What is ponatinib (Iclusig®)?

Ponatinib (Iclusig®) can be used to treat some types of leukaemia, such as chronic myeloid leukaemia (CML). It may sometimes be used to treat other cancers. It is best to read this information with our general information about targeted therapies and the type of leukaemia you have.

How ponatinib works

Ponatinib is a type of targeted therapy drug called a tyrosine kinase inhibitor (TKI). Kinases are proteins that control how cells in the body grow and divide. Ponatinib works by blocking (inhibiting) the protein (kinases) from sending signals to the leukaemia cells to grow. This can help to stop these cells from making more leukaemia cells.

When ponatinib is used

Your doctor will talk to you about this treatment and its possible side effects before you agree (consent) to have treatment.

Ponatinib is a treatment for people who have:

  • Chronic myeloid leukaemia (CML), where the leukaemia cells have a gene change (mutation) called T315I.
  • Acute lymphoblastic leukaemia (ALL), that is Philadelphia chromosome positive, where the leukaemia cells have a gene change (mutation) called T315I.
  • Either leukaemia mentioned above (CML or ALL), which does not have the T315I gene change and has already been treated with other tyrosine kinase inhibitor drugs.

Ponatinib may also be used to treat other types of cancer as part of a clinical trial.

Taking ponatinib tablets

You will take ponatinib tablets once a day. Take the tablets at the same time each day with or without food. Swallow the tablets whole with a large glass of water. They should not be chewed, dissolved, or crushed. If it is difficult to swallow your tablets, speak to your doctor or pharmacist.

It is important to take your drugs exactly as your nurse or pharmacist explained. This is to make sure they work as well as possible for you.

If you forget to take your ponatinib, check how much time there is until your next dose. If there is:

  • 12 hours or more, take it as soon as you remember.
  • Less than 12 hours, do not take it. Just take the next dose at the usual time.

You should never take a double dose.

There are some other important things to remember when taking ponatinib:

  • Do not eat grapefruit or drink grapefruit juice while you are taking ponatinib.
  • Keep the tablets in its original package, at room temperature, and away from heat and direct sunlight.
  • Keep the tablets safe and out of the reach of children.
  • Return any remaining tablets to the pharmacist if your treatment is stopped.

About side effects

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.

If you are also having treatment with other cancer drugs, you may 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.

More information

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.

Possible side effects

Risk of infection

If you have a low number of white blood cells, you are more likely to get an infection. If this happens, your doctor or nurse can advise you on how to lower your risk of infection. Your doctor may ask you to stop taking your tablets for a short time, until your white blood cell numbers recover. They may also ask you to take a lower dose of ponatinib.

Contact the hospital straight away 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.

Symptoms of an infection are:

  • feeling shaky or shivery
  • a sore throat
  • a cough
  • diarrhoea
  • needing to pass urine a lot.

It is important to follow any specific advice your cancer treatment team gives you.

Anaemia (low number of red blood cells)

Ponatinib can lower the number of red blood cells in your blood. These cells carry oxygen around the body. If the number of red blood cells is low, you may feel tired and out of breath. Tell your doctor or nurse if you feel like this. If you are very anaemic, you may need a drip to give you extra red blood cells. This is called a blood transfusion.

Bruising and bleeding

Ponatinib can lower the number of platelets in your blood. Platelets are cells that help the blood to clot.

This can sometimes cause bleeding problems, such as:

  • nosebleeds
  • bleeding gums
  • tiny red or purple (blood) spots or rashes on the skin
  • getting bruises more easily.

Tell your doctor if you are taking any medicines that may affect bleeding. If you are unsure whether your other medicines affect bleeding, tell your doctor about all of them.

Contact your doctor right away if you have any:

  • unusual bleeding including vomiting or coughing up blood
  • unexpected vaginal bleeding
  • blood in your poo (stools)
  • blood in your urine.

Feeling tired

Feeling tired is a common side effect. Try to pace yourself and plan your day so you have time to rest. Gentle exercise, like short walks, can give you more energy. If you feel sleepy, do not drive or operate machinery.

Feeling sick

Your doctor will prescribe anti-sickness (anti-emetic) drugs to help prevent or control sickness. Take the drugs exactly as your nurse or pharmacist explains to you. It’s easier to prevent sickness than to treat it after it has started.

If you still feel sick or are vomiting, contact the hospital as soon as possible. They can give you advice and change the anti-sickness drug to one that works better for you.


Ponatinib can cause diarrhoea. If you have diarrhoea, tell your doctor. They can give you anti-diarrhoea drugs that can help.

Make sure you drink at least 2 litres (3.5 pints) of fluids every day. It can help to avoid alcohol, caffeine, milk products, high-fat foods, and high-fibre foods.


This treatment can cause constipation. Here are some tips that may help:

  • Drink at least 2 litres (3½ pints) of fluids each day.
  • Eat high-fibre foods, such as fruit, vegetables and wholemeal bread.
  • Do regular gentle exercise, like going for short walks.

If you have constipation, contact the hospital for advice. Your doctor can give you drugs called laxatives to help.

Indigestion or tummy pain

Some people have indigestion or tummy pain (stomach or abdomen) when taking this treatment. If you have this, tell your doctor as they can give you drugs to help.

Let your doctor know straight away if you have any signs of:

  • bleeding from the back passage
  • black poo (stools)
  • you are vomiting up blood
  • your vomit looks like coffee grounds. 

Skin changes

Ponatinib may affect your skin. Your doctor or nurse can tell you what to expect. If your skin feels dry, try using an unperfumed moisturising cream every day. Ponatinib can cause a rash, which may be itchy. Always tell your doctor or nurse about any skin changes. They can give you advice and may give you creams or medicines to help.

Sometimes, ponatinib can cause severe skin reactions. Contact your doctor straight away if you have large blisters, peeling skin, or sores in your mouth.

Muscle, joint, or bone pain

You may have pain in your back, muscles, joints, or bones. Your doctor can give you painkillers to help. Some people may also have muscle spasms. If you have this, ask your doctor for advice.

Blood clots

Ponatinib can cause a blood clot. This can cause different symptoms depending on where the clot is. These may include:

  • pain, redness, and swelling in a leg or arm
  • breathlessness
  • chest pain
  • weakness down one side of your body
  • blurred vision or confusion.

Blood clots can be very serious. If you have any of these symptoms, it is important you, or someone with you, contacts a doctor straight away. Most clots can be treated with drugs that thin the blood. Your doctor or nurse can give you more information.

Narrowing of blood vessels

Ponatinib can make your blood vessels more narrow. This may increase the risk of having a stroke. It is important to tell your doctor if you have had any strokes or heart problems. Your doctor may talk to you about this and the benefits and risks of ponatinib. They may also talk to you about lifestyle changes, which can lower the risk of a stroke or heart problems. For example, they may advise you about following a healthy diet or smoking and how to stop.

If you feel weak or numb in one side of your body or find it hard to speak, contact a doctor immediately.

Effects on the heart

This treatment can affect the way the heart works. You may have tests to see how well your heart is working. These may be done before, during, and sometimes after treatment. If the treatment is causing heart problems, your doctor can change the dose of treatment you are having.

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.

High blood pressure (hypertension)

Your blood pressure may go up, so it should be checked regularly during your treatment. Let your doctor know if you have headaches, nosebleeds, or feel dizzy. They can give you tablets to control the high blood pressure. If you already take tablets for high blood pressure, tell your doctor before you start treatment.

Raised blood sugar levels

Ponatinib may raise your blood sugar levels. This may make you feel thirsty, tired, and make you pass urine more than usual. Tell your doctor or nurse if you have these symptoms.

Tell your doctor if you have diabetes as your blood sugar levels may be higher than usual. Your doctor will talk to you about how to manage this. They may change your insulin or tablet dose.

Effects on eyes

Your eyes may feel dry and sore, or get red and inflamed. Your doctor can give you eye drops to help with this.

If you have blurred vision or have any changes to your eyesight, contact your doctor straight away.


Ponatinib can cause headaches. If you have headaches, tell your doctor or nurse. They can give you painkillers to help.

Build up of fluid (oedema)

Sometimes, fluid can build-up in your legs and ankles, which can cause swelling. This is known as oedema. Always tell your doctor if you feel breathless or see any swelling in your legs or arms. A build-up of fluid may be treated with drugs (diuretics) that make you pass more urine.


Ponatinib can make you dehydrated. Try to drink around 2 litres (3.5 pints) of fluids every day while having treatment.

Tell your doctor or nurse if you have signs of dehydration, such as:

  • feeling thirsty
  • having dark or strong-smelling urine
  • dizziness
  • dry mouth and eyes
  • passing little urine less than 4 times a day.

Sore mouth

You may get a sore mouth or mouth ulcers. This can make you more likely to get a mouth infection.

Use a soft toothbrush to clean your teeth or dentures in the morning, at night and after meals.

If your mouth is sore:

  • tell your nurse or doctor – they can give you a mouthwash or medicines to help
  • try to drink plenty of fluids
  • avoid alcohol, tobacco, and foods that irritate your mouth.

Less common side effects

Changes in the way the liver and kidney works

Ponatinib may affect how your liver and kidneys work. Your doctor or nurse will take regular blood samples to check your kidneys and liver are working properly. If the treatment is affecting your liver, your doctor may change the dose of treatment you are having.

Hair loss

Ponatinib may cause your hair to thin. Talk to your nurse if you are worried about this.

Numb or tingling hands or feet (peripheral neuropathy)

Ponatinib can affect the nerves, which can cause numb, tingling or painful hands. This is called peripheral neuropathy. You may also find it hard to fasten buttons or do other fiddly tasks. Tell your doctor if you have these symptoms.

Thyroid changes

Ponatinib can affect the thyroid gland. You will have regular blood tests to check if the treatment is affecting your thyroid.

Swollen pancreas (pancreatitis)

Ponatinib can make your pancreas swell (pancreatitis). Tell your doctor straight away if you get a sharp pain in your upper tummy area (abdomen) with sickness and vomiting. They may change how much ponatinib you take and can give you medicines that can help.

Hepatitis B reactivation

If you had Hepatitis B (a liver infection) in the past, ponatinib can make it active again. Your doctor or nurse will talk to you about this and may test you for Hepatitis B before, during, and after treatment.

Tumour lysis syndrome (TLS)

Rarely, ponatinib may cause the cancer cells to break down very quickly. This releases uric acid (a waste product) into the blood. The kidneys usually get rid of uric acid but may not be able to cope with large amounts. Too much uric acid can affect the heart and kidneys. Doctors call this tumour lysis syndrome (TLS).

Your doctor may give you drugs to lower the risk of TLS. You may also be given fluid through a drip to help protect your kidneys.

Other information


Do not drive if you have dizziness or blurred vision, or if you feel tired or sleepy. Talk to your doctor if you are not sure whether it is safe for you to drive.

Other medicines

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.


Ponatinib contains a small amount of lactose. If you have a lactose intolerance, talk to your doctor before you start taking this.


Some cancer drugs can affect whether you can get pregnant or make someone pregnant. There may be ways to preserve fertility for men and women. If you are worried about fertility, it is important to talk with your doctor before you start treatment.

Erectile dysfunction

Some men may have difficulty getting or maintaining an erection while taking ponatinib. Talk with your doctor or nurse if you are worried about this.


Your doctor will advise you not to become pregnant or father a child while taking ponatinib. This is because it may harm a developing baby. It is important to use effective contraception.

Ponatinib may make the contraceptive pill less effective. Women are advised to use another type of contraception along with the contraceptive pill. Talk to your doctor if you need more information about this.


Women are advised not to breastfeed while having this treatment. This is because the drugs could be passed to the baby through breast milk.

Medical and dental treatment

If you need medical treatment for any reason other than cancer, always tell the doctors and nurses that 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.

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