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

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 ¹. 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 ².

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 ³. 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 ².

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 ⁴. 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 ².

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) ⁵. 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 ².

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 ⁶. 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 ².

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 ⁷. 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 ².

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 ⁸. 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 ².

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 ⁹. 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 ².

Glossary Terms

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.

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

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.

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.

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.

Treatment with drugs that kill cancer cells.

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.

Treatment using more than one anticancer drug.

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

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 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 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.

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.

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.

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

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

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

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.

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.

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.

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

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.

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.

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.

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.

Treatment.

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).

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