Treatment for chronic myeloid leukaemia (CML)

If you have high white blood cell counts or worrying symptoms, your doctors might start treatment before all your test results come back. This is likely to be a course of tablets called hydroxycarbamide (also known as hydroxyurea). This is a type of chemotherapy that helps lower your white blood cell count.

We have separate information about hydroxycarbamide.

Once your blood counts improve, your haematology team usually reduce the dose before stopping it completely.

The treatment you have depends on the phase of your CML.

• If your CML is in the chronic phase, the usual treatment is a type of targeted therapy called a TKI tyrosine kinase inhibitor (TKI).
• If your CML is in the blast phase, your doctors might suggest adding chemotherapy to your TKI. They could advise a stem cell transplant as well.

Depending on your circumstances, your haematology team might suggest other treatments. They will explain what they recommend for you.

TKIs

Tyrosine kinase inhibitors (TKIs) are a type of targeted therapy. They block an abnormal protein called BCR-ABL, which is a type of tyrosine kinase. This abnormal protein is present in people with CML. It encourages the blood-forming cells in your bone marrow to make too many white blood cells. Blocking the protein helps stop the leukaemia cells from multiplying.

TKIs are tablets that you take by mouth. They are generally highly effective at keeping CML under control. Each TKI has a different profile and might cause different side effects. We have separate information on each of the TKIs used to treat CML:

• Imatinib
• Nilotinib
• Dasatinib
• Bosutinib
• Ponatinib
• Asciminib

Your doctors will suggest the most suitable TKI for you based on:

• Your personal characteristics such as your age, any other medical conditions you have, and your treatment preference
• Features of your CML, like its phase and the exact genetic changes in your leukaemia cells
• How you’ve responded to any previous treatment you have had

Chemotherapy

You might be offered chemotherapy as well as a TKI if your CML is in the blast phase. Different combinations of chemotherapy medicines may be used. Your haematology team will tell you what they recommend. They will explain what the treatment is, how you have it, and what side effects that you might get.

• Macmillan Cancer Support have more information about particular chemotherapy combinations.

Rarely, some people have leukaemia cells in the central nervous system. If this is the case, you may also need a course of chemotherapy directly into the fluid surrounding your brain and spinal cord. This is called intrathecal chemotherapy.

Stem cell transplant

A stem cell transplant involves having high-dose chemotherapy to kill blood-forming stem cells in your bone marrow. These are then replaced by healthy stem cells from a matched donor.

A stem cell transplant is very intensive. It is only suitable for people who are fit enough to have it. Your haematology team might suggest a stem cell transplant if your CML is in the blast phase. They might also consider it if you’ve already had treatment with several different TKIs.

Your haematology team will let you know early on if a stem cell transplant is an option for you. They will discuss it with you and explain its benefits and risks.

We have separate information about stem cell transplants.

Supportive care

During your treatment and afterwards, you might also need medicine to prevent or treat symptoms or side effects. This is called supportive care. It does not treat your CML itself, but it helps you feel better.

As well as medicines to help you, supportive care can include:

• Psychological support
• Support with exercise or physiotherapy
• Social support
• Spiritual wellbeing such as mindfulness

Your haematology team should talk to you to find out what support they can offer you.

If your treatment is not successful, your haematology team will suggest changing to a different treatment. This may happen if:

• Your CML does not respond to the treatment
• Your CML responds to the treatment at first but then stops responding
• You have side effects that are difficult to manage

If your treatment is not successful, your haematology team will suggest the most suitable next treatment based on your individual circumstances. For most people, the usual second treatment is a different TKI. If second-line TKI treatment is not successful, the usual next treatment is another TKI.

TKIs all work in slightly different ways and have different side effects. Some are effective against particular gene changes. If one TKI is not successful, a different TKI may be.

TKIs are effective for most people, but if they are not suitable for you, your haematology team will discuss other treatment options This could include a stem cell transplant or supportive care.

We have more information about second- and later-line treatment for CML.