General Information About Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Stages of Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Recurrent Childhood Acute Myeloid Leukemia
Treatment Option Overview
Treatment Options for Childhood Acute Myeloid Leukemia, Myelodysplastic Syndromes, and Juvenile Myelomonocytic Leukemia

Newly Diagnosed Childhood Acute Myeloid Leukemia Childhood Acute Myeloid Leukemia in Remission Recurrent Childhood Acute Myeloid Leukemia Acute Promyelocytic Leukemia Recurrent Acute Promyelocytic Leukemia Children with Down Syndrome and AML Childhood Chronic Myelogenous Leukemia Juvenile Myelomonocytic Leukemia Transient Myeloproliferative Disorder Myelodysplastic Syndromes

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Changes to This Summary (03/26/2009)
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General Information About Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

Key Points for This Section

Leukemia and other diseases of the blood and bone marrow may affect red blood cells, white blood cells, and platelets. Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal blood cells. Other myeloid diseases can affect the blood and bone marrow. Chronic myelogenous leukemia Juvenile myelomonocytic leukemia Transient myeloproliferative disorder Myelodysplastic syndromes The risk factors for developing childhood AML, childhood CML, JMML, TMD, and MDS are similar. Possible signs of childhood AML, childhood CML, JMML, or MDS include fever, feeling tired, and easy bleeding or bruising. Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, childhood CML, JMML, TMD, and MDS. Certain factors affect prognosis (chance of recovery) and treatment options.

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 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.

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Blood cell development. A blood stem cell goes through several steps to become a red blood cell, platelet, or white blood cell.

Childhood acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes a large number of abnormal 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 myeloid stem cells usually develop into 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.

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.

Chronic myelogenous leukemia

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 leukemia

Juvenile myelomonocytic leukemia (JMML) is a rare childhood cancer that occurs more often in children around the age of 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 disorder

Transient 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 syndromes

In myelodysplastic syndromes (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 treatment for MDS depends on how much lower than normal the number of red blood cells, white blood cells, or platelets is. MDS may progress to AML.

This summary is about childhood AML, childhood CML, JMML, TMD, and MDS. See the following PDQ summaries for more information about other types of leukemia and diseases of the blood and bone marrow:

• Adult Acute Myeloid Leukemia Treatment ¹
• Chronic Myelogenous Leukemia Treatment ²
• Adult Acute Lymphoblastic Leukemia Treatment ³
• Childhood Acute Lymphoblastic Leukemia Treatment ⁴
• Chronic Lymphocytic Leukemia Treatment ⁵
• Hairy Cell Leukemia Treatment ⁶
• Myelodysplastic Syndromes Treatment ⁷
• Myelodysplastic and Myeloproliferative Diseases Treatment ⁸

The risk factors for developing childhood AML, childhood CML, JMML, TMD, 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. People who think they may be at risk should discuss this with their doctor. Possible risk factors for childhood AML, childhood CML, JMML, TMD, and MDS include the following:

• Having a brother or sister, especially a twin, with leukemia.
• Being Hispanic.
• Being exposed to cigarette smoke or alcohol before birth.
• Having a history of MDS (also called preleukemia) or aplastic anemia.
• Past treatment with chemotherapy or radiation therapy.
• Being exposed to ionizing radiation or chemicals such as benzene.
• Having certain genetic disorders, such as Down syndrome, Fanconi anemia, neurofibromatosis type 1, or Noonan syndrome.

Possible signs of childhood AML, childhood CML, JMML, or MDS include fever, feeling tired, and easy bleeding or bruising.

These and other symptoms may be caused by childhood AML, childhood CML, JMML, or MDS. Other conditions may cause the same symptoms. A doctor should be consulted if any of the following problems occur:

• Fever with or without an infection.
• Night sweats.
• Shortness of breath.
• Weakness or feeling tired.
• Easy bruising or bleeding.
• Petechiae (flat, pinpoint spots under the skin caused by bleeding).
• Pain in the bones or joints.
• Pain or feeling of fullness below the ribs.
• Painless lumps in the neck, underarm, stomach, groin, or other parts of the body. When seen in childhood AML, these lumps, called leukemia cutis, may be blue or purple.
• Painless lumps that are sometimes around the eyes. These lumps, called chloromas, are sometimes seen in childhood AML and may be blue-green.
• An eczema -like skin rash.

The symptoms of TMD may include the following:

• Swelling all over the body.
• Shortness of breath.
• Trouble breathing.
• Weakness or feeling tired.
• Pain below the ribs.

Tests that examine the blood and bone marrow are used to detect (find) and diagnose childhood AML, childhood CML, JMML, TMD, and MDS.

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.

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  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.
• 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.
• Peripheral blood smear: A procedure in which a sample of blood is checked for blast cells, number and kinds of white blood cells, number of platelets, and changes in the shape of the blood cells.
• Biopsy: The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for signs of cancer. Biopsies that may be done for childhood AML include the following:
  • 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.

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    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.

  • Tumor biopsy: A biopsy of a chloroma may be done.
  • Lymph node biopsy: The removal of all or part of a lymph node.
• Cytogenetic analysis: A laboratory test in which 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, that may include special staining of the blood and bone marrow cells. This process is used to diagnose the subtype of AML by comparing the cancer cells to normal cells of the immune system.
• 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.

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  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.

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 age of the child at diagnosis.
• Number of white blood cells in the blood at diagnosis.
• Whether the AML was caused by previous anticancer treatment.
• The subtype of AML.
• Whether there are certain chromosomal changes in the leukemia cells.
• Whether the child has Down syndrome. Most children with AML and Down syndrome can be cured of their leukemia.
• How well the leukemia responds to initial treatment.
• Whether the AML is newly diagnosed or has recurred (come back) after being treated.
• The length of time since treatment ended, for AML that has recurred.

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 age of the child at diagnosis.
• How many red blood cells, white blood cells, or platelets are in the blood.
• Whether the JMML is untreated or has recurred after treatment.

The prognosis (chance of recovery) and treatment options for MDS depend on the following:

• Whether the MDS was caused by previous cancer treatment.
• How low the numbers of red blood cells, white blood cells, or platelets are.
• Whether the MDS is untreated or has recurred after treatment.

Stages of Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

Key Points for This Section

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. There are three ways that cancer spreads in the body. There is no standard staging system for childhood AML, childhood chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).

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 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:

• Lumbar puncture: A procedure used to collect cerebrospinal fluid (CSF) 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.
• Biopsy of the testicles, ovaries, or skin: The removal of cells or tissues from the testicles, ovaries, or skin so they can be viewed under a microscope to check for signs of cancer. This is done only if something unusual about the testicles, ovaries, or skin is found during the physical exam.

There are three ways that cancer spreads in the body.

When cancer cells spread outside the blood, a solid tumor may form. This process is called metastasis. The three ways that cancer cells spread in the body are:

• Through the blood. Cancer cells travel through the blood, invade solid tissues in the body, such as the brain or heart, and form a solid tumor.
• Through the lymph system. Cancer cells invade the lymph system, travel through the lymph vessels, and form a solid tumor in other parts of the body.
• Through solid tissue. Cancer cells that have formed a solid tumor spread to tissues in the surrounding area.

The new (metastatic) tumor is the same type of cancer as the primary cancer. For example, if leukemia cells spread to the brain, the cancer cells in the brain are actually leukemia cells. The disease is metastatic leukemia, not brain cancer.

There is no standard staging system for childhood AML, childhood chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).

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:

• More than 20% of the cells in the bone marrow are blasts (leukemia cells).

  or

• Less than 20% of the cells in the bone marrow are blasts and there is a specific change in the chromosome.

Childhood AML in remission

In childhood AML in remission, the disease has been treated and the following are true:

• The complete blood count is almost 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, spinal cord, or other parts of the body.

Recurrent Childhood Acute Myeloid Leukemia

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.

Treatment Option Overview

Key Points for This Section

There are different types of treatment for children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS). Children with AML, CML, JMML, TMD, or MDS should have their treatment planned by a team of health care providers who are experts in treating childhood leukemia and other diseases of the blood. Some cancer treatments cause side effects months or years after treatment has ended. The treatment of childhood AML usually has two phases. Six types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or MDS. Chemotherapy Radiation therapy Stem cell transplantation Other drug therapy Watchful waiting Supportive care New types of treatment are being tested in clinical trials. Biologic therapy Patients may want to think about taking part in a clinical trial. Patients can enter clinical trials before, during, or after starting their cancer treatment. Follow-up tests may be needed.

There are different types of treatment for children with acute myeloid leukemia (AML), chronic myelogenous leukemia (CML), juvenile myelomonocytic leukemia (JMML), transient myeloproliferative disorder (TMD), or myelodysplastic syndromes (MDS).

Different types of treatment are available for children with AML, CML, JMML, TMD, or 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 cancer in children is rare, taking part in a clinical trial should be considered. Some clinical trials are open only to patients who have not started treatment.

Children with AML, CML, JMML, TMD, or MDS should have their treatment 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 health care providers who are experts in treating children with leukemia and who specialize in certain areas of medicine. These may include the following specialists:

• Hematologist.
• Medical oncologist.
• Pediatric surgeon.
• Radiation oncologist.
• Neurologist.
• Neuropathologist.
• Neuroradiologist.
• Pediatric nurse specialist.
• Social worker.
• Rehabilitation specialist.
• Psychologist.

Some cancer treatments cause side effects months or years after treatment has ended.

Regular follow-up exams are very important. Some cancer treatments cause side effects that continue or appear months or years after cancer treatment has ended. These are called late effects. Late effects of cancer treatment may include:

• Physical problems.
• Changes in mood, feelings, thinking, learning, or memory.
• Second cancers (new types of cancer).

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 effects cancer treatment can have on their child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer ⁹ for more information).

The treatment of childhood AML usually has two phases.

The treatment of childhood AML is done in phases:

• 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.
• Consolidation /intensification therapy: This is the second phase of treatment. It begins once the leukemia is in remission. The purpose of postremission therapy is to kill any remaining leukemia cells that may not be active but could begin to regrow and cause a relapse.

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 MDS.

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 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. Anticancer drugs 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, an anticancer drug is injected into the fluid-filled space to kill leukemia cells that may have spread there. This is called intrathecal chemotherapy.

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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.

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. 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 transplantation

Stem 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 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 (APL).

A type of anticancer drug called a tyrosine kinase inhibitor blocks the enzyme, tyrosine kinase. Tyrosine kinase causes stem cells to develop into more white blood cells (granulocytes or blasts) than the body needs. Imatinib (Gleevec) is a type of tyrosine kinase inhibitor.

Watchful waiting

Watchful waiting is closely monitoring a patient’s condition without giving any treatment until symptoms appear or change. It is sometimes used to treat MDS or TMD.

Supportive care

Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:

• Transfusion therapy: A way of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or cancer treatment. The blood may be donated from another person or it may have been taken from the person earlier and stored until needed.
• Drug therapy, such as antibiotics.
• Leukapheresis: A procedure in which a special machine is used to remove white blood cells from the blood. Blood is taken from the patient and put through a blood cell separator where the white blood cells are removed. The rest of the blood is then returned to the patient's bloodstream.

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 Web site ¹⁰.

Biologic therapy

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.

Natural killer (NK) cells are white blood cells that can kill tumor cells. These may be taken from a donor and given to the patient by infusion to help kill leukemia cells.

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. See the Treatment Options section that follows for links to current treatment clinical trials. These have been retrieved from NCI's clinical trials database.

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. This is sometimes called re-staging.

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.

Treatment Options for Childhood Acute Myeloid Leukemia, Myelodysplastic Syndromes, and Juvenile Myelomonocytic Leukemia

A link to a list of current clinical trials is included for each treatment section. For some types or stages of cancer, there may not be any trials listed. Check with your doctor for clinical trials that are not listed here but may be right for you.

Treatment of newly diagnosed childhood acute myeloid leukemia may include the following:

• Combination chemotherapy plus central nervous system sanctuary therapy (intrathecal chemotherapy with or without radiation therapy to the brain).
• A clinical trial comparing different chemotherapy regimens (doses and schedules of treatment).

Treatment of newly diagnosed childhood acute leukemia with a granulocytic sarcoma (chloroma) may include chemotherapy with or without radiation therapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with untreated childhood acute myeloid leukemia and other myeloid malignancies ¹¹. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

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:

• Combination chemotherapy.
• Stem cell transplant.
• A clinical trial of combination chemotherapy with or without a monoclonal antibody and/or stem cell transplant.
• A clinical trial of a natural killer cell transplant after chemotherapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood acute myeloid leukemia in remission ¹³. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

Treatment of recurrent childhood acute myeloid leukemia may include the following:

• Combination chemotherapy
• Combination chemotherapy and stem cell transplant.
• A clinical trial of a new anticancer drug.
• A clinical trial of biologic therapy.
• A clinical trial of stem cell transplant using different sources of stem cells.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with recurrent childhood acute myeloid leukemia ¹⁴. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

Treatment of acute promyelocytic leukemia may include the following:

• All-trans retinoic acid (ATRA) plus chemotherapy.
• Arsenic trioxide therapy.
• Combination chemotherapy.
• A clinical trial of chemotherapy and ATRA with or without arsenic trioxide.
• A clinical trial of a monoclonal antibody with ATRA.
• A clinical trial of ATRA plus arsenic trioxide.

Supportive care treatments are used to manage problems caused by the disease, such as infection, bleeding, and anemia.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood acute promyelocytic leukemia (M3) ¹⁵. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

Treatment of recurrent acute promyelocytic leukemia may include all-trans retinoic acid or arsenic trioxide therapy.

Treatment of acute myeloid leukemia in children who have Down syndrome may include the following:

• Combination chemotherapy.
• A clinical trial of lower- dose chemotherapy.

Treatment for childhood chronic myelogenous leukemia may include the following:

• Stem cell transplant.
• Drug therapy with Gleevec or other tyrosine kinase inhibitors.
• A clinical trial of stem cell transplant using lower doses of chemotherapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood chronic myelogenous leukemia ¹⁶. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

Treatment of juvenile myelomonocytic leukemia is usually stem cell transplant.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with juvenile myelomonocytic leukemia ¹⁷. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

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:

• Transfusion therapy.
• Leukapheresis.
• Chemotherapy.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with acute myeloid leukemia/transient myeloproliferative disorder ¹⁸. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

Treatment of myelodysplastic syndromes (MDS) may include the following:

• Stem cell transplant.
• Combination chemotherapy.
• A clinical trial of a new anticancer drug.

Supportive care treatments are used to manage problems caused by the disease, such as infection, bleeding, and anemia.

If the MDS progresses to acute myeloid leukemia (AML), treatment will be the same as treatment for the newly diagnosed patient with AML.

Check for U.S. clinical trials from NCI's PDQ Cancer Clinical Trials Registry that are now accepting patients with childhood myelodysplastic syndromes ¹⁹. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. General information about clinical trials is available from the NCI Web site ¹².

To Learn More About Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies

For more information from the National Cancer Institute about childhood acute myeloid leukemia and other myeloid malignancies, see the following:

• What You Need to Know About™ Leukemia ²⁰
• Understanding Cancer Series: Blood Stem Cell Transplants ²¹
• Bone Marrow Transplantation and Peripheral Blood Stem Cell Transplantation: Questions and Answers ²²

For more childhood cancer information and other general cancer resources from the National Cancer Institute, see the following:

• What You Need to Know About™ Cancer - An Overview ²³
• Childhood Cancers ²⁴
• CureSearch - National Childhood Cancer Foundation Children’s Oncology Group ²⁵
• Late Effects of Treatment for Childhood Cancer ⁹
• Young People with Cancer: A Handbook for Parents ²⁶
• Care for Children and Adolescents with Cancer: Questions and Answers ²⁷
• Understanding Cancer Series: Cancer ²⁸
• Staging: Questions and Answers ²⁹
• Coping with Cancer ³⁰
• Support and Resources ³¹
• Cancer Library ³²
• Information for Survivors/Caregivers/Advocates ³³

Get More Information From NCI

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.

Chat online

The NCI's LiveHelp® ³⁴ 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.

Write to us

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 ³⁵ 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 ³⁶. 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.

Changes to This Summary (03/26/2009)

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.

Changes were made to this summary to match those made to the health professional version.

About PDQ

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 ³⁵. 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." In the United States, about two-thirds of children with cancer are treated in a clinical trial at some point in their illness.

Listings of clinical trials are included in PDQ and are available online at NCI's Web site ³⁷. Descriptions of the trials are available in health professional and patient versions. For additional help in locating a childhood cancer clinical trial, call the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237), TTY at 1-800-332-8615.

The PDQ database contains listings of groups specializing in clinical trials.

The Children's Oncology Group (COG) is the major group that organizes clinical trials for childhood cancers in the United States. Information about contacting COG is available on the NCI Web site ³⁵ or from the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237), TTY at 1-800-332-8615.

The PDQ database contains listings of cancer health professionals and hospitals with cancer programs.

Because cancer in children and adolescents is rare, the majority of children with cancer are treated by health professionals specializing in childhood cancers, at hospitals or cancer centers with special facilities to treat them. The PDQ database contains listings of health professionals who specialize in childhood cancer and listings of hospitals with cancer programs. For help locating childhood cancer health professionals or a hospital with cancer programs, call the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237), TTY at 1-800-332-8615.

Glossary Terms

The area of the body that contains the pancreas, stomach, intestines, liver, gallbladder, and other organs.

Not normal. An abnormal lesion or growth may be cancer, premalignant (likely to become cancer), or benign (not cancer).

Symptoms or signs that begin and worsen quickly; not chronic.

An aggressive (fast-growing) disease in which too many myeloblasts (immature white blood cells that are not lymphoblasts) are found in the bone marrow and blood. Also called acute myeloblastic leukemia, acute myelogenous leukemia, acute nonlymphocytic leukemia, AML, and ANLL.

An aggressive (fast-growing) type of acute myeloid leukemia in which there are too many immature blood-forming cells in the blood and bone marrow. It is usually marked by an exchange of parts of chromosomes 15 and 17. Also called APL and promyelocytic leukemia.

A nutrient that that body needs in small amounts to function and stay healthy. All-trans retinoic acid is made in the body from vitamin A and helps cells to grow and develop, especially in the embryo. A form of all-trans retinoic acid made in the laboratory is put on the skin to treat conditions such as acne and is taken by mouth to treat acute promyelocytic leukemia (a fast-growing cancer in which there are too many immature blood-forming cells in the blood and bone marrow). All-trans retinoic acid is being studied in the prevention and treatment of other types of cancer. Also called ATRA, retinoic acid, tretinoin, and vitamin A acid.

An aggressive (fast-growing) disease in which too many myeloblasts (immature white blood cells that are not lymphoblasts) are found in the bone marrow and blood. Also called acute myeloblastic leukemia, acute myelogenous leukemia, acute myeloid leukemia, acute nonlymphocytic leukemia, and ANLL.

A condition in which the number of red blood cells is below normal.

A drug used to treat infections caused by bacteria and other microorganisms.

A type of protein made by plasma cells (a type of white blood cell) in response to an antigen (foreign substance). Each antibody can bind to only one specific antigen. The purpose of this binding is to help destroy the antigen. Antibodies can work in several ways, depending on the nature of the antigen. Some antibodies destroy antigens directly. Others make it easier for white blood cells to destroy the antigen.

A substance that causes the immune system to make a specific immune response.

A condition in which the bone marrow is unable to produce blood cells.

A drug used to treat acute promyelocytic leukemia (APL) that has not gotten better or that has come back after treatment with other anticancer drugs. It is also being studied in the treatment of other types of cancer. Also called Trisenox.

A chemical that is used widely by the chemical industry, and is also found in tobacco smoke, vehicle emissions, and gasoline fumes. Exposure to benzene may increase the risk of developing leukemia.

Treatment to boost or restore the ability of the immune system to fight cancer, infections, and other diseases. Also used to lessen certain side effects that may be caused by some cancer treatments. Agents used in biological therapy include monoclonal antibodies, growth factors, and vaccines. These agents may also have a direct antitumor effect. Also called biological response modifier therapy, biotherapy, BRM therapy, and immunotherapy.

The removal of cells or tissues for examination by a pathologist. The pathologist may study the tissue under a microscope or perform other tests on the cells or tissue. There are many different types of biopsy procedures. The most common types include: (1) incisional biopsy, in which only a sample of tissue is removed; (2) excisional biopsy, in which an entire lump or suspicious area is removed; and (3) needle biopsy, in which a sample of tissue or fluid is removed with a needle. When a wide needle is used, the procedure is called a core biopsy. When a thin needle is used, the procedure is called a fine-needle aspiration biopsy.

An immature blood cell.

A tissue with red blood cells, white blood cells, platelets, and other substances suspended in fluid called plasma. Blood takes oxygen and nutrients to the tissues, and carries away wastes.

A procedure in which a sample of blood is examined to measure the amounts of certain substances made in the body. An abnormal amount of a substance can be a sign of disease in the organ or tissue that produces it.

A mass of blood that forms when blood platelets, proteins, and cells stick together. When a blood clot is attached to the wall of a blood vessel, it is called a thrombus. When it moves through the bloodstream and blocks the flow of blood in another part of the body, it is called an embolus.

A network of blood vessels with closely spaced cells that makes it difficult for potentially toxic substances (such as anticancer drugs) to penetrate the blood vessel walls and enter the brain. Also called BBB.

The soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.

The removal of a small sample of bone marrow (usually from the hip) through a needle for examination under a microscope.

The removal of a sample of tissue from the bone marrow with a needle for examination under a microscope.

A term for diseases in which abnormal cells divide without control and can invade nearby tissues. Cancer cells can also spread to other parts of the body through the blood and lymph systems. There are several main types of cancer. Carcinoma is cancer that begins in the skin or in tissues that line or cover internal organs. Sarcoma is cancer that begins in bone, cartilage, fat, muscle, blood vessels, or other connective or supportive tissue. Leukemia is cancer that starts in blood-forming tissue such as the bone marrow, and causes large numbers of abnormal blood cells to be produced and enter the blood. Lymphoma and multiple myeloma are cancers that begin in the cells of the immune system. Central nervous system cancers are cancers that begin in the tissues of the brain and spinal cord.

A flexible tube used to deliver fluids into or withdraw fluids from the body.

A hollow area or hole. It may describe a body cavity (such as the space within the abdomen) or a hole in a tooth caused by decay.

The individual unit that makes up the tissues of the body. All living things are made up of one or more cells.

The brain and spinal cord. Also called CNS.

Chemotherapy or radiation therapy given to the central nervous system (CNS) as a preventive treatment. It kills cancer cells that may be in the brain and spinal cord, even though no cancer has been detected there. Also called central nervous system prophylaxis, CNS prophylaxis, and CNS sanctuary therapy.

The fluid that flows in and around the hollow spaces of the brain and spinal cord, and between two of the meninges (the thin layers of tissue that cover and protect the brain and spinal cord). Cerebrospinal fluid is made by tissue called the choroid plexus in the ventricles (hollow spaces) in the brain. Also called CSF.

Treatment with drugs that kill cancer cells.

An x-ray of the structures inside the chest. An x-ray is a type of high-energy radiation that can go through the body and onto film, making pictures of areas inside the chest, which can be used to diagnose disease.

A malignant, green-colored tumor of myeloid cells (a type of immature white blood cell). This tumor is usually associated with myelogenous leukemia. Also called granulocytic sarcoma.

Part of a cell that contains genetic information. Except for sperm and eggs, all human cells contain 46 chromosomes.

A disease or condition that persists or progresses over a long period of time.

A slowly progressing disease in which too many white blood cells (not lymphocytes) are made in the bone marrow. Also called chronic granulocytic leukemia, chronic myeloid leukemia, and CML.

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.

A slowly progressing disease in which too many white blood cells (not lymphocytes) are made in the bone marrow. Also called chronic granulocytic leukemia, chronic myelogenous leukemia, and chronic myeloid leukemia.

The brain and spinal cord. Also called central nervous system.

Chemotherapy or radiation therapy given to the central nervous system (CNS) as a preventive treatment. It kills cancer cells that may be in the brain and spinal cord, even though no cancer has been detected there. Also called central nervous system prophylaxis, central nervous system sanctuary therapy, and CNS prophylaxis.

Treatment using more than one anticancer drug.

A test to check the number of red blood cells, white blood cells, and platelets in a sample of blood. Also called blood cell count and CBC.

Treatment that is given after cancer has disappeared following the initial therapy. Consolidation therapy is used to kill any cancer cells that may be left in the body. It may include radiation therapy, a stem cell transplant, or treatment with drugs that kill cancer cells. Also called intensification therapy and postremission therapy.

The study of chromosomes and chromosomal abnormalities.

The process of identifying a disease, such as cancer, from its signs and symptoms.

In medicine, a disturbance of normal functioning of the mind or body. Disorders may be caused by genetic factors, disease, or trauma.

The amount of medicine taken, or radiation given, at one time.

A disorder caused by the presence of an extra chromosome 21 and characterized by mental retardation and distinguishing physical features.

Any substance, other than food, that is used to prevent, diagnose, treat or relieve symptoms of a disease or abnormal condition. Also refers to a substance that alters mood or body function, or that can be habit-forming or addictive, especially a narcotic.

A group of conditions in which the skin becomes inflamed, forms blisters, and becomes crusty, thick, and scaly. Eczema causes burning and itching, and may occur over a long period of time. Atopic dermatitis is the most common type of eczema.

A protein that speeds up chemical reactions in the body.

A type of radiation therapy that uses a machine to aim high-energy rays at the cancer from outside of the body. Also called external-beam radiation therapy.

A rare inherited disorder in which the bone marrow does not make blood cells. It is usually diagnosed in children between 2 and 15 years old. Symptoms include frequent infections, easy bleeding, and extreme tiredness. People with Fanconi anemia may have a small skeleton and brown spots on the skin. They also have an increased risk of developing certain types of cancer.

An increase in body temperature above normal (98.6 degrees F), usually caused by disease.

A substance that flows smoothly and takes the shape of its container. Liquids and gases are fluids.

Monitoring a person's health over time after treatment. This includes keeping track of the health of people who participate in a clinical study or clinical trial for a period of time, both during the study and after the study ends.

Inherited; having to do with information that is passed from parents to offspring through genes in sperm and egg cells.

A drug used to treat different types of leukemia and other cancers of the blood, gastrointestinal stromal tumors, skin tumors called dermatofibrosarcoma protuberans, and a rare condition called systemic mastocytosis. It is also being studied in the treatment of other types of cancer. Gleevec blocks the protein made by the bcr/abl oncogene. It is a type of tyrosine kinase inhibitor. Also called imatinib mesylate and STI571.

A type of immune cell that has granules (small particles) with enzymes that are released during infections, allergic reactions, and asthma. Neutrophils, eosinophils, and basophils are granulocytes. A granulocyte is a type of white blood cell. Also called granular leukocyte, PMN, and polymorphonuclear leukocyte.

A malignant, green-colored tumor of myeloid cells (a type of immature white blood cell). This tumor is usually associated with myelogenous leukemia. Also called chloroma.

The area where the thigh meets the abdomen.

A doctor who specializes in treating blood disorders.

The substance inside red blood cells that binds to oxygen in the lungs and carries it to the tissues.

A drug used to treat different types of leukemia and other cancers of the blood, gastrointestinal stromal tumors, skin tumors called dermatofibrosarcoma protuberans, and a rare condition called systemic mastocytosis. It is also being studied in the treatment of other types of cancer. Imatinib mesylate blocks the protein made by the bcr/abl oncogene. It is a type of tyrosine kinase inhibitor. Also called Gleevec and STI571.

The complex group of organs and cells that defends the body against infections and other diseases.

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 specific types of leukemia and lymphoma by comparing the cancer cells to normal cells of the immune system.

Initial treatment used to reduce a cancer. Induction therapy is followed by other treatments, such as chemotherapy, radiation therapy, and hormone therapy to get rid of cancer that remains. Also called first-line therapy, primary therapy, and primary treatment.

Invasion and multiplication of germs in the body. Infections can occur in any part of the body and can spread throughout the body. The germs may be bacteria, viruses, yeast, or fungi. They can cause a fever and other problems, depending on where the infection occurs. When the body’s natural defense system is strong, it can often fight the germs and prevent infection. Some cancer treatments can weaken the natural defense system.

A method of putting fluids, including drugs, into the bloodstream. Also called intravenous infusion.

Use of a syringe and needle to push fluids or drugs into the body; often called a "shot."

Treatment that is given after cancer has disappeared following the initial therapy. Intensification therapy is used to kill any cancer cells that may be left in the body. It may include radiation therapy, a stem cell transplant, or treatment with drugs that kill cancer cells. Also called consolidation therapy and postremission therapy.

A type of radiation therapy in which radioactive material sealed in needles, seeds, wires, or catheters is placed directly into or near a tumor. Also called brachytherapy, implant radiation therapy, and radiation brachytherapy.

Treatment in which anticancer drugs are injected into the fluid-filled space between the thin layers of tissue that cover the brain and spinal cord.

A type of radiation made (or given off ) by x-ray procedures, radioactive substances, rays that enter the Earth's atmosphere from outer space, and other sources. At high doses, ionizing radiation increases chemical activity inside cells and can lead to health risks, including cancer.

A rare form of childhood leukemia in which cancer cells often spread into tissues such as the skin, lung, and intestines. Also called juvenile myelomonocytic leukemia.

A rare form of childhood leukemia in which cancer cells often spread into tissues such as the skin, lung, and intestines. Also called JMML.

Side effects of cancer treatment that appear months or years after treatment has ended. Late effects include physical and mental problems and second cancers.

Removal of the blood to collect specific blood cells. The remaining blood is returned to the body.

Cancer that starts in blood-forming tissue such as the bone marrow and causes large numbers of blood cells to be produced and enter the bloodstream.

A procedure in which a thin needle called a spinal needle is put into the lower part of the spinal column to collect cerebrospinal fluid or to give drugs. Also called spinal tap.

A rounded mass of lymphatic tissue that is surrounded by a capsule of connective tissue. Lymph nodes filter lymph (lymphatic fluid), and they store lymphocytes (white blood cells). They are located along lymphatic vessels. Also called lymph gland.

A thin tube that carries lymph (lymphatic fluid) and white blood cells through the lymphatic system. Also called lymphatic vessel.

The tissues and organs that produce, store, and carry white blood cells that fight infections and other diseases. This system includes the bone marrow, spleen, thymus, lymph nodes, and lymphatic vessels (a network of thin tubes that carry lymph and white blood cells). Lymphatic vessels branch, like blood vessels, into all the tissues of the body.

Referring to lymphocytes, a type of white blood cell. Also refers to tissue in which lymphocytes develop.

A diagnostic indication that disease may develop.

A doctor who specializes in diagnosing and treating cancer using chemotherapy, hormonal therapy, and biological therapy. A medical oncologist often is the main health care provider for someone who has cancer. A medical oncologist also gives supportive care and may coordinate treatment given by other specialists.

Refers to the practices and procedures used for the prevention, treatment, or relief of symptoms of a diseases or abnormal conditions. This term may also refer to a legal drug used for the same purpose.

The spread of cancer from one part of the body to another. A tumor formed by cells that have spread is called a “metastatic tumor” or a “metastasis.” The metastatic tumor contains cells that are like those in the original (primary) tumor. The plural form of metastasis is metastases (meh-TAS-tuh-SEEZ).

Having to do with metastasis, which is the spread of cancer from one part of the body to another.

A type of protein made in the laboratory that can locate and bind to substances in the body, including tumor cells. There are many kinds of monoclonal antibodies. Each monoclonal antibody is made to find one substance. Monoclonal antibodies are being used to treat some types of cancer and are being studied in the treatment of other types. They can be used alone or to carry drugs, toxins, or radioactive materials directly to a tumor.

A type of immune cell that is made in the bone marrow and travels through the blood to tissues in the body where it becomes a macrophage. Macrophages surround and kill microorganisms, ingest foreign material, remove dead cells, and boost immune responses. A monocyte is a type of white blood cell and a type of phagocyte.

A group of diseases in which the bone marrow does not make enough healthy blood cells. Also called preleukemia and smoldering leukemia.

Having to do with or resembling the bone marrow. May also refer to certain types of hematopoietic (blood-forming) cells found in the bone marrow. Sometimes used as a synonym for myelogenous; for example, acute myeloid leukemia and acute myelogenous leukemia are the same disease.

A group of slow growing blood cancers, including chronic myelogenous leukemia, in which large numbers of abnormal red blood cells, white blood cells, or platelets grow and spread in the bone marrow and the peripheral blood.

The National Cancer Institute, part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. The National Cancer Institute conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the National Cancer Institute Web site at http://www.cancer.gov. Also called NCI.

A type of immune cell that has granules (small particles) with enzymes that can kill tumor cells or cells infected with a virus. A natural killer cell is a type of white blood cell. Also called NK cell and NK-LGL.

NCI, part of the National Institutes of Health of the United States Department of Health and Human Services, is the Federal Government's principal agency for cancer research. It conducts, coordinates, and funds cancer research, training, health information dissemination, and other programs with respect to the cause, diagnosis, prevention, and treatment of cancer. Access the NCI Web site at http://www.cancer.gov. Also called National Cancer Institute.

A rare genetic condition that causes brown spots and tumors on the skin, freckling in skin areas not exposed to the sun, tumors on the nerves, and developmental changes in the nervous system, muscles, bone, and skin. Also called NF1.

A doctor who specializes in the diagnosis and treatment of disorders of the nervous system.

A pathologist who specializes in diseases of the nervous system. A pathologist identifies disease by studying cells and tissues under a microscope.

A doctor trained in radiology who specializes in creating and interpreting pictures of the nervous system. The pictures are produced using forms of radiation, such as x-rays, sound waves, or other types of energy.

A doctor who specializes in treating cancer. Some oncologists specialize in a particular type of cancer treatment. For example, a radiation oncologist specializes in treating cancer with radiation.

A part of the body that performs a specific function. For example, the heart is an organ.

One of a pair of female reproductive glands in which the ova, or eggs, are formed. The ovaries are located in the pelvis, one on each side of the uterus.

A colorless, odorless gas. It is needed for animal and plant life. Oxygen that is breathed in enters the blood from the lungs and travels to the tissues.

A doctor who identifies diseases by studying cells and tissues under a microscope.

PDQ is an online database developed and maintained by the National Cancer Institute. Designed to make the most current, credible, and accurate cancer information available to health professionals and the public, PDQ contains peer-reviewed summaries on cancer treatment, screening, prevention, genetics, complementary and alternative medicine, and supportive care; a registry of cancer clinical trials from around the world; and directories of physicians, professionals who provide genetics services, and organizations that provide cancer care. Most of this information, and more specific information about PDQ, can be found on the NCI's Web site at http://www.cancer.gov/cancertopics/pdq. Also called Physician Data Query.

Having to do with children.

A registered nurse with an advanced degree in nursing who specializes in the care of children.

A surgeon who specializes in the treatment of children. A surgeon removes or repairs a part of the body by operating on the patient.

A procedure in which a sample of blood is viewed under a microscope to count different circulating blood cells (red blood cells, white blood cells, platelets, etc.) and see whether the cells look normal.

Pinpoint, unraised, round red spots under the skin caused by bleeding.

An exam of the body to check for general signs of disease.

A tiny piece of a cell found in the blood that breaks off from a large cell found in the bone marrow. Platelets help wounds heal and prevent bleeding by forming blood clots. Also called thrombocyte.

Treatment that is given after cancer has disappeared following the initial therapy. Postremission therapy is used to kill any cancer cells that may be left in the body. It may include radiation therapy, a stem cell transplant, or treatment with drugs that kill cancer cells. Also called consolidation therapy and intensification therapy.

A group of diseases in which the bone marrow does not make enough healthy blood cells. Also called myelodysplastic syndromes and smoldering leukemia.

The original tumor.

The likely outcome or course of a disease; the chance of recovery or recurrence.

A molecule made up of amino acids that are needed for the body to function properly. Proteins are the basis of body structures such as skin and hair and of substances such as enzymes, cytokines, and antibodies.

A specialist who can talk with patients and their families about emotional and personal matters, and can help them make decisions.

Energy released in the form of particle or electromagnetic waves. Common sources of radiation include radon gas, cosmic rays from outer space, medical x-rays, and energy given off by a radioisotope (unstable form of a chemical element that releases radiation as it breaks down and becomes more stable).

A doctor who specializes in using radiation to treat cancer.

The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called irradiation and radiotherapy.

Giving off radiation.

A small, radioactive pellet that is placed in or near a tumor. Cancer cells are killed by the energy given off as the radioactive material breaks down and becomes more stable.

To come back or to return.

Cancer that has recurred (come back), usually after a period of time during which the cancer could not be detected. The cancer may come back to the same place as the original (primary) tumor or to another place in the body. Also called recurrence.

A cell that carries oxygen to all parts of the body. Also called erythrocyte and RBC.

A treatment plan that specifies the dosage, the schedule, and the duration of treatment.

Treatment with anticancer drugs directed to a specific area of the body.

A healthcare professional who helps people recover from an illness or injury and return to daily life. Examples of rehabilitation specialists are physical therapists and occupational therapists.

The return of signs and symptoms of cancer after a period of improvement.

A decrease in or disappearance of signs and symptoms of cancer. In partial remission, some, but not all, signs and symptoms of cancer have disappeared. In complete remission, all signs and symptoms of cancer have disappeared, although cancer still may be in the body.

Something that may increase the chance of developing a disease. Some examples of risk factors for cancer include age, a family history of certain cancers, use of tobacco products, certain eating habits, obesity, lack of exercise, exposure to radiation or other cancer-causing agents, and certain genetic changes.

Refers to a new primary cancer in a person with a history of cancer.

A problem that occurs when treatment affects healthy tissues or organs. Some common side effects of cancer treatment are fatigue, pain, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

A professional trained to talk with people and their families about emotional or physical needs, and to find them support services.

An abnormal mass of tissue that usually does not contain cysts or liquid areas. Solid tumors may be benign (not cancer), or malignant (cancer). Different types of solid tumors are named for the type of cells that form them. Examples of solid tumors are sarcomas, carcinomas, and lymphomas. Leukemias (cancers of the blood) generally do not form solid tumors.

The bones, muscles, tendons, and other tissues that reach from the base of the skull to the tailbone. The spinal column encloses the spinal cord and the fluid surrounding the spinal cord. Also called backbone, spine, and vertebral column.

A column of nerve tissue that runs from the base of the skull down the back. It is surrounded by three protective membranes, and is enclosed within the vertebrae (back bones). The spinal cord and the brain make up the central nervous system, and spinal cord nerves carry most messages between the brain and the rest of the body.

The extent of a cancer in the body. Staging is usually based on the size of the tumor, whether lymph nodes contain cancer, and whether the cancer has spread from the original site to other parts of the body.

In medicine, treatment that experts agree is appropriate, accepted, and widely used. Health care providers are obligated to provide patients with standard therapy. Also called best practice and standard of care.

A cell from which other types of cells develop. For example, blood cells develop from blood-forming stem cells.

A method of replacing immature blood-forming cells that were destroyed by cancer treatment. The stem cells are given to the person after treatment to help the bone marrow recover and continue producing healthy blood cells.

An organ that is part of the digestive system. The stomach helps digest food by mixing it with digestive juices and churning it into a thin liquid.

Care given to improve the quality of life of patients who have a serious or life-threatening disease. The goal of supportive care is to prevent or treat as early as possible the symptoms of a disease, side effects caused by treatment of a disease, and psychological, social, and spiritual problems related to a disease or its treatment. Also called comfort care, palliative care, and symptom management.

An indication that a person has a condition or disease. Some examples of symptoms are headache, fever, fatigue, nausea, vomiting, and pain.

Treatment with anticancer drugs that travel through the blood to cells all over the body.

One of two egg-shaped glands inside the scrotum that produce sperm and male hormones. Also called testis.

Treatment.

A group or layer of cells that work together to perform a specific function.

A poison produced by certain animals, plants, or bacteria.

The infusion of components of blood or whole blood into the bloodstream. The blood may be donated from another person, or it may have been taken from the person earlier and stored until needed.

A surgical procedure in which tissue or an organ is transferred from one area of a person’s body to another area, or from one person (the donor) to another person (the recipient).

An abnormal mass of tissue that results when cells divide more than they should or do not die when they should. Tumors may be benign (not cancer), or malignant (cancer). Also called neoplasm.

A drug that interferes with cell communication and growth and may prevent tumor growth. Some tyrosine kinase inhibitors are used to treat cancer.

A blood vessel that carries blood to the heart from tissues and organs in the body.

Closely monitoring a patient's condition but withholding treatment until symptoms appear or change. Also called active surveillance, expectant management, and observation.

A type of immune cell. Most white blood cells are made in the bone marrow and are found in the blood and lymph tissue. White blood cells help the body fight infections and other diseases. Granulocytes, monocytes, and lymphocytes are white blood cells. Also called leukocyte and WBC.

A type of high-energy radiation. In low doses, x-rays are used to diagnose diseases by making pictures of the inside of the body. In high doses, x-rays are used to treat cancer.