How is CML treated?

Chemotherapy

The aim of treatment is to put the CML into remission. 

Remission is when your bone marrow is producing blood cells normally and you have fewer than 5% of the immature leukaemia cells in your bone marrow. The leukaemia cells can no longer be detected in your body and you feel well. If your remission lasts indefinitely, you are said to be cured.

Treatment for different phases of CML

Chronic phase

Treatment with a targeted therapy drug called a TKI (tyrosine kinase inhibitor) is usually very effective at controlling the disease in the chronic phase. Once the disease is under control, most people can live a normal life. You will keep taking TKIs and have regular blood tests to check how well you are responding to treatment.

Accelerated phase

Your doctor may suggest different TKIs or chemotherapy, depending on your previous treatment, blood results, symptoms and overall health.

Blast (acute) phase

You may be given TKIs or high-dose chemotherapy to reduce the high number of blast cells in your bone marrow. The aim of treatment is to get the disease stabilised so that it can be controlled better. In a few cases a stem cell transplant may be recommended.

Types of treatment

Targeted therapies

Drugs that target genetic changes. Nearly everyone with CML will be treated with targeted therapies called TKIs. If your CML doesn’t respond to one type of TKI, you will usually be given a different type. 

You will stay on TKIs permanently as long as they are working to control your CML, even if your blood tests are normal and you feel well. If you stop taking TKIs the effects of the CML may return.

TKIs can damage an unborn baby. Talk to your doctor about your options before treatment starts if you were planning to have children in the future.

Chemotherapy

Chemotherapy is when you use drugs to kill cancer cells. Chemotherapy may be used:

  • If TKIs are not effective or not suitable
  • To control symptoms when you are first diagnosed
  • To try to stabilise blast-phase CML
  • As a high-dose treatment before a transplant

Chemotherapy for CML is often given in tablet form. For CML in the accelerated or blast phase you may have a combination of chemotherapy drugs, usually through a drip.

Stem cell transplants

This is where all the blood cells in your bone marrow are destroyed and replaced with healthy stem cells. These healthy stem cells should grow into healthy blood cells. Most people’s CML is well controlled by drug treatments. Your doctor may recommend a transplant if your CML doesn’t respond well to the various TKIs or becomes resistant to them. 

A transplant may be suggested at any phase of CML. 

Stem cell transplants may not be suitable for everyone. 

Will I get side-effects?

The type of side-effects you get will depend on the type of treatment, the dose, the duration and your own general health. A lot of treatments cause fatigue. Read more about coping with side-effects and symptoms. 

How will I know if my treatment is working?

You will have blood tests to see how the cancer is responding to treatment. 

Complete haematologic response (CHR)

Your full blood count is normal and no leukaemia (blast) cells can be detected in the blood. Your spleen is a normal size.

Partial haematologic response

There has been some improvement in your blood count, but there are still signs or symptoms of CML. The spleen may be enlarged. Most people get a haematological response within 3 months of starting treatment with TKIs.

Testing for the Philadelphia chromosome (cytogenetic response)

This is a more sensitive test that measures the amount of cells in your bone marrow that have the Philadelphia chromosome (Ph+ cells). If no Ph+ cells are found it is called a complete cytogenetic response.

Presence of the abnormal BCR-ABL gene (molecular response)

A molecular response is based on the results of the PCR test, which looks for the BCR-ABL gene in the blood or bone marrow. This is a very sensitive test that can detect one leukaemia cell in up to 10,000 normal blood cells.

  • Complete molecular response (CMR): The PCR test cannot detect any BCR-ABL gene in your blood.
  • Major molecular response (MMR): A tiny amount of the BCR-ABL gene is found in your blood.

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