Last Modified : 2006-06-30
Normally, the bone marrow makes stem cells (immature cells) that develop into mature blood cells. There are three types of mature blood cells:
Cancers that are "acute" usually get worse quickly if they are not treated. Cancers that are "chronic" usually get worse slowly. Acute myeloid leukemia (AML) is also called acute myelogenous leukemia, acute myeloblastic leukemia, acute granulocytic leukemia, or acute nonlymphocytic leukemia.
In AML, the stem cells usually develop into a type of white blood cell called myeloblasts (or myeloid blasts). The myeloblasts, or leukemia cells, in AML are abnormal and do not mature into healthy white blood cells. These blood cells are unable to do their usual work and 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.
There are subtypes of AML based on the type of blood cell that is affected. The treatment of AML is different when it is a subtype called acute promyelocytic leukemia (APL) or when the child has Down syndrome.
Other myeloid diseases can affect the blood and bone marrow.In chronic myelogenous leukemia (CML), too many bone marrow stem cells develop into a type of white blood cell called granulocytes. Some of these bone marrow stem cells never become mature white blood cells. These are called blasts. Over time, the granulocytes and blasts crowd out the red blood cells and platelets in the bone marrow. CML is rare in children.
Juvenile myelomonocytic leukemiaJuvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children younger than 2 years. In JMML, too many bone marrow stem cells develop into 2 types of white blood cells called myelocytes and monocytes. Some of these bone marrow 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.
Transient myeloproliferative disorderTransient myeloproliferative disorder (TMD) is a disorder of the bone marrow that can develop in newborns who have Down syndrome. This disorder usually goes away on its own within the first 3 weeks of life. Infants who have Down syndrome and TMD have an increased chance of developing AML before the age of 3 years.
Myelodysplastic syndromesIn myelodysplastic syndromes, 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 treatment for myelodysplastic syndromes depends on how much lower than normal the number of red blood cells, white blood cells, or platelets is. Myelodysplastic syndromes may progress to AML.
This summary is about childhood AML, childhood CML, JMML, TMD, and myelodysplastic syndromes. See the following PDQ summaries for more information about other types of leukemia and diseases of the blood and bone marrow:
Anything that increases your risk of getting a disease is called a risk factor. Possible risk factors for childhood AML, childhood CML, JMML, TMD, and myelodysplastic syndrome include the following:
These and other symptoms may be caused by childhood AML, childhood CML, JMML, or myelodysplastic syndromes. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:
The symptoms of TMD may include the following:
The following tests and procedures may be used:
The prognosis (chance of recovery) and treatment options for childhood AML depend on the following:
The prognosis and treatment options for childhood CML depend on how long it has been since the patient was diagnosed and how many blast cells are in the blood.
The prognosis (chance of recovery) and treatment options for JMML depend on the following:
The prognosis (chance of recovery) and treatment options for myelodysplastic syndromes depend on the following:
The extent or spread of cancer is usually described as stages. In childhood 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:
Childhood AML is described as newly diagnosed, in remission, or recurrent.
Newly diagnosed childhood AML
Newly diagnosed childhood AML has not been treated except to relieve symptoms such as fever, bleeding, or pain, and one of the following is true:
or
Childhood AML in remission
In childhood AML in remission, the disease has been treated and the following are true:
Recurrent childhood acute myeloid leukemia (AML) has recurred (come back) after it has been treated. The cancer may come back in the blood and bone marrow or in other parts of the body.
Different types of treatment are available for children with AML, CML, JMML, TMD, or myelodysplastic syndromes. 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 cancer in children is rare, taking part in a clinical trial should be considered. Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI's Web site. Choosing the most appropriate cancer treatment is a decision that ideally involves the patient, family, and health care team.
Children with AML, CML, JMML, TMD, or myelodysplastic syndromes should have their treatment planned by a team of doctors with expertise in treating childhood leukemia and other diseases of the blood.Your child's treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist may refer you to other pediatric doctors who have experience and expertise in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists:
Regular follow-up exams are very important. Some cancer treatments cause side effects that continue or appear years after cancer treatment has ended. These 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 doctors about the possible late effects caused by some treatments. 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) sanctuary therapy may be given during the induction phase of therapy. Because chemotherapy that is given by mouth or injected into a vein may not reach leukemia cells in the CNS (brain and spinal cord), the cells are able to find "sanctuary" (hide) in the CNS. Intrathecal chemotherapy and radiation therapy are able to reach and kill leukemia cells in the CNS and prevent the cancer from recurring (coming back). CNS sanctuary therapy is also called CNS prophylaxis.
Six types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or myelodysplastic syndromes.Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping the cells 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). Combination chemotherapy is treatment using more than one anticancer drug.
The way the chemotherapy is given depends on the type of cancer being treated.
In AML, the leukemia cells may spread to the brain and/or spinal cord. Chemotherapy given by mouth or vein to treat AML cannot cross the blood-brain barrier and enter the fluid that surrounds the brain and spinal cord. Instead, intrathecal chemotherapy is injected into the fluid-filled space to kill leukemia cells that may have spread there.
Radiation therapyRadiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells. 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. External radiation therapy may be used to treat childhood AML that has spread, or may spread, to the brain and spinal cord. When used this way, it is called central nervous system (CNS) sanctuary therapy or CNS prophylaxis.
Stem cell transplantationStem cell transplant is a way 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.
Other drug therapyArsenic 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 (APL).
Imatinib (Gleevec) is a type of anticancer drug called a tyrosine kinase inhibitor. It blocks the enzyme, tyrosine kinase, that causes stem cells to develop into more white blood cells (granulocytes or blasts) than the body needs.
Watchful waitingWatchful waiting is closely monitoring a patient’s condition without giving any treatment until symptoms appear or change. It is sometimes used to treat myelodysplastic syndromes or TMD.
Supportive careSupportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:
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.
Monoclonal antibody therapy is a certain type of biologic therapy. Monoclonal antibody therapy is 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.
Treatment of newly diagnosed childhood acute myeloid leukemia (AML) is combination chemotherapy. CNS sanctuary therapy is intrathecal chemotherapy with or without radiation therapy to the brain.
Treatment of newly diagnosed childhood acute leukemia with a granulocytic sarcoma (chloroma) may include chemotherapy with or without radiation therapy.
Information about ongoing clinical trials is available from the NCI Web site.
Treatment of childhood acute myeloid leukemia (AML) during the remission phase (consolidation/intensification therapy) depends on the subtype of AML and may include the following:
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.
Treatment of recurrent childhood acute myeloid leukemia (AML) may include the following:
Treatment of recurrent acute promyelocytic leukemia may include all-trans retinoic acid (ATRA) or arsenic trioxide therapy
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.
Treatment of acute promyelocytic leukemia may include the following:
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.
Treatment of AML in children who have Down syndrome may include the following:
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.
Treatment for childhood chronic myelogenous leukemia may include the following:
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.
Treatment of juvenile myelomonocytic leukemia may include the following:
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.
Transient myeloproliferative disorder (TMD) usually goes away on its own. For TMD that does not go away on its own, treatment may include the following:
Information about ongoing clinical trials is available from the NCI Web site.
Treatment of myelodysplastic syndromes may include the following:
Supportive care treatments are used to manage problems caused by the disease, such as infection, bleeding, and anemia.
If the myelodysplastic syndrome progresses to acute myeloid leukemia (AML), treatment will be the same as treatment for the newly diagnosed patient with AML.
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.