A targeted treatment for certain leukaemia and mast cell disorders
Midostaurin
A targeted treatment taken as capsules for FLT3-positive acute myeloid leukaemia and certain mast cell disorders.
What is Midostaurin?
Midostaurin is a specialist targeted cancer medicine used to treat a type of acute myeloid leukaemia (a blood cancer) that has a change in a gene called FLT3, and to treat some serious disorders of mast cells (a type of immune cell). It works by blocking faulty proteins that drive these cells to grow. It is taken by mouth as capsules, with food, and is usually given alongside an anti-sickness medicine because it commonly causes nausea and vomiting. It can affect the heart's rhythm (the QT interval) and the lungs, so these are monitored. It is harmful to a developing baby, so reliable contraception is important.
Education and reference only. This is a plain-language guide to Midostaurin — 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.
What it is
Midostaurin is a targeted cancer treatment used in two main situations: a type of acute myeloid leukaemia, a fast-developing blood cancer, that has a change in a gene called FLT3; and certain serious disorders in which the body makes too many mast cells, a kind of immune cell. In both, faulty signalling proteins drive the abnormal cells to grow, and midostaurin is designed to block them. It is taken by mouth as capsules, with food. In leukaemia it is usually combined with chemotherapy, while in mast cell disorders it is used on its own. It is prescribed and closely monitored by a specialist cancer or blood team.
How it works
Both FLT3-positive leukaemia and these mast cell disorders are driven by faulty proteins, called kinases, that send constant 'grow and survive' signals. Midostaurin blocks several of these proteins, including the faulty FLT3 and a related protein called KIT involved in mast cell disease, so the abnormal cells stop being pushed to multiply and many die off. It is taken with food, which helps the body absorb it. Because it acts on several proteins, it can affect normal processes too, which is part of why it is monitored carefully and often combined with supportive medicines.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist targeted cancer medicine used in the UK for a type of acute leukaemia with an FLT3 gene change, and for some serious mast cell disorders.
Practical use
How to take Midostaurin
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Take the capsules by mouth with food, swallowing them whole, twice a day as prescribed.
- Take your anti-sickness medicine as advised, since nausea and vomiting are common.
- Report any new or worsening breathlessness, cough or chest symptoms promptly, as these can signal lung problems.
- Tell your team about all your other medicines, as midostaurin interacts with several of them.
- Use reliable contraception during treatment and for the time your team advises, as it is harmful to a developing baby.
Weighing it up
Advantages & disadvantages of Midostaurin
Advantages
- Targets the faulty proteins driving FLT3-positive leukaemia and certain mast cell disorders.
- Taken by mouth as capsules rather than as an infusion.
- Can be combined with chemotherapy in leukaemia to improve treatment.
Disadvantages
- Commonly causes nausea and vomiting, usually needing an anti-sickness medicine.
- Can affect the heart's rhythm and the lungs, needing monitoring.
- Lowers infection-fighting blood cells and interacts with several other medicines.
Practical use
Good to know
A very practical point with midostaurin is that it commonly causes nausea and vomiting, so it is usually given together with an anti-sickness (antiemetic) medicine, and it is taken with food, which both helps absorption and eases the stomach. Two important safety areas are watched closely. It can affect the heart's electrical rhythm (the QT interval), so heart tracings and blood salts such as potassium are checked, and it can affect the lungs, so any new or worsening breathlessness or cough should be reported promptly as this can signal serious lung inflammation. Because it lowers the body's ability to fight infection and can affect blood counts, infections are taken seriously. It interacts with several other medicines, especially those that change how the liver handles it, so a full medicines list matters. It is harmful to a developing baby, so reliable contraception is needed.
Who should not take it / use with caution
- People who have had a serious allergic reaction to midostaurin should not take it.
- It is used with caution in people with certain heart-rhythm problems or those on medicines that affect the QT interval.
- It is not used in pregnancy because it is harmful to a developing baby, and reliable contraception is needed.
- It is used with care alongside medicines that strongly change how the liver handles it.
Monitoring
- Regular blood counts and checks for signs of infection or bleeding.
- Heart tracings and blood salts such as potassium to watch the QT interval.
- Watching for lung symptoms, and scans or tests to see how the disease is responding.
Side effects
- Nausea and vomiting, which is why an anti-sickness medicine is usually given.
- Tiredness, fever, diarrhoea, mouth soreness and headache.
- Lower blood counts, raising the risk of infection, anaemia and bleeding.
- Less commonly but importantly, changes in heart rhythm (the QT interval) or serious lung inflammation.
Key interactions
- Medicines that strongly change how the liver handles it can raise or lower its levels and effects.
- Medicines that affect the heart's QT interval should be reviewed when taking midostaurin.
- Give a full medicines list, including supplements, to your specialist team.
Available as: Capsules taken by mouth.
Answers
Midostaurin: frequently asked questions
What is midostaurin used for?
It is used to treat a type of acute myeloid leukaemia with an FLT3 gene change, and certain serious mast cell disorders, by blocking the faulty proteins that drive these cells to grow.
Why am I given an anti-sickness medicine with it?
Midostaurin commonly causes nausea and vomiting, so an anti-sickness (antiemetic) medicine is usually given alongside it, and it is taken with food.
Why does my team check my heart and lungs?
It can affect the heart's electrical rhythm (the QT interval) and can cause serious lung inflammation, so heart tracings are done and any breathing symptoms are reviewed promptly.
Should I take it with food?
Yes. Taking the capsules with food helps the body absorb the medicine and can also ease the stomach.
Do I need contraception?
Yes. Midostaurin is harmful to a developing baby, so reliable contraception is needed during treatment and for the time your team advises afterwards.
Authoritative sources
- BNF
- NICE CKS
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