Adult Acute Myeloid Leukemia Treatment (PDQ®)
Topics in This Section
- General Information About Adult Acute Myeloid Leukemia
- Treatment Option Overview
- Treatment Options for Adult Acute Myeloid Leukemia
- Untreated Adult Acute Myeloid Leukemia
- Adult Acute Myeloid Leukemia in Remission
- Recurrent Adult Acute Myeloid Leukemia
- Get More Information From NCI
- Changes to This Summary (02/22/2008)
- About PDQ
Adult acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. This type of cancer usually gets worse quickly if it is not treated. It is the most common type of acute leukemia in adults. AML is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, and acute nonlymphocytic leukemia.
Normally, the bone marrow makes blood stem cells (immature cells) that develop into mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. The lymphoid stem cell develops into a white blood cell. The myeloid stem cell develops into one of three types of mature blood cells:
- Red blood cells that carry oxygen and other materials to all tissues of the body.
- White blood cells that fight infection and disease.
- Platelets that help prevent bleeding by causing blood clots to form.

In AML, the myeloid stem cells usually develop into 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 develop into 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.
This summary is about adult AML. See the following PDQ summaries for information about other types of leukemia:
- Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment
- Chronic Myelogenous Leukemia Treatment
- Adult Acute Lymphoblastic Leukemia Treatment
- Childhood Acute Lymphoblastic Leukemia Treatment
- Chronic Lymphocytic Leukemia Treatment
- Hairy Cell Leukemia Treatment
The 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.
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. People who think they may be at risk should discuss this with their doctor. Possible risk factors for AML include the following:
- Being male.
- Smoking, especially after age 60.
- Having had treatment with chemotherapy or radiation therapy in the past.
- Having had treatment for childhood acute lymphoblastic leukemia (ALL) in the past.
- Being exposed to atomic bomb radiation or the chemical benzene.
- Having a history of a blood disorder such as myelodysplastic syndrome.
The early signs of AML may be like those caused by the flu or other common diseases. A doctor should be consulted if any of the following problems occur:
- Fever.
- Shortness of breath.
- Easy bruising or bleeding.
- Petechiae (flat, pinpoint spots under the skin caused by bleeding).
- Weakness or feeling tired.
- Weight loss or loss of appetite.
The following tests and procedures may be used:
- Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.
- 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.
- Blood chemistry studies: A procedure in which a blood sample is checked to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease in the organ or tissue that makes it.
- Peripheral blood smear: A procedure in which a sample of blood is checked for the presence of blast cells, number and kinds of white blood cells, the number of platelets, and changes in the shape of blood cells.
- 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 Jamshidi needle (a long, hollow needle) is inserted into the patient’s hip bone. Samples of blood, bone, and bone marrow are removed for examination under a microscope.
- Cytogenetic analysis: A laboratory test in which the cells in a sample of blood or bone marrow are viewed under a microscope to look for certain changes in the chromosomes.
- Immunophenotyping: A process used to identify cells, based on the types of antigens or markers on the surface of the cell. This process is used to diagnose the subtype of AML by comparing the cancer cells to normal cells of the immune system.
The extent or spread of cancer is usually described as stages. In adult 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:
- Chest x-ray: An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.
- Lumbar puncture: A procedure used to collect cerebrospinal fluid from the spinal column. This is done by placing a needle into the spinal column. 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.
- Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs in the abdomen and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later.
The disease is described as untreated, in remission, or recurrent.
Untreated adult AML
In untreated adult AML, the disease is newly diagnosed. It has not been treated except to relieve 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 blast (leukemia cells).
- There are signs or symptoms of leukemia.
Adult AML in remission
In adult 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.
Recurrent Adult AML
Recurrent AML is cancer that has recurred (come back) after it has been treated. The AML may come back in the blood or bone marrow.
The prognosis (chance of recovery) and treatment options depend on:
- The age of the patient.
- The subtype of AML.
- Whether the patient received chemotherapy 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 spread to the central nervous system.
- Whether the cancer has been treated before or recurred (come back).
It is important that acute leukemia be treated right away.
Different types of treatment are available for patients with adult acute myeloid leukemia (AML). Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. Before starting treatment, patients may want to think about taking part in a clinical trial. 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.
Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI Web site. Choosing the most appropriate cancer treatment is a decision that ideally involves the patient, family, and health care team.
The 2 treatment phases of adult AML are:
- Remission induction therapy: This is the first phase of treatment. Its purpose is to kill the leukemia cells in the blood and bone marrow. This puts the leukemia into remission.
- Maintenance therapy: This is the second phase of treatment. It begins after the leukemia is in remission. The purpose of maintenance 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.
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 spinal column (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, or may 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 the cancer being treated and whether it has spread to the brain and spinal cord.

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. There are two types of radiation therapy. External radiation therapy uses a machine outside the body to send radiation toward the cancer. Internal radiation therapy uses a radioactive substance sealed in needles, seeds, wires, or catheters that are placed directly into or near the cancer. The way the radiation therapy is given depends on the type and stage of the cancer being treated.
Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells that are abnormal or destroyed by the cancer treatment. 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 chemotherapy is completed, 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.
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.
Biologic therapy is a treatment that uses the patient's immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body's natural defenses against cancer. This type of cancer treatment is also called biotherapy or immunotherapy.
One type of biologic therapy is monoclonal antibody therapy, a cancer treatment that uses antibodies made in the laboratory from a single type of immune system cell. These antibodies can identify substances on cancer cells or normal substances that may help cancer cells grow. The antibodies attach to the substances and 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.
This summary section refers to specific treatments under study in clinical trials, but it may not mention every new treatment being studied. Information about ongoing clinical trials is available from the NCI Web site.
Standard treatment of untreated adult acute myeloid leukemia (AML) during the remission induction phase depends on the subtype of AML and may include the following:
- Combination chemotherapy.
- High-dose combination chemotherapy.
- Stem cell transplant using donor stem cells.
- All-trans retinoic acid (ATRA) plus chemotherapy.
- Intrathecal chemotherapy.
Information about ongoing clinical trials is available from the NCI Web site.
Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with untreated adult acute myeloid leukemia.
Standard treatment of adult AML during the remission phase depends on the subtype of AML and may include the following:
- Combination chemotherapy.
- High-dose chemotherapy, with or without radiation therapy, and stem cell transplant using the patient's stem cells.
- High-dose chemotherapy and stem cell transplant using donor stem cells.
Information about ongoing clinical trials is available from the NCI Web site.
Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with adult acute myeloid leukemia in remission.
There is no standard treatment for recurrent adult AML. Treatment depends on the subtype of AML and may include the following:
- Combination chemotherapy.
- Biologic therapy with monoclonal antibodies.
- Stem cell transplant.
- Arsenic trioxide therapy.
Information about ongoing clinical trials is available from the NCI Web site.
Check for clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with recurrent adult acute myeloid leukemia.
Call 1-800-4-CANCER
For more information, U.S. residents may call the National Cancer Institute's (NCI's) Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237) Monday through Friday from 9:00 a.m. to 4:30 p.m. Deaf and hard-of-hearing callers with TTY equipment may call 1-800-332-8615. The call is free and a trained Cancer Information Specialist is available to answer your questions.
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The NCI's LiveHelp® online chat service provides Internet users with the ability to chat online with an Information Specialist. The service is available from 9:00 a.m. to 11:00 p.m. Eastern time, Monday through Friday. Information Specialists can help Internet users find information on NCI Web sites and answer questions about cancer.
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For more information from the NCI, please write to this address:
- NCI Public Inquiries Office
- Suite 3036A
- 6116 Executive Boulevard, MSC8322
- Bethesda, MD 20892-8322
Search the NCI Web site
The NCI Web site provides online access to information on cancer, clinical trials, and other Web sites and organizations that offer support and resources for cancer patients and their families. For a quick search, use our “Best Bets” search box in the upper right hand corner of each Web page. The results that are most closely related to your search term will be listed as Best Bets at the top of the list of search results.
There are also many other places to get materials and information about cancer treatment and services. Hospitals in your area may have information about local and regional agencies that have information on finances, getting to and from treatment, receiving care at home, and dealing with problems related to cancer treatment.
Find Publications
The NCI has booklets and other materials for patients, health professionals, and the public. These publications discuss types of cancer, methods of cancer treatment, coping with cancer, and clinical trials. Some publications provide information on tests for cancer, cancer causes and prevention, cancer statistics, and NCI research activities. NCI materials on these and other topics may be ordered online or printed directly from the NCI Publications Locator. These materials can also be ordered by telephone from the Cancer Information Service toll-free at 1-800-4-CANCER (1-800-422-6237), TTY at 1-800-332-8615.
The PDQ cancer information summaries are reviewed regularly and updated as new information becomes available. This section describes the latest changes made to this summary as of the date above.
Images were added to this summary.
PDQ is a comprehensive cancer database available on NCI's Web site.
PDQ is the National Cancer Institute's (NCI's) comprehensive cancer information database. Most of the information contained in PDQ is available online at NCI's Web site. PDQ is provided as a service of the NCI. The NCI is part of the National Institutes of Health, the federal government's focal point for biomedical research.
PDQ contains cancer information summaries.
The PDQ database contains summaries of the latest published information on cancer prevention, detection, genetics, treatment, supportive care, and complementary and alternative medicine. Most summaries are available in two versions. The health professional versions provide detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions provide current and accurate cancer information.
The PDQ cancer information summaries are developed by cancer experts and reviewed regularly.
Editorial Boards made up of experts in oncology and related specialties are responsible for writing and maintaining the cancer information summaries. The summaries are reviewed regularly and changes are made as new information becomes available. The date on each summary ("Date Last Modified") indicates the time of the most recent change.
PDQ also contains information on clinical trials.
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.
Listings of clinical trials are included in PDQ and are available online at NCI's Web site. Descriptions of the trials are available in health professional and patient versions. Many cancer doctors who take part in clinical trials are also listed in PDQ. For more information, call the Cancer Information Service 1-800-4-CANCER (1-800-422-6237); TTY at 1-800-332-8615.

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