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Home > Patients and Visitors > Acute Myeloid Leukemia Treatment (PDQ®): Treatment - Patient Information [NCI]
This information is produced and provided by the National Cancer Institute (NCI). The information in this topic may have changed since it was written. For the most current information, contact the National Cancer Institute via the Internet web site at http://cancer.gov or call 1-800-4-CANCER.
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:
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.
This summary is about adult AML. See the following PDQ summaries for information related to AML:
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 your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn't mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Possible risk factors for AML include the following:
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:
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.
The following tests and procedures may be used:
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis and treatment options depend on:
It is important that acute leukemia be treated right away.
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 extent or spread of cancer is usually described as stages. 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:
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:
AML in remission
In AML in remission, the disease has been treated and the following are true:
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.
There are different types of treatment for patients with acute myeloid leukemia.
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:
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:
Five types of treatment are used:
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.
See Drugs Approved for Acute Myeloid Leukemia for more information.
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
Chemotherapy is 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 is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells. Targeted therapies usually cause less harm to normal cells than chemotherapy or radiation therapy do. There are different types of targeted therapy:
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.
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.
For information about the treatments listed below, see the section.
Standard treatment of untreated acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following:
For older adults or patients too frail to receive intensive chemotherapy, the following may be continued as long as the patient benefits or until toxic effects occur:
Use our clinical trial search to find NCI-supported cancer clinical trials that are accepting patients. You can search for trials based on the type of cancer, the age of the patient, and where the trials are being done. General information about clinical trials is also available.
For information about the treatments listed below, see the Treatment Option Overview section.
Treatment of acute myeloid leukemia (AML) during the remission phase depends on the subtype of AML and may include the following:
There is no standard treatment for refractory or recurrent acute myeloid leukemia (AML). Treatment depends on the subtype of AML and may include the following:
Treatment of newly diagnosed acute promyelocytic leukemia (APL) includes:
Treatment of recurrent acute promyelocytic leukemia includes:
For more information from the National Cancer Institute about adult acute myeloid leukemia, see the following:
For general cancer information and other resources from the National Cancer Institute, see the following:
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Purpose of This Summary
This PDQ cancer information summary has current information about the treatment of adult acute myeloid leukemia. It is meant to inform and help patients, families, and caregivers. It does not give formal guidelines or recommendations for making decisions about health care.
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Clinical Trial Information
A clinical trial is a study to answer a scientific question, such as whether one treatment is better than another. Trials are based on past studies and what has been learned in the laboratory. Each trial answers certain scientific questions in order to find new and better ways to help cancer patients. During treatment clinical trials, information is collected about the effects of a new treatment and how well it works. If a clinical trial shows that a new treatment is better than one currently being used, the new treatment may become "standard." 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.
Clinical trials can be found online at NCI's website. For more information, call the Cancer Information Service (CIS), NCI's contact center, at 1-800-4-CANCER (1-800-422-6237).
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PDQ® Adult Treatment Editorial Board. PDQ Acute Myeloid Leukemia Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/adult-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389377]
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Last Revised: 2022-03-04
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