Acute Myeloid Leukemia Treatment (PDQ®): Treatment - Patient Information [NCI]
General Information About Acute Myeloid Leukemia
Adult acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells.
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia in adults. This type of cancer usually gets worse quickly if it is not treated. AML is also called acute myelogenous leukemia and acute nonlymphocytic leukemia.
Anatomy of the bone. The bone is made up of compact bone, spongy bone, and bone marrow. Compact bone makes up the outer layer of the bone. Spongy bone is found mostly at the ends of bones and contains red marrow. Bone marrow is found in the center of most bones and has many blood vessels. There are two types of bone marrow: red and yellow. Red marrow contains blood stem cells that can become red blood cells, white blood cells, or platelets. Yellow marrow is made mostly of fat.
Leukemia may affect red blood cells, white blood cells, and platelets.
Normally, the bone marrow makes blood stem cells (immature cells) that become mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. A lymphoid stem cell becomes a white blood cell.
A myeloid stem cell becomes one of three types of mature blood cells:
- Red blood cells that carry oxygen and other substances to all tissues of the body.
- Granulocytes, which are white blood cells that help fight infection and disease.
- Platelets that form blood clots to stop bleeding.
Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.
In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Sometimes in AML, too many stem cells become abnormal red blood cells or platelets. These abnormal white blood cells, red blood cells, or platelets are also called leukemia cells or blasts. Leukemia cells can build up in the bone marrow and blood so there is less room for healthy white blood cells, red blood cells, and platelets. When this happens, infection, anemia, or easy bleeding may occur.
The leukemia cells can spread outside the blood to other parts of the body, including the central nervous system (brain and spinal cord), skin, and gums. Sometimes leukemia cells form a solid tumor called a myeloid sarcoma. Myeloid sarcoma is also called extramedullary myeloid tumor, granulocytic sarcoma, or chloroma.
There are different subtypes of AML.
Most AML subtypes are based on how mature (developed) the cancer cells are at the time of diagnosis, and how different they are from normal cells.
Acute promyelocytic leukemia (APL) is a subtype of AML. This leukemia occurs when genes on chromosome 15 switch places with some genes on chromosome 17, and an abnormal gene called PML::RARA is made. The PML::RARA gene sends a message that stops promyelocytes (a type of white blood cell) from maturing. Problems with severe bleeding and blood clots may occur. This is a serious health problem that needs treatment as soon as possible. APL usually occurs in middle-aged adults.
Smoking, previous chemotherapy treatment, and exposure to radiation may affect the risk of AML.
Anything that increases a person's chance of getting a disease is called a risk factor. Not every person with one or more of these risk factors will develop AML, and it can develop in people who don't have any known risk factors. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following:
- Being male.
- Older age.
- Smoking.
- Having had treatment with chemotherapy or radiation therapy in the past.
- Being exposed to radiation in the environment (such as nuclear radiation) or to the chemical benzene.
- Having a personal history of a blood disorder such as myelodysplastic syndrome.
- Having certain syndromes or inherited disorders.
Signs and symptoms of AML include fever, feeling tired, and easy bruising or bleeding.
The early signs and symptoms of AML may be like those caused by the flu or other common diseases. Check with your doctor if you have any of the following:
- Weakness.
- Fever.
- Infection.
- Paleness or loss of normal skin color.
- Bleeding.
Less common signs or symptoms may be caused by clusters of leukemia cells in the central nervous system (CNS) or testicles, or a tumor of myeloid cells called a chloroma.
Symptoms of acute leukemia often develop between 4 and 6 weeks before diagnosis.
Tests that examine the blood and bone marrow are used to diagnose AML.
In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:
- Complete blood count (CBC): A procedure in which a sample of blood is drawn and checked for the following:
- The number of red blood cells, white blood cells, and platelets.
- The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
- The portion of the sample made up of red blood cells.
Complete blood count (CBC). Blood is collected by inserting a needle into a vein and allowing the blood to flow into a tube. The blood sample is sent to the laboratory and the red blood cells, white blood cells, and platelets are counted. The CBC is used to test for, diagnose, and monitor many different conditions. - Peripheral blood smear: A procedure in which a sample of blood is checked for blast cells, the number and kinds of white blood cells, the number of platelets, and changes in the shape of blood cells.
- Flow cytometry: A laboratory test that measures the number of cells in a sample, the percentage of live cells in a sample, and certain characteristics of the cells, such as size, shape, and the presence of tumor (or other) markers on the cell surface. The cells from a sample of a patient's blood, bone marrow, or other tissue are stained with a fluorescent dye, placed in a fluid, and then passed one at a time through a beam of light. The test results are based on how the cells that were stained with the fluorescent dye react to the beam of light. This test is used to help diagnose and manage certain types of cancers, such as leukemia and lymphoma.
- Bone marrow aspiration and biopsy: The removal of bone marrow, blood, and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for signs of cancer.
Bone marrow aspiration and biopsy. After a small area of skin is numbed, a bone marrow needle is inserted into the patient's hip bone. Samples of blood, bone, and bone marrow are removed for examination under a microscope. - Tumor biopsy: A biopsy of a tumor made up of leukemia cells, also known as a myeloid sarcoma (chloroma), may be done.
- Cytogenetic analysis: A laboratory test in which the chromosomes of cells in a sample of blood or bone marrow are counted and checked for any changes, such as broken, missing, rearranged, or extra chromosomes. Changes in certain chromosomes may be a sign of cancer. Cytogenetic analysis is used to help diagnose cancer, plan treatment, or find out how well treatment is working. Other tests, such as fluorescence in situ hybridization (FISH), may also be done to look for certain changes in the chromosomes.
- Molecular testing: A laboratory test to check for certain genes, proteins, or other molecules in a sample of blood or bone marrow. Molecular tests also check for certain changes in a gene or chromosome that may cause or affect the chance of developing AML. A molecular test may be used to help plan treatment, find out how well treatment is working, or make a prognosis.
- Immunophenotyping: A laboratory test that uses antibodies to identify cancer cells based on the types of antigens or markers on the surface of the cells. This test is used to help diagnose specific types of leukemia. For example, a cytochemistry study may test the cells in a sample of tissue using chemicals (dyes) to look for certain changes in the sample. A chemical may cause a color change in one type of leukemia cell but not in another type of leukemia cell.
- Reverse transcription–polymerase chain reaction test (RT–PCR): A laboratory test in which the amount of a genetic substance called mRNA made by a specific gene is measured. An enzyme called reverse transcriptase is used to convert a specific piece of RNA into a matching piece of DNA, which can be amplified (made in large numbers) by another enzyme called DNA polymerase. The amplified DNA copies help tell whether a specific mRNA is being made by a gene. RT-PCR can be used to check the activation of certain genes that may indicate the presence of cancer cells. This test may be used to look for certain changes in a gene or chromosome, which may help diagnose cancer. This test is used to diagnose certain types of AML including acute promyelocytic leukemia (APL).
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis and treatment options depend on the following:
- The age of the patient. Older age at diagnosis may be linked to lower remission rates and more complications.
- Whether the leukemia has spread to the central nervous system.
- Whether the patient has a systemic infection at the time of diagnosis.
- Whether the patient has a very high white blood cell count at the time of diagnosis.
- The subtype of AML.
- Whether the patient received chemotherapy or radiation therapy in the past to treat a different cancer.
- Whether there is a history of a blood disorder such as myelodysplastic syndrome.
- Whether the cancer has been treated before or recurred (come back).
It is important that acute leukemia be treated right away.
Stages of Acute Myeloid Leukemia
Once acute myeloid leukemia (AML) has been diagnosed, tests are done to find out if the cancer has spread to other parts of the body.
The process used to find out if cancer has spread is called staging. In acute myeloid leukemia (AML), the subtype of AML and whether the leukemia has spread outside the blood and bone marrow are used instead of the stage to plan treatment.
The following tests and procedures may be used to determine if the leukemia has spread:
- Lumbar puncture: A procedure used to collect a sample of cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the CSF around the spinal cord and removing a sample of the fluid. The sample of CSF is checked under a microscope for signs that leukemia cells have spread to the brain and spinal cord. This procedure is also called an LP or spinal tap.
Lumbar puncture. A patient lies in a curled position on a table. After a small area on the lower back is numbed, a spinal needle (a long, thin needle) is inserted into the lower part of the spinal column to remove cerebrospinal fluid (CSF, shown in blue). The fluid may be sent to a laboratory for testing. - CT scan (CAT scan): A procedure that makes a series of detailed pictures of the abdomen, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organ or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
There is no standard staging system for AML.
The disease is described as untreated, in remission, refractory, or recurrent.
Newly diagnosed (untreated) AML
In untreated AML, the disease is newly diagnosed. It has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and the following are true:
- The complete blood count is abnormal.
- At least 20% of the cells in the bone marrow are blasts (leukemia cells) or there are certain gene changes.
- There are signs or symptoms of leukemia.
AML in remission
In AML in remission, the disease has been treated and the following are true:
- The complete blood count is normal.
- Less than 5% of the cells in the bone marrow are blasts (leukemia cells).
- There are no signs or symptoms of leukemia in the brain and spinal cord or elsewhere in the body.
Refractory or recurrent AML
After treatment with chemotherapy, some patients with newly diagnosed AML will not go into remission. This is called refractory cancer. In contrast, recurrent AML is cancer that has recurred (come back) after remission. The AML may come back in the blood or bone marrow.
Treatment Option Overview
There are different types of treatment for patients with acute myeloid leukemia (AML).
Different types of treatment are available for patients with acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients with cancer. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.
The treatment of AML usually has two phases.
The two treatment phases of AML are:
- Remission induction therapy: This is the first phase of treatment. The goal is to kill the leukemia cells in the blood and bone marrow. This puts the leukemia into remission.
- Postremission therapy: This is the second phase of treatment. It begins after the leukemia is in remission. The goal of postremission therapy is to kill any remaining leukemia cells that may not be active but could begin to regrow and cause a relapse. This phase is also called remission continuation therapy.
Patients receive supportive care for side effects of treatment.
Patients must be closely monitored during treatment of AML. Myelosuppression, a condition which results in fewer red blood cells, white blood cells, and platelets, is a side effect of both AML and treatment with chemotherapy. Supportive care during remission induction therapy may include:
- Red blood cell and platelet transfusions.
- Antibiotics and antifungals for treatment of infections.
The following types of treatment are used:
Chemotherapy
Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Intrathecal chemotherapy may be used to treat adult AML that has spread to the brain and spinal cord. Combination chemotherapy is treatment using more than one anticancer drug.
The way the chemotherapy is given depends on the subtype of AML being treated and whether leukemia cells have spread to the brain and spinal cord.
Intrathecal chemotherapy. Anticancer drugs are injected into the intrathecal space, which is the space that holds the cerebrospinal fluid (CSF, shown in blue). There are two different ways to do this. One way, shown in the top part of the figure, is to inject the drugs into an Ommaya reservoir (a dome-shaped container that is placed under the scalp during surgery; it holds the drugs as they flow through a small tube into the brain). The other way, shown in the bottom part of the figure, is to inject the drugs directly into the CSF in the lower part of the spinal column, after a small area on the lower back is numbed.
For more information, see Drugs Approved for Acute Myeloid Leukemia.
Radiation therapy
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body with cancer. Total-body irradiation sends radiation toward the whole body. It is a type of external radiation that may be used to prepare the body for a stem cell transplant when the leukemia has recurred.
Chemotherapy with stem cell transplant
High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy and/or total-body irradiation, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
Stem cell transplant. (Step 1): Blood is taken from a vein in the arm of the donor. The patient or another person may be the donor. The blood flows through a machine that removes the stem cells. Then the blood is returned to the donor through a vein in the other arm. (Step 2): The patient receives chemotherapy to kill blood-forming cells. The patient may receive radiation therapy (not shown). (Step 3): The patient receives stem cells through a catheter placed into a blood vessel in the chest.
Targeted therapy
Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. There are different types of targeted therapy.
Monoclonal antibodies: immune system proteins made in the laboratory to treat many diseases, including cancer. As a cancer treatment, these antibodies can attach to a specific target on cancer cells or other cells that may help cancer cells grow. The antibodies are able to then kill the cancer cells, block their growth, or keep them from spreading. Monoclonal antibodies are given by infusion. They may be used alone or to carry drugs, toxins, or radioactive material directly to cancer cells.
- Gemtuzumab ozogamicin is a type of antibody-drug conjugate used to treat patients with newly diagnosed or relapsed AML. It contains a monoclonal antibody that binds to CD33, which is found on some leukemia cells, and also contains a toxic substance, which may help kill cancer cells.
Other targeted therapies include:
- Midostaurin: a protein kinase inhibitor used with certain types of chemotherapy to treat newly diagnosed patients with AML that has a mutation in the FLT3 gene.
- Gilteritinib: a tyrosine kinase inhibitor that may be used to treat patients with AML that has come back or did not get better with other treatment and has a mutation in the FLT3 gene.
Less-intensive targeted therapies in older or frail patients who cannot receive other treatments include:
- Glasdegib plus low-dose chemotherapy.
- Ivosidenib with or without low-dose chemotherapy for patients with AML that has a mutation in the IDH1 gene.
- Enasidenib for patients with AML that has a mutation in the IDH2 gene.
For more information, see Drugs Approved for Acute Myeloid Leukemia.
Other drug therapy
Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a subtype of AML called acute promyelocytic leukemia.
For more information, see Drugs Approved for Acute Myeloid Leukemia.
New types of treatment are being tested in clinical trials.
This summary section describes treatments that are being studied in clinical trials. It may not mention every new treatment being studied. Information about clinical trials is available from the NCI website.
Treatment for acute myeloid leukemia may cause side effects.
For information about side effects caused by treatment for cancer, see our Side Effects page.
Patients may want to think about taking part in a clinical trial.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for cancer are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way cancer will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Patients can enter clinical trials before, during, or after starting their cancer treatment.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Clinical trials are taking place in many parts of the country. Information about clinical trials supported by NCI can be found on NCI's clinical trials search webpage. Clinical trials supported by other organizations can be found on the ClinicalTrials.gov website.
Follow-up tests may be needed.
Some of the tests that were done to diagnose the cancer or to find out the stage of the cancer may be repeated. Some tests will be repeated in order to see how well the treatment is working. Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.