Treatment
Targeted drugs are grouped together depending on how they work. The main targeted cancer drugs in AML include:
midostaurin
gemtuzumab ozogamicin (Mylotarg)
venetoclax
gilteritinib
ivosidenib
You might have other targeted cancer drugs if you are having treatment as part of a .
You might have a targeted cancer drug with chemotherapy. Or you might have it on its own.
Treatment for AML is generally divided into intensive and non intensive treatment.
Intensive treatment aims to cure your AML. You might have this if your doctor thinks you are fit and well enough to have this treatment.
The targeted cancer drug you might have in the intensive treatment includes:
midostaurin OR
gemtuzumab ozogamicin (Mylotarg)
Non intensive treatment aims to control your cancer for as long as possible.
Non intensive treatment generally causes 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 might have a drug called venetoclax or ivosidenib as part of your non intensive treatment.
AML that isn’t responding to treatment is called resistant or refractory leukaemia. AML that comes back after treatment has finished is called relapse. In these situations, you might have a targeted drug called gilteritinib.
When you are diagnosed with AML you have several different tests that look for changes:
on the surface of your leukaemia cells
in the genes and chromosomes of your leukaemia cells
Your doctor looks at a sample of your cancer from a bone marrow test to do this. Your doctor may use a blood test to do this, but this is less common.
The results of the tests show whether a targeted cancer drug is suitable for you.
Midostaurin is a type of targeted drug called a cancer growth blocker. Cancer growth blockers stop the growth factors that trigger the cancer cell to divide and grow.
You might have midostaurin if your AML has a called FLT-3. You may have it:
alongside chemotherapy to get rid of your leukaemia (induction) and reduce the risk of it coming back (consolidation)
on its own as a maintenance treatment following consolidation
You have this drug through a drip into your vein.
Go to information about midostaurin for maintainence treatment
This is another type of cancer growth blocker. You have venetoclax with a chemotherapy drug. This is either azacitidine or low dose cytarabine.
You may have this combination of drugs as a first treatment for AML if you are unable to have intensive chemotherapy.
Venetoclax comes as tablets that you take once a day.
Find out more about venetoclax
This is also a cancer growth blocker. You might have gilteritinib if your AML has a gene change (mutation) called FLT-3. It’s for people with AML that has come back or relapsed.
You have gilteritinib on its own. It comes as tablets that you take once a day.
Find out more about having gilteritinib
Gemtuzumab ozogamicin belongs to a group of targeted drugs called monoclonal antibodies (MABs). MABS target specific proteins on cancer cells and work in different ways to kill the cancer cell and stop it from growing.
You might have gemtuzumab ozogamicin if there is a protein receptor called CD33 on your leukaemia cells. The gemtuzumab sticks to all the CD33 proteins it finds. Then ozogamicin enters the cells and kills them.
You have gemtuzumab ozogamicin with the chemotherapy drugs daunorubicin and cytarabine. We have more information about this combination of drugs in our cancer drugs section.
Go to gemtuzumab ozogamicin, daunorubicin and cytarabine
Ivosidenib is also a type of cancer growth blocker. You might have ivosidenib if your AML has a gene change (mutation) called IDH1. You have this with another drug called azacitidine.
You may have this combination of drugs as a first treatment for AML if you are unable to have intensive chemotherapy.
Ivosidenib comes as a tablet that you take once a day.
Targeted cancer drugs can cause different side effects. Some of these can be serious. Your doctor or nurse will talk to you about this. Always tell them about any side effects you have and follow the advice they give you.
Choose a drug on our A to Z list to read about side effects and find out more about the drug.
Researchers are looking at:
new targeted cancer drugs
different combinations of these drugs with other treatment
Find clinical trials with targeted cancer drugs for AML
This treatment can be difficult to cope with. Your nurse will give you an advice line number to call if you have any problems at home.
Tell your doctor or nurse about any problems or side effects you have.
Last reviewed: 16 Apr 2024
Next review due: 16 Apr 2027
Chemotherapy is the main treatment for acute myeloid leukaemia (AML). Find out how your doctor decides your treatment, detailed information about the each treatment, the side effects and follow up.
Get support to cope during and after acute myeloid leukaemia (AML) treatment, including information on diet, physical activity, sex, fertility and life after a transplant.
Treatment for AML is generally divided into intensive and non intensive treatment. The main treatment in both situations is chemotherapy. Find out about how your doctor decides which treatment you need and the types of treatment you might have.
Find out about the latest UK research looking at acute myeloid leukaemia (AML), as well as how you can take part.
AML starts from young white blood cells called granulocytes or monocytes in the bone marrow. Find out about symptoms, how it is diagnosed and treated, and how to cope.
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