AML World Awareness Day 21 April 2019


AML Treatment

The aim of AML treatment is to help control the abnormal leukemia cells and restore a balance of healthy blood cells in the bone marrow.

AML is not a single disease but rather a disease of many types, so different types of AML can have different outcomes and unique therapies.


The main standard treatment for most people with AML is chemotherapy. Chemotherapy involves using anti-cancer drugs to destroy abnormal leukemia blood cells and bring about remission.

Remission is when there are no more abnormal leukemia cells in the blood and bone marrow and normal blood cells are developing again. Chemotherapy treatment is usually given in two main phases – induction and consolidation.

Induction phase

This is the first phase of treatment and its aim is to kill abnormal leukemia cells and stop them from multiplying in the blood and bone marrow. The most commonly used induction chemotherapy drugs include cytarabine and an anthracycline drug such as daunorubicin or idarubicin. Other drugs for induction include a formulation of daunorubicin and cytarabine (Vyxeos), which is approved in the United States and Europe to treat patients with specific types of AML, including therapy-related AML and AML with myelodysplasia-related changes.

Chemotherapy can sometimes be administered with drugs that target specific genes (targeted therapy) that have changed in abnormal cells.

Consolidation phase

This phase of chemotherapy treatment aims to kill any remaining abnormal leukemia cells that may be present after induction chemotherapy but which cannot be seen. This then reduces the risk of the leukemia coming back. Consolidation phase includes further treatment with chemotherapy.

In some instances, treatment with chemotherapy and targeted therapy is sometimes followed by a stem-cell transplant. The anti-leukemic treatment (chemotherapy and targeted therapy) will destroy some blood cells (abnormal and normal) and then a stem-cell transplant will replace them with stem cells that produce normal healthy blood cells.

Targeted therapy

In the past years, new drugs have been developed targeting specific genetic abnormalities or specific proteins found in leukemia cells. These targeted drugs work differently to the standard chemotherapeutic agents and can be useful for patients who have a specific type of gene abnormality. These targeted drugs also have different side effects to chemotherapeutic agents. Some approved targeted treatments for specific types of AML are shown below.

Midostaurin (Rydapt)

Abnormal changes in the FLT3 gene happen in approximately 30% of AML cases. These gene changes contribute to why leukemia cells become abnormal in AML. Midostaurin is a drug that targets this abnormal FLT3 gene and blocks its function. Midostaurin is approved in the US (by the FDA) and in Europe (by the EMA), to be used with standard chemotherapy to treat adult patients, newly diagnosed with AML, who have abnormal changes in the FLT3 gene.

Enasidenib (Idhifa)

In some AML cases, there are abnormal changes in the IDH2 gene. Enasidenib is a drug that blocks the function of the IDH2 gene in leukemia cells. Enasidenib is approved in the US by the FDA to treat patients whose AML returned after treatment (relapse) or patients who did not respond to initial therapy (refractory) with abnormal changes in the IDH2 gene.

Gemtuzumab ozogamicin (Mylotarg)

Gemtuzumab ozogamicin contains an antibody which is a synthetic immune protein, connected to a chemotherapeutic drug. The antibody attaches to a specific protein called CD33 present on the leukemia cell’s surface. In the US, gemtuzumab ozogamicin is FDA-approved for newly diagnosed adult AML patients who have CD33 protein on their leukemia cells. It is also FDA-approved for adults and children who have relapsed or refractory AML. In Europe, gemtuzumab ozogamicin is approved for the treatment of patients age 15 years and above who have CD33 protein on their leukemia cells.

Ivosidenib (Tibsovo)

In some AML cases, there are abnormal changes in the IDH1 gene. Ivosidenib is a drug that blocks the function of the IDH1 gene in leukemia cells. Ivosidenib is approved in the US by the FDA to treat patients whose AML returned after treatment (relapse) or patients who did not respond to initial therapy (refractory) with abnormal changes in the IDH1 gene.

Acute promyelocytic leukemia (APL) treatment

APL is treated differently to other forms of AML. APL cells have a change in the genes that makes them sensitive to specific drugs. Often, treatment of APL consists of a non-chemotherapy drug called ATRA (all-trans-retinoic acid, Tretinoin) in combination with either an anthracycline chemotherapy drug or arsenic trioxide (Trisenox).

In the US, the FDA have approved the use of arsenic trioxide in combination with ATRA for treating adults with newly diagnosed low-risk APL who have the presence of the abnormal PML/RARα gene. Arsenic trioxide is also approved by the EMA and the FDA to treat patients with relapsed or refractory APL who have abnormal changes in the PML/RARα gene.

Some specific types of AML currently do not have any approved therapy. However, there might be a new treatment that is targeted for a specific type of AML that is not yet approved but is available in a clinical trial (research studies).

What is a clinical trial?

A clinical trial is a research study that can be used to improve current treatments or to get more information on a new promising treatment in terms of how well it works and its safety.

An AML doctor might suggest a new treatment for a specific type of AML that is not yet approved but is available in a clinical trial. Several ongoing clinical trials aim to support new promising treatments for AML.

Taking part in a clinical trial might be an option for people with AML as the trials give access to new therapies that are not yet approved. If you are offered the chance to take part in a clinical trial, you could be one of the first people to benefit from a new treatment. However, there is also a chance that the new medicine may turn out to be no better, or could be worse, than the standard treatment.

What treatment side effects might I have?

Different treatments can result in different side effects. For most patients, side effects are temporary and will go away when treatment has finished.

The reaction to a treatment varies from one patient to another. Side effects also vary depending on the type of treatment and how long it lasts. And, the side effects associated with chemotherapy are different from targeted therapy. Side effects from targeted therapy depend mainly on what the drug is targeting.

Common side effects from treatment include tiredness, increased risk of infection, nausea (feeling sick), vomiting (being sick), hair loss, rashes and anemia (low levels of red blood cells).

Sometimes, AML treatment can lead to side effects that do not stop once the treatment has finished, or side effects that develop a long time after treatment has finished. An example of such a side effect is increased injury to the heart muscle. Treatments may also sometimes lead to infertility and the risk of developing a different type of cancer.

You're not alone

There are several resources that are available for you. These can provide further in-depth information about acute myeloid leukemia (AML) and also offer you support.

Find your local support