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. 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. The treatment of childhood AML usually has two phases. Six types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or myelodysplastic syndromes. 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.

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. Some clinical trials are open only to patients who have not started treatment.

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.

Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric doctors 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.

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:

• Physical problems.
• Changes in mood, feelings, thinking, learning, or memory.
• Having a new type 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 possible late effects caused by some treatments. See the PDQ summary on Late Effects of Treatment for Childhood Cancer ¹ 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 myelodysplastic syndromes.

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.

Enlarge

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).

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 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 myelodysplastic syndromes 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 ².

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.

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.

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 cancerous, premalignant (likely to become cancer), or benign.

APL. 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.

ATRA. A form of vitamin A that is made by the body and can also be made in the laboratory. It is used to treat acute promyelocytic leukemia, usually together with other drugs, and to treat acne. It is being studied in the treatment and prevention of other types of cancer. Also called ATRA, tretinoin, retinoic acid, and vitamin A acid.

A quickly progressing disease in which too many immature white blood cells (not lymphocytes) are found in the blood and bone marrow. Also called acute myelogenous leukemia, acute myeloid leukemia, acute myeloblastic leukemia, acute nonlymphocytic leukemia, and ANLL.

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 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 immunotherapy, biotherapy, biological response modifier therapy, and BRM therapy.

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 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.

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

A term for diseases in which abnormal cells divide without control. Cancer cells can invade nearby tissues and can 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.

Treatment with drugs that kill cancer cells.

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 a clinical study.

Chronic myelogenous leukemia. A slowly progressing disease in which too many white blood cells are made in the bone marrow. Also called chronic myelogenous leukemia, chronic myeloid leukemia, and chronic granulocytic leukemia.

Central nervous system. 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 sanctuary therapy, central nervous system prophylaxis, and CNS prophylaxis.

Treatment using more than one anticancer drug.

A type of high-dose chemotherapy often given as the second phase (after induction therapy) of a cancer treatment regimen for leukemia. Also called intensification therapy.

The process of identifying a disease by the signs and symptoms.

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 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.

Liquid.

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.

A type of white blood cell that fights bacterial infection. Neutrophils, eosinophils, and basophils are granulocytes.

A doctor who specializes in treating blood disorders.

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.

Treatment designed to be used as a first step toward shrinking the cancer and in evaluating response to drugs and other agents. Induction therapy is followed by additional therapy to eliminate whatever cancer remains.

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."

A type of high-dose chemotherapy often given as the second phase (after induction therapy) of a cancer treatment regimen for leukemia. Also called consolidation 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, radiation brachytherapy, and implant radiation 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.

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 juvenile myelomonocytic leukemia.

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 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.

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.

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 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.

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 type of blood cell that helps prevent bleeding by causing blood clots to form. Also called a thrombocyte.

Anticancer drugs given to kill cancer cells that survive after remission induction therapy.

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 particles or electromagnetic waves. Common sources of radiation include radon gas, cosmic rays from outer space, and medical x-rays.

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 radiotherapy and irradiation.

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.

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

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.

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.

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 spine, backbone, 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 standard of care or best practice.

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 palliative care, comfort 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.

Treatment.

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 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 observation.

Refers to a blood cell that does not contain hemoglobin. White blood cells include lymphocytes, neutrophils, eosinophils, macrophages, and mast cells. These cells are made by bone marrow and help the body fight infections and other diseases. Also called 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.