Acute Myeloid Leukemia Treatment: What to Expect

What is acute myeloid leukemia (AML)?

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It affects white blood cells (WBCs), making them abnormal. In some forms of AML, they may also multiply very quickly.

Other names for AML include:

• acute myelocytic leukemia
• acute myelogenous leukemia
• acute granulocytic leukemia
• acute non-lymphocytic leukemia

Read this for more information about AML.

What are the treatment options for acute myeloid leukemia (AML)?

Once the diagnosis is confirmed, your healthcare team will develop a plan to treat AML. Depending on the specific type and stage of AML, you may receive one or more of these treatments:

Chemotherapy

Chemotherapy is the main form of treatment for AML. It’s divided into two phases:

• remission induction, usually just called induction
• consolidation, also called post-remission therapy

Since AML can progress quickly, treatment is usually started as soon as possible after diagnosis. Other treatments may be used as well.

Chemotherapy, also called chemo, is the use of anti-cancer drugs to treat cancer. This is the main treatment for AML.

These drugs can be injected into a vein or under the skin, allowing the chemotherapy to travel through the bloodstream to attack cancer cells throughout the body. If leukemia has been found in the brain or spinal cord, chemo medication may be injected into the cerebrospinal fluid (CSF).

Chemo medications most often used to treat AML include:

• cytarabine, also known as cytosine arabinoside or ara-C
• anthracyclines, like daunorubicin or idarubicin

Other chemo medications may include:

• cladribine (2-CdA)
• fludarabine
• etoposide (VP-16)
• corticosteroids
• methotrexate (MTX)

Side effects of chemotherapy can vary depending on the drug, dosage, and duration. They can include:

• hair loss
• mouth sores
• loss of appetite
• nausea
• vomiting
• diarrhea
• constipation
• fatigue
• easy bruising
• bleeding

Non-chemotherapy medications

While chemotherapy is the main treatment for AML, for a subtype of AML called acute promyelocytic leukemia (APL), other non-chemotherapy drugs are more effective.

APL is caused by a specific gene mutation that affects WBCs. Some medications work better than chemo to help those cells develop normally. Two of these medications are:

• all-trans retinoic acid (ATRA)
• arsenic trioxide (ATO, Trisenox)

ATRA can be given with chemotherapy or with ATO for the initial treatment of APL. Both drugs can also be given during consolidation.

Side effects of ATRA include:

• headache
• fever
• dry skin and mouth
• mouth or throat sores
• elevated blood lipid levels
• irritated eyes

Side effects of ATO can include:

• fatigue
• nausea
• vomiting
• diarrhea
• belly pain
• nerve damage causing tingling in the hands and feet

Radiation therapy

Radiation therapy uses high-energy radiation to kill cancer cells. While it’s not the main treatment for those with AML, it can be used in treating AML. In AML, the radiation used is external beam radiation, which is similar to an X-ray.

Radiation can be used in AML to treat:

• leukemia that has spread to the brain and/or spinal cord, or to the testicles
• the whole body before a stem cell transplant
• sometimes used to shrink a tumor if it is impairing breathing
• bone pain due to leukemia that is not relieved by chemotherapy

Side effects of radiation can include:

• low blood counts, which can cause fatigue or increased risk of infection
• nausea, vomiting, or diarrhea if radiation is given to the abdomen
• sunburn-like skin changes and hair loss on the radiated area
• mouth sores and problems swallowing if given to the head/neck area

Surgery

Surgery is rarely used in AML treatment. Leukemia cells are spread through the bone marrow and blood, making the condition impossible to improve with surgery. On rare occasions, a tumor or mass related to leukemia may form that may be treated with surgery.

Prior to chemotherapy, a small surgery to place a central venous catheter (CVC) or a central line, is often done. During this procedure, a small flexible tube is placed into a large vein in the chest. The end of it is either right under the skin or sticks out in the chest or upper arm.

Having a central line installed allows the treatment team to give intravenous medication and chemotherapy through the CVC, and to draw blood from it, reducing the number of needle sticks an individual has to have.

Stem cell therapy

While chemotherapy is the main treatment for AML, it has its limits. Since high doses of these medications are toxic, the dosage must be limited. A stem cell transplant allows for higher doses of chemotherapy medications.

In a stem cell transplant, very high doses of chemotherapy medications, sometimes combined with radiation, are given. All of the individual’s original bone marrow is destroyed on purpose.

Once this stage of therapy is over, blood-forming stem cells are given. These stem cells will grow, rebuilding the bone marrow. Healthy, cancer-free stem cells replace the destroyed bone marrow.

Read this article for more information about a stem cell transplant.

Targeted therapy

Targeted therapy drugs are medications that target only certain parts of cancer cells. They can be very effective for some people with AML. Most targeted therapy drugs are taken orally, except for gemtuzumab ozogamicin (Mylotarg), which is given as an intravenous infusion.

Talk with your treatment team about the potential side effects of each drug and what you should watch for when taking it. Some targeted therapy medications include:

FLT3 inhibitors

One type of targeted therapy medication called FLT3 inhibitors targets the FLT3 gene. In some people with AML, a mutation in the FLT3 gene causes the creation of a protein, also called FLT3, that enables cancer cells to grow. Drugs in this category include:

• midostaurin (Rydapt)
• gilteritinib (Xospata)

Side effects of these drugs may include:

• low levels of white blood cells
• fever
• nausea
• vomiting
• mouth sores
• bone pain
• shortness of breath
• diarrhea
• abnormal liver function tests

IDH inhibitors

In some people with AML, there is a mutation in the IDH2 gene. These mutations stop bone marrow cells from maturing in a normal way. Medications called IDH inhibitors block IDH proteins produced by these mutated genes, allowing these bone marrow cells to grow normally and remain healthy.

Drugs in this category include:

• ivosidenib (Tibsovo)
• enasidenib (Idhiva)

Side effects can include:

• nausea
• vomiting
• diarrhea
• fatigue
• joint pain
• shortness of breath
• loss of appetite
• increased levels of bilirubin

Monoclonal antibody for CD33

AML cells contain a protein called CD33. A medication called gemtuzumab ozogamicin (Mylotarg) attaches to this CD33 protein and helps deliver chemotherapy medications directly to cancer cells so that these drugs are more effective.

Common side effects include:

• increased risk of infection
• fever
• nausea
• vomiting
• mouth sores
• constipation
• rash
• headaches

There are less common — but serious — side effects like:

• severe liver damage
• changes in the heart rhythm
• life threatening infections
• infusion reactions

BCL-2 inhibitors

Venetoclax (Venclexta) is a BCL-2 inhibitor. This drug targets BCL-2, which is a protein that helps cancer cells live longer. The drug stops the BCL-2 protein from helping cancer cells survive so that these cancer cells die sooner. This medication can be used along with other chemotherapy drugs.

Side effects include:

• low white blood cells (increased risk of infection)
• anemia
• diarrhea
• nausea
• fatigue
• tumor lysis syndrome

Hedgehog pathway inhibitors

AML can cause cellular mutations that prevent cells like bone marrow cells from developing and functioning normally. These mutations may affect the pathway cells use to send necessary signals. This pathway is called hedgehog. For some people with AML, especially those over age 75, strong chemo medications may be so harmful that chemo is not an option. For these individuals, a medication called, Glasdegib (Daurismo), may help them live longer. This medication helps stop the mutations and allows bone marrow cells to function normally.

Side effects of this medication may include:

• low white and red blood cell counts (increased risk of infection and anemia)
• bleeding
• nausea
• low platelet counts
• mouth sores

What is refractory AML?

Refractory AML happens when an individual is not in remission even after one to two cycles of induction chemotherapy, which means they have a blast count of 5 percent or more. Ten to 40 percent of people with AML have refractory AML.

If treatment isn’t successful with one course of chemo, another one may be done. If a person is still not in remission after the second course of chemo, they may be given other medications or an increased dose of their current chemotherapy medications.

Other treatment options include stem cell transplant or a clinical trial of new therapies.

What is remission?

When an individual has no evidence of disease after treatment, it’s called remission or complete remission. Remission means these three criteria are met:

• The bone marrow has fewer than 5 percent blast cells.
• Blood cell counts are within normal limits.
• There are no signs or symptoms of leukemia.

If there is no evidence at all of leukemia cells in the bone marrow, using highly sensitive tests, it’s called complete molecular remission. Minimal residual disease (MRD) occurs when, after treatment, leukemia cells cannot be seen in the bone marrow with standard tests but more sensitive tests like PCR tests do find leukemia cells.

Even after an individual has entered remission, they will likely need follow-up care and will need to be monitored by their doctor and healthcare team. This may mean additional tests, more frequent physical exams, and other care.

The bottom line

Although chemotherapy is the main treatment for AML, there are a variety of treatment options, depending on the AML subtype or whether you have a specific mutation. Treatment also depends on your response to initial treatment and whether or not remission is sustained.

Your treatment team will explain all of your treatment options and help you choose the treatment plan that is best for you and your individual situation.