A targeted tablet for FLT3-mutated acute myeloid leukaemia
Gilteritinib
A targeted tablet used to treat acute myeloid leukaemia that carries an FLT3 gene change.
What is Gilteritinib?
Gilteritinib is a specialist targeted cancer medicine used to treat acute myeloid leukaemia (a cancer of the blood and bone marrow) in people whose leukaemia has a particular gene change called an FLT3 mutation. It is taken by mouth as a tablet under the care of a blood-cancer team. Important safety issues include a serious reaction called differentiation syndrome, effects on the heart's rhythm (the QT interval), a rare brain condition called PRES, inflammation of the pancreas and changes in liver blood tests. It can harm an unborn baby, so reliable contraception is essential.
Education and reference only. This is a plain-language guide to Gilteritinib — 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
Gilteritinib is a targeted treatment for acute myeloid leukaemia, a fast-developing cancer of the blood and bone marrow. It is used when the leukaemia carries a specific gene change known as an FLT3 mutation, which a laboratory test confirms. By targeting this change, it works differently from standard chemotherapy. It is taken by mouth as a tablet, usually once treatment has been planned by a specialist blood-cancer team, and it is generally taken continuously while it keeps working and is tolerated.
How it works
In FLT3-mutated leukaemia, a faulty signal drives the cancer cells to grow and survive. Gilteritinib blocks the FLT3 protein that carries this signal, switching off the growth message so the leukaemia cells are less able to multiply and more likely to die. Because it targets the specific change driving the cancer, it is used in people whose leukaemia has been shown to have an FLT3 mutation. It is taken every day so that the blocking effect is maintained over time.
Company & origin
Originated / developed by: Specialist manufacturer.
A specialist cancer medicine used in the UK to treat acute myeloid leukaemia that has a particular gene change called an FLT3 mutation.
Practical use
How to take Gilteritinib
General, dose-free guidance — always follow your prescriber's and the leaflet's specific instructions.
- Take the tablets by mouth as your blood-cancer team prescribes, swallowed whole and at about the same time each day.
- Report straight away any fever, breathlessness, swelling or sudden weight gain, as these can signal differentiation syndrome.
- Attend appointments for heart tracings and blood tests to check your heart rhythm, blood salts, liver and pancreas.
- Use reliable contraception during treatment and for the advised time afterwards, as it can harm an unborn baby.
- Tell your team about any other medicines, as some interact with gilteritinib.
Weighing it up
Advantages & disadvantages of Gilteritinib
Advantages
- Targets the specific FLT3 gene change driving the leukaemia, offering a tailored treatment.
- Taken by mouth as a tablet rather than given by infusion.
- Provides a treatment option for a hard-to-treat form of acute myeloid leukaemia.
Disadvantages
- Can cause differentiation syndrome, a serious reaction needing prompt treatment.
- Can affect the heart's rhythm and, rarely, cause a serious brain condition (PRES) or pancreas inflammation.
- Can harm an unborn baby, so reliable contraception is essential.
Practical use
Good to know
An important early effect to watch for is differentiation syndrome, where large numbers of leukaemia cells mature at once and cause symptoms such as fever, breathlessness, swelling and weight gain; this needs prompt treatment, so any such symptoms should be reported straight away. Gilteritinib can also affect the heart's electrical rhythm (the QT interval), so heart tracings and blood salts are checked. A rare but serious brain condition called PRES, causing headache, confusion, vision changes or seizures, can occur and needs urgent attention. The pancreas can become inflamed, and liver blood tests can change, so these are monitored. It can seriously harm an unborn baby, so effective contraception is needed during and for a time after treatment. Regular blood tests track both the leukaemia and side effects.
Who should not take it / use with caution
- People who have had a serious allergic reaction to gilteritinib should not take it.
- It should not be used in pregnancy because it can harm an unborn baby.
- It is used with caution in people with heart-rhythm problems or those on other medicines that affect the QT interval.
Monitoring
- Regular blood tests to track the leukaemia, liver function and pancreas.
- Heart tracings and blood-salt checks to watch the heart's rhythm.
- Watching for symptoms of differentiation syndrome or the brain condition PRES.
Side effects
- Tiredness, muscle or joint aches, and changes in blood counts seen on blood tests.
- Changes in liver blood tests and, sometimes, inflammation of the pancreas.
- Less commonly but seriously, differentiation syndrome, heart-rhythm changes or the brain condition PRES.
Key interactions
- Some medicines change the levels of gilteritinib, so a full medicines list is important.
- Medicines that affect the heart's QT interval are reviewed when taking gilteritinib.
- Certain other cancer-related treatments may reduce its effect, so combinations are checked by the team.
Available as: Tablets taken by mouth.
Answers
Gilteritinib: frequently asked questions
What is gilteritinib used for?
It is used to treat acute myeloid leukaemia in people whose leukaemia carries a particular gene change called an FLT3 mutation, confirmed by a laboratory test.
What is differentiation syndrome?
It is a serious reaction in which many leukaemia cells mature at once, causing fever, breathlessness, swelling and weight gain; it needs prompt treatment, so report such symptoms straight away.
Does it affect the heart?
It can affect the heart's electrical rhythm (the QT interval), so heart tracings and blood salts are checked, and other medicines that do the same are reviewed.
Can I take it if I might become pregnant?
Gilteritinib can seriously harm an unborn baby, so reliable contraception is needed during treatment and for the advised time afterwards.
Why do I need regular blood tests?
Blood tests track how the leukaemia is responding and watch for side effects on the liver, pancreas and blood counts.
Authoritative sources
- BNF
- NICE CKS
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