Midostaurin (Rydapt®) is used to treat acute myeloid leukaemia (AML). It is used if tests show that the leukaemia cells have a gene change called an FLT3 mutation. Sometimes, midostaurin is used to treat other conditions. It is best to read this information with our general information about the type of cancer you have.
Midostaurin is a type of targeted therapy drug called a cancer growth inhibitor. Your doctor will talk to you about this treatment and its possible side effects before you agree (consent) to have treatment.
You may have midostaurin capsules as part of your first treatment for AML. You take it for 14 days between cycles of chemotherapy. Midostaurin can also be given after this on its own for up to a year.
During treatment you will see a leukaemia doctor (a haematologist), a cancer nurse or specialist nurse, and a specialist pharmacist. This is who we mean when we mention doctor, nurse or pharmacist in this information.
Your doctor, nurse or pharmacist will discuss your treatment plan with you. They will explain when and how long you should take midostaurin for. They may give you a copy of your treatment plan to keep. They will give you the capsules to take home if you are having treatment as an outpatient.
Always take the capsules exactly as explained. This is important to make sure they work as well as possible for you.
Your nurse or pharmacist may also give you anti-sickness drugs and other medicines to take home. Take all your capsules or tablets exactly as they have been explained to you.
Taking midostaurin capsules
You should take midostaurin capsules with food. Swallow them whole with a glass of water. Do not chew, open or crush them. Take them at the same times every day.
If you forget to take the capsules, take the next dose at the usual time. Do not take a double dose.
Other things to remember about your capsules:
- Keep them in the original package.
- Keep them safe and out of sight and reach of children.
- If you are sick just after taking the capsules, contact the hospital. Do not take another dose.
- If your treatment is stopped, return any unused capsules to the pharmacist.
We explain the most common side effects of this treatment here. We also include some less common side effects. You may get some of the side effects we mention, but you are unlikely to get all of them.
You may also have some side effects that we have not listed here. Always tell your doctor, nurse or pharmacist about any side effects you have.
Your doctor can give you drugs to help control some side effects. It is important to take them exactly as your nurse or pharmacist explains. This means they will be more likely to work for you. Your nurse will give you advice about managing your side effects. After your treatment is over, most side effects start to improve.
Serious and life-threatening side effects
Some cancer treatments can cause severe side effects. Rarely, these may be life-threatening. Your cancer doctor or nurse can explain the risk of these side effects to you.
Contact the hospital
Your nurse will give you telephone numbers for the hospital. If you feel unwell or need advice, you can call them at any time of the day or night. Save these numbers in your phone or keep them somewhere safe.
We cannot list every side effect for this treatment. There are some rare side effects that are not listed. You can visit the electronic Medicines Compendium (eMC) for more detailed information.
Risk of infection
This treatment can reduce the number of white blood cells in your blood. These cells fight infection. If the number of white blood cells is low, you are more likely to get an infection. A low white blood cell count is sometimes called neutropenia.
An infection can be very serious when the number of white blood cells is low. It is important to get any infection treated as soon as possible. Contact the hospital straight away on the 24-hour contact number you have been given if:
- your temperature goes over 37.5°C (99.5°F)
- you suddenly feel unwell, even with a normal temperature
- you have symptoms of an infection
- your temperature goes below 36°C (96.8°F).
Symptoms of an infection include:
- feeling shivery and shaking
- a sore throat
- a cough
- needing to pass urine (pee) a lot, or discomfort when you pass urine.
It is important to follow any specific advice your cancer treatment team gives you.
The number of white blood cells will usually return to normal before your next treatment. You will have a blood test before having more treatment. If your white blood cell count is low, your doctor may delay your treatment for a short time, until your cell count increases.
Bruising and bleeding
Midostaurin may reduce the number of platelets in your blood. This is more common if you have chemotherapy at the same time. Platelets are cells that help the blood to clot. Tell your doctor if you have any bruising or bleeding that you cannot explain. This includes:
- bleeding gums
- tiny red or purple spots on the skin that may look like a rash.
You may need a drip to give you extra platelets. This is called a platelet transfusion.
Anaemia (low number of red blood cells)
Effects on the heart
This treatment can affect the way your heart works. Your doctor may do tests to see how well your heart is working. You may have these tests before, during, and sometimes after treatment.
Contact a doctor straight away if you:
- have pain or tightness in your chest
- feel breathless or dizzy
- feel your heart is beating too fast or too slowly.
Other conditions can cause these symptoms. But it is important to get them checked by a doctor.
You may feel sick during treatment. Your doctor will give you anti-sickness drugs to help prevent or control sickness. Take the drugs exactly as your nurse or pharmacist tells you. It is easier to prevent sickness than to treat it after it has started.
If you feel sick, take small sips of fluids and eat small amounts often. Contact the hospital as soon as possible if you:
- continue to feel sick
- vomit more than once in 24 hours.
The hospital will give you advice and may change the anti-sickness drug to one that works better for you.
This treatment may cause diarrhoea. Diarrhoea means passing more stools (poo) than is usual for you, or having watery or loose stools. If you have a stoma, it will be more active than usual.
If you have diarrhoea:
- try to drink at least 2 litres (3½ pints) of fluids each day
- avoid alcohol, caffeine, milk products, high-fat foods and high-fibre foods
- contact the hospital for advice.
Raised blood sugar levels
This treatment may raise your blood sugar levels. You will have regular blood tests to check this. Symptoms of raised blood sugar include:
- feeling thirsty
- needing to pass urine (pee) more often
- feeling tired.
Tell your doctor or nurse if you have these symptoms during treatment or after it finishes.
If you have diabetes, your blood sugar levels may be higher than usual. Your doctor will talk to you about how to manage this.
Effects on the liver
This treatment may affect how your liver works. This is usually mild and goes back to normal after treatment. You will have regular blood tests to check how well your liver is working.
This treatment may affect your skin. Your doctor or nurse can tell you what to expect. If you have this treatment with chemotherapy, skin reactions can sometimes be serious.
Always tell your doctor or nurse about any skin changes including rashes or itching. They can give you advice and may give you creams or medicines to help.
This treatment may cause headaches. If you have headaches, tell your doctor. They may give you painkillers to help.
Build-up of fluid
This treatment may cause your ankles and legs to swell. This is due to fluid building up. Tell your doctor or nurse if this happens, as there are medicines that can help. If the swelling is uncomfortable, they may give you support stockings to wear. The swelling gets better after your treatment ends.
Effects on the lungs
This treatment can cause changes to the lungs. Tell your doctor if you develop:
- a cough
You should also tell them if any existing breathing problems get worse. You may have tests to check your lungs.
Blood clot risk
Cancer and some cancer treatments can increase the risk of a blood clot. Symptoms of a blood clot include:
- throbbing pain, redness or swelling in a leg or arm
- suddenly feeling breathless or coughing
- sharp chest pain, which may be worse when you cough or take a deep breath.
If you have any of these symptoms, contact the hospital straight away on the 24-hour contact number you have been given. If you cannot get through to your doctor, call the NHS urgent advice number on 111.
A blood clot is serious, but it can be treated with drugs that thin the blood (anticoagulants). Your doctor or nurse can give you more information.
You can help reduce the risk of developing a blood clot by:
- staying active during treatment
- drinking plenty of fluids, especially water.
You may be given anticoagulants to help prevent a clot.
Some medicines can affect how this treatment works or be harmful when you are having it. Always tell your cancer doctor about any drugs you are taking or planning to take, such as:
- medicines you have been prescribed
- medicines you buy in a shop or chemist
- vitamins, herbal drugs and complementary therapies.
Tell other doctors, pharmacists or dentists who prescribe or give you medicines that you are having this cancer treatment.
You can visit the electronic Medicines Compendium (eMC) for more detailed information about your treatment.
Vaccinations can reduce your risk of getting certain infections. Your doctor or nurse may talk to you about having vaccinations.
Doctors usually recommend that people with cancer have a flu vaccination and a coronavirus vaccination. These are both inactivated vaccinations that can help reduce the risk of infection. People with weak immune systems can have these, as they are not live vaccinations.
If your immune system is weak, you need to avoid live vaccinations. This is because they can make you unwell. Live vaccines, such as shingles, contain a very weak version of the illness they are vaccinating you against. Your cancer doctor or GP can tell you more about live and inactivated vaccinations.
Your doctor will advise you not to get pregnant or make someone pregnant while having this treatment. The drugs may harm a developing baby. It is important to use contraception during your treatment and for a while after treatment finishes. Your doctor, nurse or pharmacist can tell you more about this.
You are advised not to breastfeed while having this treatment, or for some time after treatment finishes. This is because the drugs could be passed to the baby through breast milk.
Your doctor or nurse can give you more information.
Medical and dental treatment
If you need medical treatment for any reason other than cancer, always tell the doctors and nurses you are having cancer treatment. Give them the contact details for your cancer doctor so they can ask for advice.
If you think you need dental treatment, talk to your cancer doctor or nurse. Always tell your dentist you are having cancer treatment.
This information has been written, revised and edited by Macmillan Cancer Support’s Cancer Information Development team. It has been reviewed by expert health professionals and people living with cancer.
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