Excerpt for 21st Century Pediatric Cancer Sourcebook: Childhood Acute Myeloid Leukemia (AML), Myeloid Malignancies, Chronic Myelogenous Leukemia (CML), Juvenile Myelomonocytic Leukemia (JMML), TMD, MDS by Progressive Management, available in its entirety at Smashwords

21st Century Pediatric Cancer Sourcebook: Childhood Acute Myeloid Leukemia (AML), Myeloid Malignancies, Chronic Myelogenous Leukemia (CML), Juvenile Myelomonocytic Leukemia (JMML), TMD, MDS

Edition 1.0 - March 2011

National Cancer Institute

Smashwords Edition

Copyright 2011 Progressive Management

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American Cancer Society (ACS) * http://www.cancer.org/

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CONTENTS

PART ONE

Chapter 1A: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment - Patient Version

Chapter 2A: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment - Professional Version

PART TWO

Chapter 1B Late Effects of Treatment for Childhood Cancer - Patient Version

Chapter 2B: Pediatric Supportive Care

Chapter 3B: Clinical Trials Background Information

Chapter 4B: Cancer Clinical Trials -The Basic Workbook

Chapter 5B: Cancer Clinical Trials - The In-Depth Program

Chapter 6B: Clinical Trials at NIH

Chapter 7B: How To Find A Cancer Treatment Trial: A Ten Step Guide

Chapter 8B: Taking Part in Cancer Treatment Research Studies

Chapter 9B: Cancer Clinical Trials

Chapter 10B: Access to Investigational Drugs

Chapter 11B: Clinical Trials Conducted by the National Cancer Institute's Center for Cancer Research at the National Institutes of Health Clinical Center

Chapter 12B: Taking Time: Support for People with Cancer

Chapter 13B: Facing Forward - Life After Cancer Treatment

Chapter 14B: When Someone You Love Is Being Treated For Cancer

Chapter 15B: Living Beyond Cancer: Finding a New Balance

Chapter 16B: Caring for the Caregiver

Chapter 17B: Young People With Cancer, A Handbook For Parents

Chapter 18B: When Cancer Returns

Chapter 19B: When Someone You Love Has Advanced Cancer / Support for Caregivers

Chapter 20B: Chemotherapy and You

Chapter 21B: Managing Chemotherapy Side Effects - Anemia

Chapter 22B: Managing Chemotherapy Side Effects - Appetite Changes

Chapter 23B: Managing Chemotherapy Side Effects - Bleeding Problems

Chapter 24B: Managing Chemotherapy Side Effects - Constipation

Chapter 25B: Managing Chemotherapy Side Effects - Memory Changes

Chapter 26B: Managing Chemotherapy Side Effects - Mouth and Throat Changes

Chapter 27B: Managing Chemotherapy Side Effects - Nerve Changes

Chapter 28B: Managing Chemotherapy Side Effects - Pain

Chapter 29B: Managing Chemotherapy Side Effects - Skin and Nail Changes

Chapter 30B: Managing Chemotherapy Side Effects - Swelling (Fluid retention)

Chapter 31B: Targeted Cancer Therapies

Chapter 32B: Cancer Vaccines

Chapter 33B : Follow-up Care After Cancer Treatment

Chapter 34B: Radiation Therapy and You

Chapter 35B: Understanding Radiation Therapy - What To Know About Brachytherapy (A Type of Internal Radiation Therapy)

Chapter 36B: Understanding Radiation Therapy, What To Know About External Beam Radiation Therapy

Chapter 37B: Radiation Therapy for Cancer

Chapter 38B: Managing Radiation Therapy Side Effects - What To Do When Your Mouth or Throat Hurts

Chapter 39B: What To Do About Hair Loss (Alopecia)

Chapter 40B: Managing Radiation Therapy Side Effects - What To Do When You Have Loose Stools (Diarrhea)

Chapter 41B: Managing Radiation Therapy Side Effects - What To Do About Feeling Sick to Your Stomach and Throwing Up (Nausea and Vomiting)

Chapter 42B: Managing Radiation Therapy Side Effects - Changes When You Urinate

Chapter 43B: Managing Radiation Therapy Side Effects What To Do About Mild Skin Changes

Chapter 44B: Managing Radiation Therapy Side Effects What To Do When You Feel Weak or Tired (Fatigue)

Chapter 45B: General Cancer Information And Resources

Chapter 46B: Cancer And The Environment - What You Need to Know, What You Can Do

Chapter 47B: Guide To Leading Medical Websites, Internet Resources For Medical And Health Information

Chapter 48B: FDA Warning: Beware of Online Cancer Fraud

Chapter 49B: FDA Office of Oncology Drug Products

Chapter 50B: Understanding the HIPAA Privacy Rule

Chapter 51B: Patient Protection and Affordable Care Act (PPACA or ACA) - Understanding Obamacare and Your Health Care Insurance Options, New Plans, Programs, Bill of Rights

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Chapter 1A: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment - Patient Version

Last Modified: 10/28/2010

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.

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.

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.

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.

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.

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.

Seven types of standard treatment are used for childhood AML, childhood CML, JMML, TMD, or MDS.

Chemotherapy * Radiation therapy * Stem cell transplantation * Targeted therapy with a tyrosine kinase inhibitor * Other drug therapy * Watchful waiting * Supportive care

New types of treatment are being tested in clinical trials.

Targeted 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.

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.

Seven 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 cerebrospinal fluid (intrathecal chemotherapy), an organ, or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). Combination chemotherapy is treatment using more than one 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.

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.

Targeted therapy with a tyrosine kinase inhibitor

Targeted therapy is a treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Tyrosine kinase inhibitor (TKI) therapy is a type of targeted therapy that blocks signals needed for tumors to grow. TKIs blocks the enzyme, tyrosine kinase, that causes stem cells to develop into more white blood cells (granulocytes or blasts) than the body needs. Imatinib (Gleevec) is one of the TKIs used to treat childhood CML.

TKIs may be used in combination with other anticancer drugs as adjuvant therapy (treatment given after the initial treatment, to lower the risk that the cancer will come back).

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).

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.

Targeted therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Monoclonal antibodies, proteasome inhibitors, and natural killer (NK) cells are three types of targeted therapies being studied in the treatment of childhood AML.

Monoclonal antibody therapy 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. Monoclonal antibodies may be used in combination with chemotherapy as adjuvant therapy.

Proteasome inhibitors break down proteins in cancer cells and kill them. Bortezomib is a proteasome inhibitor used to treat childhood acute promyelocytic leukemia.

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 listing of clinical trials.

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.

Newly Diagnosed Childhood Acute Myeloid Leukemia

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 list of cancer clinical trials 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.

Childhood Acute Myeloid Leukemia in Remission

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 targeted therapy with natural killer cell transplant after chemotherapy.

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with childhood acute myeloid leukemia in remission. 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.

Recurrent Childhood Acute Myeloid Leukemia

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 targeted therapy with a tyrosine kinase inhibitor and combination chemotherapy.

A clinical trial of stem cell transplant using different sources of stem cells.

Acute Promyelocytic Leukemia

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.

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

Recurrent Acute Promyelocytic Leukemia

Treatment of recurrent acute promyelocytic leukemia may include the following:

All-trans retinoic acid therapy (ATRA).

Arsenic trioxide therapy

A clinical trial of targeted therapy with a proteasome inhibitor and ATRA.

Children with Down Syndrome and AML

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

Combination chemotherapy.

A clinical trial of lower- dose chemotherapy.

Childhood Chronic Myelogenous Leukemia

Treatment for childhood chronic myelogenous leukemia may include the following:

Stem cell transplant.

Targeted therapy with Gleevec or other tyrosine kinase inhibitors.

A clinical trial of stem cell transplant using lower doses of chemotherapy.

Juvenile Myelomonocytic Leukemia

Treatment of juvenile myelomonocytic leukemia is usually stem cell transplant.

Transient Myeloproliferative Disorder

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.

Myelodysplastic Syndromes

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

Stem cell transplant.

Combination chemotherapy.

A clinical trial of a new anticancer drug or targeted therapy.

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 list of cancer clinical trials that are now accepting patients with childhood myelodysplastic syndromes. 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.

Glossary Terms

abnormal

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

acute (uh-KYOOT)

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

acute myeloid leukemia (uh-KYOOT MY-eh-loyd loo-KEE-mee-uh)

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.

acute promyelocytic leukemia (uh-KYOOT PRO-MY-eh-loh-SIH-tik loo-KEE-mee-uh)

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.

anemia (uh-NEE-mee-uh)

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

antigen (AN-tih-jen)

Any substance that causes the body to make a specific immune response.

aplastic anemia (AY-PLAS-tik uh-NEE-mee-uh)

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

benzene

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.

biopsy (BY-op-see)

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.

blast

An immature blood cell.

blood

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.

blood chemistry study (blud KEH-mih-stree STUH-dee)

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.

blood clot

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.

bone marrow (bone MAYR-oh)

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

bone marrow aspiration (bone MAYR-oh AS-pih-RAY-shun)

A procedure in which a small sample of bone marrow is removed, usually from the hip bone, breastbone, or thigh bone. A small area of skin and the surface of the bone underneath are numbed with an anesthetic. Then, a special wide needle is pushed into the bone. A sample of liquid bone marrow is removed with a syringe attached to the needle. The bone marrow is sent to a laboratory to be looked at under a microscope. This procedure may be done at the same time as a bone marrow biopsy.

bone marrow biopsy (bone MAYR-oh BY-op-see)

A procedure in which a small sample of bone with bone marrow inside it is removed, usually from the hip bone. A small area of skin and the surface of the bone underneath are numbed with an anesthetic. Then, a special, wide needle is pushed into the bone and rotated to remove a sample of bone with the bone marrow inside it. The sample is sent to a laboratory to be looked at under a microscope. This procedure may be done at the same time as a bone marrow aspiration.

cell (sel)

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

central nervous system (SEN-trul NER-vus SIS-tem)

The brain and spinal cord. Also called CNS.

cerebrospinal fluid (seh-REE-broh-SPY-nul FLOO-id)

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.

chemotherapy (KEE-moh-THAYR-uh-pee)

Treatment with drugs that kill cancer cells.

chest x-ray

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.

chloroma

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.

chromosome (KROH-muh-some)

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

chronic (KRAH-nik)

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

chronic myelogenous leukemia (KRAH-nik MY-eh-LAH-jeh-nus loo-KEE-mee-uh)

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.

complete blood count

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.

cytogenetics (SY-toh-jeh-NEH-tix)

The study of chromosomes and chromosomal abnormalities.

diagnosis (DY-ug-NOH-sis)

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

disorder (dis-OR-der)

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

Down syndrome (...SIN-drome)

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

eczema (EK-zeh-muh)

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.

Fanconi anemia (fan-KOH-nee uh-NEE-mee-uh)

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.

fever (FEE-ver)

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

genetic (jeh-NEH-tik)

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

granulocyte (GRAN-yoo-loh-SITE)

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.

granulocytic sarcoma (GRAN-yoo-loh-SIH-tik sar-KOH-muh)

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.

groin

The area where the thigh meets the abdomen.

hemoglobin (HEE-moh-GLOH-bin)

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

immunophenotyping (IM-yoo-no-FEE-no-tie-ping)

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.

infection

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.

ionizing radiation (I-uh-NYZ-ing RAY-dee-AY-shun)

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.

juvenile myelomonocytic leukemia (JOO-veh-NILE MY-eh-loh-MAH-noh-SIH-tik loo-KEE-mee-uh)

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

laboratory test (LA-bruh-tor-ee...)

A medical procedure that involves testing a sample of blood, urine, or other substance from the body. Tests can help determine a diagnosis, plan treatment, check to see if treatment is working, or monitor the disease over time.

leukemia (loo-KEE-mee-uh)

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.

lumbar puncture (LUM-bar PUNK-cher)

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.

lymph node (limf node)

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.

lymphoid (LIM-foyd)

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

marker

A diagnostic indication that disease may develop.

monocyte (MAH-noh-site)

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.

myelodysplastic syndromes (MY-eh-loh-dis-PLAS-tik SIN-dromz)

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

myeloid (MY-eh-loyd)

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.

myeloproliferative disorder (MY-eh-loh-pruh-LIH-feh-RUH-tiv dis-OR-der)

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.

neurofibromatosis type 1 (NOOR-oh-FY-broh-muh-TOH-sis tipe 1)

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.

organ

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

oxygen (OK-sih-jen)

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.

pathologist (puh-THAH-loh-jist)

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

peripheral blood smear (peh-RIH-feh-rul blud smeer)

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.

petechiae (peh-TEH-kee-a)

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

physical examination (FIH-zih-kul eg-ZA-mih-NAY-shun)

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

platelet (PLATE-let)

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.

preleukemia (PREE-loo-KEE-mee-a)

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

prognosis (prog-NO-sis)

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

protein (PRO-teen)

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.

radiation therapy (RAY-dee-AY-shun THAYR-uh-pee)

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.

recurrent cancer (ree-KER-ent KAN-ser)

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.

red blood cell

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

risk factor (... FAK-ter)

Something that increases the chance of developing a disease. Some examples of risk factors for cancer are age, a family history of certain cancers, use of tobacco products, being exposed to radiation or certain chemicals, infection with certain viruses or bacteria, and certain genetic changes.

solid tumor

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.

spinal column (SPY-nul KAH-lum)

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.

spinal cord (SPY-nul kord)

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.

stem cell

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

stomach (STUH-muk)

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.

symptom

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

tissue (TISH-oo)

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

tumor (TOO-mer)

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.

white blood cell

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.

x-ray

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.

* * * * * * * * * * * *

Chapter 2A: Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment - Last Modified: 10/20/2010 - Health Professional Version

Table of Contents

General Information Myeloid Leukemias in Children * Classification of Pediatric Myeloid Malignancies French-American-British (FAB) Classification for Childhood Acute Myeloid Leukemia * World Health Organization (WHO) Classification System * Histochemical Evaluation * Immunophenotypic Evaluation * Cytogenetic Evaluation and Molecular Abnormalities * Classification of Myelodysplastic Syndromes in Children * Diagnostic Classification of Juvenile Myelomonocytic Leukemia * Stage Information Newly Diagnosed * Remission * Treatment Overview for Acute Myeloid Leukemia Prognostic Factors in Childhood Acute Myeloid Leukemia * Treatment of Newly Diagnosed Acute Myeloid Leukemia Induction Chemotherapy * Treatment options under clinical evaluation * Central Nervous System (CNS) Prophylaxis for Acute Myeloid Leukemia (AML) * Granulocytic Sarcoma (GS)/Chloroma * Current Clinical Trials * Postremission Therapy for Acute Myeloid Leukemia Treatment Options Under Clinical Evaluation * Current Clinical Trials * Acute Promyelocytic Leukemia Treatment Options Under Clinical Evaluation * Current Clinical Trials * Children with Down Syndrome Treatment Options Under Clinical Evaluation * Myelodysplastic Syndromes Treatment Options Under Clinical Evaluation * Current Clinical Trials * Juvenile Myelomonocytic Leukemia Current Clinical Trials * Chronic Myelogenous Leukemia Treatment Options Under Clinical Evaluation * Current Clinical Trials * Recurrent Childhood Acute Myeloid Leukemia and Other Myeloid Mal Isolated Central Nervous System Relapse * Recurrent Acute Promyelocytic Leukemia * Treatment Options Under Clinical Evaluation * Current Clinical Trials * Survivorship and Adverse Late Sequelae

Fortunately, cancer in children and adolescents is rare, although the overall incidence of childhood cancer has been slowly increasing since 1975.[1] Children and adolescents with cancer should be referred to medical centers that have a multidisciplinary team of cancer specialists with experience treating the cancers that occur during childhood and adolescence. This multidisciplinary team approach incorporates the skills of the primary care physician, pediatric surgical subspecialists, radiation oncologists, pediatric medical oncologists/hematologists, rehabilitation specialists, pediatric nurse specialists, social workers, and others in order to ensure that children receive treatment, supportive care, and rehabilitation that will achieve optimal survival and quality of life.

Guidelines for pediatric cancer centers and their role in the treatment of children with cancer have been outlined by the American Academy of Pediatrics.[2] At these pediatric cancer centers, clinical trials are available for most types of cancer that occur in children and adolescents, and the opportunity to participate in these trials is offered to most patients/families. Clinical trials for children and adolescents with cancer are generally designed to compare potentially better therapy with therapy that is currently accepted as standard. Most of the progress made in identifying curative therapies for childhood cancers has been achieved through clinical trials. Information about ongoing clinical trials is available from the NCI Web site.

Dramatic improvements in survival have been achieved for children and adolescents with cancer.[1] Between 1975 and 2002, childhood cancer mortality has decreased by more than 50%. For acute myeloid leukemia, the 5-year survival rate has increased over the same time from less than 20% to 58% for children younger than 15 years and from less than 20% to approximately 40% for adolescents aged 15 to 19 years.[1] Childhood and adolescent cancer survivors require close follow-up because cancer therapy side effects may persist or develop months or years after treatment.

Myeloid Leukemias in Children

The myeloid leukemias in childhood represent a spectrum of hematopoietic malignancies. More than 90% of myeloid leukemias are acute and the remainder includes chronic and/or subacute myeloproliferative disorders such as chronic myelogenous leukemia (CML) and juvenile myelomonocytic leukemia (JMML). Myelodysplastic syndromes represent less than 5% of myeloid malignancies in children.


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