so anticoagulation is monitored more closely.

Available as: Tablets taken by mouth.

Answers

Mitotane: frequently asked questions

What is mitotane used for?

It is a specialist medicine used to treat adrenocortical carcinoma, a rare cancer of the outer part of the adrenal gland.

Why do I also need to take a steroid?

Mitotane suppresses the body's own production of cortisol, so steroid replacement is given alongside to make up for it and prevent a dangerous shortage.

What are 'sick day rules'?

They are instructions to increase your steroid replacement during illness, injury or stress, and to seek urgent help if you cannot keep medicines down, to avoid a life-threatening adrenal crisis.

Why is contraception so important?

Mitotane is harmful to a developing baby and lingers in the body for a long time, so reliable contraception is needed during treatment and for a long time afterwards.

Why do I feel drowsy or unsteady?

Drowsiness, confusion, unsteadiness or slurred speech are common and can be a sign that the level in your blood is too high, so report them; your team monitors your blood levels.

Authoritative sources

  • BNF
  • NICE CKS

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