Treatment options for chronic myeloid leukaemia (CML)

A team of health professionals decides what treatment options you have. The most common treatments for chronic myeloid leukaemia (CML) are targeted cancer drugs. These drugs are called tyrosine kinase inhibitors (TKIs).

Deciding which treatment you need

A team of doctors, and other professionals discuss the best treatment and care for you. They are the multidisciplinary team (MDT). Your MDT might include:

• a haematologist - a doctor specialising in blood cancers
• a haemato-pathologist – a doctor who examines bone marrow or lymph node biopsies
• a radiologist – a doctor specialising in reporting x-rays and scans
• a specialist haematology nurse – also called a clinical nurse specialist (CNS)
• a palliative care doctor - a doctor specialising in controlling cancer symptoms

The MDT look at a number of different factors including: 

• the phase of your CML
• whether you have other health conditions
• your age and level of fitness
• the side effects of the different drugs
• the results of genetic tests on your CML cells looking for abnormal chromosomes
• your personal situation and preference

Your doctor will talk to you about your treatment options. They will discuss the benefits and the possible side effects with you.

The aim of treatment is to put your CML into remission. Remission means there are no signs of CML in your blood. The doctors refer to remission as ‘response to treatment’.

The main treatments for CML

The main treatment for CML are targeted cancer drugs. Other possible treatments include chemotherapy and a stem cell transplant.

Targeted cancer drugs

Targeted cancer drugs can change the way that cells work and help the body control the growth of cancer. There are different types of targeted cancer drugs. The main type for CML are:

• tyrosine kinase inhibitors (TKIs)

Examples of TKI drugs for CML are imatinib, bosutinib, dasatinib and nilotinib

Chemotherapy

Chemotherapy uses anti cancer (cytotoxic) drugs to destroy cancer cells. The drugs circulate throughout the body in your bloodstream.

Fludarabine, idarubicin and cytarabine are examples of chemotherapy drugs for CML.

You usually only have chemotherapy if you have more advanced stages of CML.

Stem cell transplant

You have a stem cell transplant after very high doses of chemotherapy. The chemotherapy kills the cancer cells and also the stem cells in your bone marrow. 

After the chemotherapy you have the new stem cells into your bloodstream through a drip. You usually have a stem cell transplant using stem cells from a donor. 

Treatment by phase

There are 3 phases (stages) of CML:

• chronic phase
• accelerated phase
• blast phase

Chronic phase

Most people are diagnosed in the chronic phase. The main aim of treatment is to control your CML and get you into remission. Remission means there's no sign of active leukaemia in your body. And you don’t have any symptoms. This remission can last for years.

The most common treatment is a type of targeted cancer drug called a tyrosine kinase inhibitor (TKI).

There are several different TKIs. The most common drug for CML is imatinib (Glivec). But your doctor might suggest a different TKI such as dasatanib (Sprycel) or nilotinib (Tasigna).

Most people respond well to this treatment. CML can stay under control for many years.

Your doctor might suggest you try a different TKI if:

• tests to look for signs of CML show that your CML hasn’t gone away
• tests detect signs of CML again after a period of time
• you have severe side effects from your current treatment

Occasionally TKI treatment does not work. In this situation your doctor might offer you chemotherapy and a stem cell transplant. 

Accelerated and blast phase

You might:

• progress to the accelerated or blast phase after having treatment for chronic phase CML
• be diagnosed in the accelerated or blast phase, although this is less common

Treatment for the accelerated or blast phase depends on whether you have already had treatment. And if so, what treatment you had.

Accelerated phase

Treatment aims to get you back into remission.

Imatinib is normally the first choice unless you have already had it in the chronic phase.

Or you might have treatment with another TKI. There are several available including:

• nilotinib (Tasigna)
• dasatinib (Sprycel)
• bosutinib (Bosulif)
• ponatinib (Iclusig)
• asciminib (Scemblix)

Your doctor might recommend a stem cell transplant if the TKI treatment doesn’t work. This is more likely if you:

• have progressed to accelerated phase from chronic phase
• are younger and fairly fit and well 

Blast phase

Your doctor might suggest:

• a combination of chemotherapy drugs (fludarabine, cytarabine and idarubicin) – you have this on its own, or together with a TKI
• a TKI on its own

Your doctor usually recommends you have a stem cell transplant if you are well enough and have a stem cell donor.

You might have also treatment to relieve symptoms.

First and second line treatments

You might hear your doctor refer to your treatment as first line or second line.

First line treatment

This is the first treatment you have when you are diagnosed with CML. The most common first line treatment for CML is a TKI drug.

Second and third line treatments

Some people have to change on to a different treatment. The second treatment you have is called second line treatment. And then the next treatment is called third line treatment, and so on.

The aim of second line treatment is to put your CML back into remission. Remission means there are no signs of CML in your blood.

Supportive treatments

You might have symptoms or problems caused by the leukaemia. Supportive treatments can help to either prevent or control these problems.  

There are several other treatments you might have for CML:

• A chemotherapy drug called hydroxycarbamide. This is to lower your white blood or platelet levels. You might have this whilst you wait for your diagnosis.
• Fluids (a drip) into your vein. This is to prevent complications caused by breaking down the leukaemia cells (tumour lysis syndrome).
• Treatment to prevent or treat infections.
• Blood products for low blood cell counts.

Monitoring your response to treatment

To begin with, you need to see your doctor regularly, maybe weekly or monthly. You usually have a blood test at each visit. Your doctor might examine you and asks you how you are.  This is to see whether you have any problems with the treatment.

The time between check ups will gradually get longer if everything is going well.

The aim of treatment is to put your CML into remission. Remission means there are no signs of CML in your blood.  The doctors refer to remission as ‘response to treatment’.

There are different tests that look for signs of CML. The test results provide information about how well the treatment is working to control your leukaemia. To monitor your response to treatment, you have blood tests every 3 to 6 months depending on your situation. You might also have bone marrow tests.

Having a break in treatment

Doctors know that it is safe for some people to stop treatment if their CML is under control. This is called a treatment break. Or your doctor might call it treatment free remission.

Your doctor can talk with you about whether a treatment break might be an option for you.

Clinical trials

Your doctor might ask if you’d like to take part in a clinical trial. Doctors and researchers do trials to make existing treatments better and develop new treatments.