Chemotherapy for acute myeloid leukaemia (AML)

Chemotherapy is the main treatment for acute myeloid leukaemia (AML). It uses anti cancer (cytotoxic) drugs to destroy cancer cells. The drugs circulate throughout the body in the bloodstream.

Treatment for AML is generally divided into intensive and non intensive treatment.

Intensive treatment

Intensive treatment aims to cure your AML. As this treatment is more intense, it can be quite demanding. You have this treatment if your doctor thinks you are fit and well enough to cope with the side effects.

You usually stay in hospital while you are having this treatment.

Non intensive treatment

Non intensive treatment aims to control your leukaemia for as long as possible.

Non intensive means that you usually have lower doses of chemotherapy. These generally cause fewer and less severe side effects. You might have this treatment if you are not that fit and well, or you have other health conditions. For example, heart or lung problems.

You usually have non intensive treatment as an outpatient. But you will visit hospital regularly to see your healthcare team. You may stay in hospital if you have symptoms or side effects that need closer monitoring and treatment.

Intensive treatment for AML

The main intensive treatment for AML is chemotherapy. Other treatments you might have include:

  • targeted cancer drugs

  • growth factors

  • stem cell or bone marrow transplants

  • radiotherapy

Intensive treatment is split into different phases of treatment. The phases are:

  • remission induction

  • consolidation

  • maintenance

Remission induction

The aim of the induction phase is to destroy as many leukaemia cells as possible. You start treatment quite quickly after your diagnosis. You have several chemotherapy drugs over a few days.

You usually stay on the ward for about 4 to 6 weeks. 

There are a number of different induction chemotherapy combinations. These include:

  • cytarabine and daunorubicin (DA)

  • liposomal cytarabine and daunorubicin (also known as CPX351, Vyxeos, L-DA)

  • fludarabine, cytarabine and G-CSF (FLAG)

  • fludarabine, cytarabine, G-CSF and idarubicin (FLAG-Ida)

  • cytarabine, daunorubicin and etoposide (ADE)

  • amsacrine, cytarabine and etoposide (MACE)

  • mitoxantrone and cytarabine (MiDAC)

We have information about each of these drugs on our cancer drugs A to Z list.

You may also have a targeted cancer drug with your chemotherapy. You have tests on your leukaemia cells to find out if you can have these drugs. The tests look for certain proteins or gene changes. You might have:

  • midostaurin OR

  • gemtuzumab ozogamicin (Mylotarg)

After treatment, you have a bone marrow test to find out how well treatment is working. This looks at the number of leukaemia cells left behind in your bone marrow after treatment. This is called measurable residual disease (MRD).

MRD is a very sensitive test that helps your doctor work out whether your leukaemia is likely to come back. This helps them to plan future treatment.

There are different levels of response. The following is a simplified guide:

Complete remission (CR) means:

  • there is no sign of leukaemia in your bone marrow

  • your blood count has returned to normal

Partial response (PR) means that there are still some leukaemia cells in your bone marrow. But there are fewer leukaemia cells and your disease has partly responded to your treatment.

In this situation you may have more than one cycle of induction treatment to get your leukaemia into complete remission. 

Refractory AML means that the treatment isn’t working. Your doctor or nurse will look at different treatment options. They can discuss this more with you.

Consolidation

When there is no sign of the leukaemia, it is called remission. You then have consolidation treatment to lower the risk of it coming back.

Consolidation treatment might include chemotherapy and targeted cancer drugs. Or you might have chemotherapy and then a stem cell transplant Open a glossary item

You normally have a combination of chemotherapy drugs for this phase. Some of the chemotherapy treatments used in this phase include:

  • intermediate dose cytarabine (IDAC)

  • high dose cytarabine (HIDAC)

  • cytarabine and daunorubicin (DA)

  • liposomal cytarabine and daunorubicin (also known as CPX351, Vyxeos, L-DA)

  • amsacrine, cytarabine and etoposide (MACE)

  • mitoxantrone and cytarabine (MiDAC)

You usually have 3 to 4 cycles of consolidation treatment. You can find information about each of these drugs on our cancer drugs A to Z list.

Chemotherapy before a stem cell or bone marrow transplant

Some people have a stem cell or bone marrow transplant using stem cells Open a glossary item from someone else (a donor). This is also known as an allogeneic transplant or allograft. Before the transplant you have treatment to prepare your body to receive the stem cells. You might hear this called conditioning treatment. 

There are two main types of conditioning treatment. These are:

  • full intensity (myeloablative) conditioning (MAC)

  • reduced intensity conditioning (RIC)

For full intensity conditioning you have very high doses of chemotherapy. With reduced intensity conditioning you have lower doses of chemotherapy. You might also have other treatments such as radiotherapy to the whole body (TBI Open a glossary item).

MAC kills all the leukaemia cells and healthy cells in your bone marrow. RIC means that some leukaemia cells and healthy cells are left behind. Both types of conditioning dampen down your immune system. This prepares your body for your transplant so it doesn’t reject the donor cells.

Maintenance

Maintenance treatment aims to reduce the risk of the leukaemia from coming back. Not everyone with AML will have this phase. The aim of maintenance is to have treatment to keep the AML away in the long term. This is usually for people with a high risk of it coming back. You might have chemotherapy or a targeted cancer drug in this phase. Chemotherapy maintenance is usually azacitidine.

If you have a targeted cancer drug it's usually a drug called midostaurin.

Non intensive treatment

Chemotherapy is the main part of non intensive treatment. The chemotherapy you have is in lower amounts (doses). You also don’t have as many drugs so you should have fewer and less severe side effects. The aim is to get the leukaemia away and keep it away for as long as possible.

Some of the chemotherapy drugs for non intensive treatment include:

  • azacitidine

  • low dose cytarabine

  • etoposide

  • decitabine

You might also have a targeted cancer drug called venetoclax with your chemotherapy.

Chemotherapy into the fluid around the spinal cord and brain

Leukaemia cells can sometimes travel to the brain and spinal cord. This is also known as the central nervous system or CNS. This is rare in AML. If this happens, you have specific treatment to this area. A doctor with specialist training carries this out. They will explain everything beforehand.

You have an injection of chemotherapy into the spine in your lower back. The chemotherapy circulates in the fluid around your spine and brain. This is called intrathecal chemotherapy.

You have 3 intrathecal chemotherapy sessions quite close together. You then have one a week for 4 to 6 weeks. You continue until there is no evidence of leukaemia cells.

You can read about how you have intrathecal chemotherapy below.

Chemotherapy for AML that comes back or resists treatment

Sometimes tests find leukaemia cells in the bone marrow while you’re having treatment. This means the leukaemia isn’t responding to the drugs you’re having. It’s called resistant or refractory leukaemia. If the leukaemia comes back after treatment this is called a relapse.

The treatment you have next depends on several factors including:

  • your age, general health and level of fitness

  • if you have gene changes (mutations) in the leukaemia cells

  • what treatment you’ve had before

  • how long you were in remission for

If you are well enough you usually have high dose chemotherapy followed by a stem cell transplant.

Some people are unable to have a stem cell transplant or intensive treatment. So you may have lower doses of chemotherapy. Some people only have a targeted cancer drug called gilteritinib.

Some of the chemotherapy drugs and combinations include:

  • fludarabine, cytarabine and G-CSF (FLAG)

  • fludarabine, cytarabine, G-CSF and idarubicin (FLAG-Ida)

  • cytarabine, daunorubicin and etoposide (ADE)

  • hydroxycarbamide

  • cytarabine

Growth factors

Granulocyte colony stimulating factor or G-CSF is a type of growth factor. You might have this during AML treatment. Growth factors are natural substances that stimulate the bone marrow to make blood cells.

After chemotherapy your white blood cell count drops. This means you are at an increased risk of developing an infection. The longer your white cell count is low, the greater your risk. Having a growth factor such as G-CSF helps your white cell count go up more quickly. This could lower the risk of infection.

The different types of G-CSF are called:

  • filgrastim

  • lenograstim

  • pegfilgrastim

Before you start chemotherapy

You need to have blood tests to make sure it’s safe to start your treatment. You have these either a few days before or on the day your treatment begins. You have blood tests before each round or cycle of treatment.

How you have chemotherapy for AML

Chemotherapy for leukaemia treatment comes in many different forms. These include tablets that you take by mouth, an injection into your muscle, or a drip into your bloodstream (intravenous). You might also have it as an injection into the fluid around the spinal cord and brain.

Chemotherapy into a vein

You have treatment through a thin short tube (a cannula) that goes into a vein in your arm each time you have treatment.

Or you might have it through a long plastic tube that goes into a large vein in your chest. This might be a:

  • central line

  • PICC line

  • Portacath - although this is rare

The aim is to keep your line in place throughout the course of your treatment. Some people need to have their line replaced a number of times during their treatment. This could be because of an infection for instance. 

Chemotherapy into the fluid around the spinal cord and brain

You have intrathecal chemotherapy in the same way you have a lumbar puncture. You lie on your side. Your doctor gives you a small injection to numb an area in your back. They then inject the drug between 2 of your spinal bones into the spinal fluid. It takes from 1 to 5 minutes. Afterwards you need to lie flat for an hour.

Diagram showing how you have a lumbar puncture

Taking your tablets or capsules

You must take tablets and capsules according to the instructions your doctor or pharmacist gives you.

You should take the right dose, not more or less.

Talk to your healthcare team before you stop taking a cancer drug, or if you have missed a dose.

If you are sick after taking chemotherapy tablets or capsules, don’t take them again straight away. Talk to your medical team for advice if you miss a dose of a cancer drug or want to stop taking treatment for any reason.

Side effects

Chemotherapy for AML can cause side effects and these can vary from person to person. It depends on the chemotherapy drugs, the dose you have and if you have them with other cancer drugs. Some of the common side effects include:

  • an increased risk of infection

  • breathlessness and looking pale

  • bruising, bleeding gums or nose bleeds

  • feeling or being sick

  • diarrhoea

  • tiredness and weakness

  • hair thinning or loss

  • sore mouth and ulcers

  • tumour lysis syndrome - this is when there are changes to the levels of substances in your blood due to the breakdown of cancer cells. It usually happens when you first start treatment.

Loss of fertility

Many people do go on to keep their fertility after AML treatment. But chemotherapy can affect some people, making it harder to get pregnant or get someone pregnant. Talk to your doctor before starting treatment if this is a concern for you. They can explain how this could affect you.

Dietary or herbal supplements

We don't yet know much scientifically about how some nutritional or herbal supplements might interact with treatment. Some could be harmful.

It is very important to tell your doctors if you are:

  • taking supplements
  • thinking of taking any supplements
  • prescribed supplements by alternative or complementary therapy practitioners 

Some supplements could make treatment side effects worse. Or it could make the treatment work less well.

Talk to your healthcare team about any other tablets or medicines you take while you are having treatment.

Research into chemotherapy for AML

Researchers are looking for new and kinder treatments to improve survival and quality of life for people with AML.

Support at home

Chemotherapy for AML can be difficult to cope with. Tell your doctor or nurse about any problems or side effects that you have. Your nurse will give you telephone numbers to call if you have any problems at home.

For general information and support you can talk to Cancer Research UK’s information nurses on freephone 0808 800 4040, Monday to Friday, 9am to 5pm

  • Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN
    H Dohner and others
    Blood, September 2022. Volume 140, Issue 12, Pages 1345 and 1377

  • Outcome of autologous stem cell transplantation in patients with favorable-risk acute myeloid leukemia in first remission
    J Chen and others
    Cancer Cell International, October 2022. Volume 22, Number 332

  • Acute myeloid leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up
    M Heuser and others
    Annals of Oncology, March 2020. Volume 31, Issue 6, Pages 697 to 712

  • Acute Myeloid Leukaemia
    C D DiNardo and others
    The Lancet, June 2023. Volume 401, Pages 2073 to 2086

  • Management of older patients with frailty and acute myeloid leukaemia: A British Society for Haematology good practice paper
    M Dennis and others
    British Journal of Haematology, October 2022. Volume 199, Issue 2, Pages 205 to 221

  • The information on this page is based on literature searches and specialist checking. We used many references and there are too many to list here. Please contact patientinformation@cancer.org.uk with details of the particular issue you are interested in if you need additional references for this information.

Last reviewed: 
23 Apr 2024
Next review due: 
23 Apr 2027

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