Home > Patients & Visitors > Health Library > Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Patient Information [NCI]
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Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells.
Childhood acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, and acute nonlymphocytic leukemia. Cancers that are acute usually get worse quickly if they are not treated. Cancers that are chronic usually get worse slowly.
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 and other diseases of the blood and bone marrow 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, or leukemia cells, in AML are abnormal and do not become healthy white blood cells. The leukemia cells can build up in the blood and bone marrow 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 granulocytic sarcoma or chloroma.
Other myeloid diseases can affect the blood and bone marrow.
Transient abnormal myelopoiesis (TAM)
TAM is a disorder of the bone marrow that can develop in newborns who have Down syndrome. It usually goes away on its own within the first 3 months of life. Infants who have TAM have an increased chance of developing AML before the age of 3 years. TAM is also called transient myeloproliferative disorder or transient leukemia.
Acute promyelocytic leukemia (APL)
APL is a subtype of AML. In APL, some 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. The promyelocytes (leukemia cells) can build up in the blood and bone marrow so there is less room for healthy white blood cells, red blood cells, and platelets. Problems with severe bleeding and blood clots may also occur. This is a serious health problem that needs treatment as soon as possible.
Juvenile myelomonocytic leukemia (JMML)
JMML is a rare childhood cancer that is most common in children around the age of 2 years and is more common in boys. In JMML, too many myeloid blood stem cells become myelocytes and monocytes (two types of white blood cells). Some of these myeloid blood stem cells never become mature white blood cells. These immature cells, called blasts, are unable to do their usual work. Over time, the myelocytes, monocytes, and blasts crowd out the red blood cells and platelets in the bone marrow. When this happens, infection, anemia, or easy bleeding may occur.
Chronic myelogenous leukemia (CML)
CML often begins in an early myeloid blood cell when a certain gene change occurs. A section of genes, that includes the ABL gene, on chromosome 9 changes place with a section of genes on chromosome 22, which has the BCR gene. This makes a very short chromosome 22 (called the Philadelphia chromosome) and a very long chromosome 9. An abnormal BCR-ABL gene is formed on chromosome 22. The BCR-ABL gene tells the blood cells to make too much of a protein called tyrosine kinase. Tyrosine kinase causes too many white blood cells (leukemia cells) to be made in the bone marrow. The leukemia cells can build up in the blood and bone marrow 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. CML is rare in children.Philadelphia chromosome. A piece of chromosome 9 and a piece of chromosome 22 break off and trade places. The BCR-ABL gene is formed on chromosome 22 where the piece of chromosome 9 attaches. The changed chromosome 22 is called the Philadelphia chromosome.
Myelodysplastic syndromes (MDS)
MDS occur less often in children than in adults. In MDS, the bone marrow makes too few red blood cells, white blood cells, and platelets. These blood cells may not mature and enter the blood. The type of MDS depends on the type of blood cell that is affected.
The treatment for MDS depends on how low the numbers of red blood cells, white blood cells, or platelets are. Over time, MDS may become AML.
This summary is about childhood AML, TAM, childhood APL, JMML, childhood CML, and childhood MDS. See the Childhood Acute Lymphoblastic Leukemia Treatment summary for information about the treatment of childhood acute lymphoblastic leukemia.
AML or MDS may occur after treatment with certain chemotherapy drugs and/or radiation therapy.
Cancer treatment with certain chemotherapy drugs and/or radiation therapy may cause therapy-related AML (t-AML) or therapy -related MDS (t-MDS). The risk of these therapy-related myeloid diseases depends on the total dose of the chemotherapy drugs used and the radiation dose and treatment field. Some patients also have an inherited risk for t-AML and t-MDS. These therapy-related diseases usually occur within 7 years after treatment, but are rare in children.
The risk factors for childhood AML, APL, JMML, CML, and MDS are similar.
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 child's doctor if you think your child may be at risk. These and other factors may increase the risk of childhood AML, APL, JMML, CML, and MDS:
Signs and symptoms of childhood AML, APL, JMML, CML, or MDS include fever, feeling tired, and easy bleeding or bruising.
These and other signs and symptoms may be caused by childhood AML, APL, JMML, CML, or MDS or by other conditions. Check with a doctor if your child has any of the following:
The signs and symptoms of TAM may include the following:
Sometimes TAM does not cause any symptoms at all and is diagnosed after a routine blood test.
Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, TAM, APL, JMML, CML, and MDS.
The following tests and procedures may be used:
The following test is a type of cytogenetic analysis:
Certain factors affect prognosis (chance of recovery) and treatment options.
The prognosis (chance of recovery) and treatment options for childhood AML depend on the following:
The prognosis for childhood APL depends on the following:
The prognosis and treatment options for JMML depend on the following:
The prognosis and treatment options for childhood CML depend on the following:
The prognosis and treatment options for MDS depend on the following:
Once childhood 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 following tests and procedures may be used to determine if the leukemia has spread from the blood to other parts of the body:
There is no standard staging system for childhood AML, childhood acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), childhood chronic myelogenous leukemia (CML), or myelodysplastic syndromes (MDS).
The extent or spread of cancer is usually described as stages. Instead of stages, treatment of childhood AML, childhood APL, JMML, childhood CML, and MDS is based on one or more of the following:
Newly diagnosed childhood AML
Newly diagnosed childhood AML has not been treated except to relieve signs and symptoms such as fever, bleeding, or pain, and one of the following is found:
Childhood AML in remission
In childhood AML in remission, the disease has been treated and the following are found:
Recurrent childhood AML has come back after it has been treated.
In recurrent childhood AML, the cancer may come back in the blood and bone marrow or in other parts of the body, such as the central nervous system (brain and spinal cord).
In refractory childhood AML, the cancer does not respond to treatment.
There are different types of treatment for children with AML, TAM, APL, JMML, CML, and MDS.
Different types of treatment are available for children with acute myeloid leukemia (AML), transient abnormal myelopoiesis (TAM), acute promyelocytic leukemia (APL), juvenile myelomonocytic leukemia (JMML), chronic myelogenous leukemia (CML), and myelodysplastic syndromes (MDS). 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.
Because AML and other myeloid disorders are rare in children, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not yet started treatment.
Treatment is planned by a team of health care providers who are experts in treating childhood leukemia and other diseases of the blood.
Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other healthcare providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists:
Treatment for childhood acute myeloid leukemia may cause side effects.
For information about side effects that begin during treatment for cancer, see our Side Effects page.
Regular follow-up exams are very important. Side effects from cancer treatment that begin after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following:
Some late effects may be treated or controlled. It is important that parents of children who are treated for AML or other blood diseases talk with their child's doctors about the effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information).
The treatment of childhood AML usually has two phases.
The treatment of childhood AML is done in phases:
Treatment called central nervous system (CNS) prophylaxis therapy may be given during the induction phase of therapy. Because standard doses of chemotherapy may not reach leukemia cells in the CNS (brain and spinal cord), the leukemia cells are able to hide in the CNS. Intrathecal chemotherapy is able to reach leukemia cells in the CNS. It is given to kill the leukemia cells and lessen the chance the leukemia will recur (come back).
Seven types of standard treatment are used for childhood AML, TAM, childhood APL, JMML, childhood CML, and MDS.
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). Combination chemotherapy is treatment using more than one chemotherapy drug.
The way the chemotherapy is given depends on the type of cancer being treated. In AML, chemotherapy given by mouth, vein, or into the cerebrospinal fluid is used.
In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML may not cross the blood-brain barrier to get into the fluid that surrounds the brain and spinal cord. Instead, chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there (intrathecal chemotherapy).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. There are two types of radiation therapy:
The way the radiation therapy is given depends on the type of the cancer being treated. In childhood AML, external radiation therapy may be used to treat a chloroma that does not respond to chemotherapy.
Stem cell transplant
Chemotherapy is given to kill cancer cells or other abnormal blood 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, 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 without harming normal cells. Types of targeted therapy include the following:
Selinexor is a targeted therapy drug that is being studied to treat refractory or recurrent childhood AML.
See Drugs Approved for Leukemia for more information.
Other drug therapy
Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndromes caused by a specific chromosome change. It is also being studied in the treatment of children with recurrent and refractory AML.
Arsenic trioxide and all-trans retinoic acid (ATRA) are drugs that kill certain types of 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 acute promyelocytic leukemia.
Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change. It is sometimes used to treat MDS or transient abnormal myelopoiesis (TAM).
Supportive care is given to lessen the problems caused by the disease or its treatment. All patients with leukemia receive supportive care treatments. Supportive care may include the following:
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.
Immunotherapy 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 biological therapy.
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 child's 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 Treatment Option Overview section.
Treatment of newly diagnosed childhood acute myeloid leukemia (AML) during the induction phase may include the following:
Treatment of childhood AML during the remission phase (consolidation /intensification therapy) depends on the subtype of AML and may include the following:
Treatment of refractory childhood AML may include the following:
Treatment of recurrent childhood AML may include the following:
Transient abnormal myelopoiesis (TAM) usually goes away on its own. For TAM that does not go away on its own or causes other health problems, treatment may include the following:
Treatment of acute myeloid leukemia (AML) in children aged 4 years or younger who have Down syndrome may include the following:
Treatment of AML in children older than 4 years who have Down syndrome may be the same as treatment for children without Down syndrome.
Treatment of newly diagnosed childhood acute promyelocytic leukemia (APL) may include the following:
Treatment of childhood APL during the remission phase (consolidation /intensification therapy) may include the following:
Treatment of recurrent childhood APL may include the following:
Treatment of juvenile myelomonocytic leukemia (JMML) may include the following:
Treatment of refractory or recurrent childhood JMML may include the following:
Treatment for childhood chronic myelogenous leukemia (CML) may include the following:
Treatment of refractory or recurrent childhood CML may include the following:
Treatment of childhood myelodysplastic syndromes (MDS) may include the following:
If the MDS becomes acute myeloid leukemia (AML), treatment will be the same as treatment for newly diagnosed AML.
For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following:
For more childhood cancer information and other general cancer resources, see the following:
Physician Data Query (PDQ) is the National Cancer Institute's (NCI's) comprehensive cancer information database. 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 come in two versions. The health professional versions have detailed information written in technical language. The patient versions are written in easy-to-understand, nontechnical language. Both versions have cancer information that is accurate and up to date and most versions are also available in Spanish.
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Purpose of This Summary
This PDQ cancer information summary has current information about the treatment of childhood acute myeloid leukemia and other myeloid malignancies. 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® Pediatric Treatment Editorial Board. PDQ Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/leukemia/patient/child-aml-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389303]
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Last Revised: 2019-07-23
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