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Leukemia - Acute Myeloid - AML - Childhood - Overview

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find some basic information about this disease and the parts of the body it may affect. This is the first page of Cancer.Net’s Guide to Childhood Acute Myeloid Leukemia (AML). To see other pages, use the menu on the side of your screen. Think of that menu as a roadmap to this full guide.

Leukemia is a cancer of the blood. It begins when normal blood cells change and grow uncontrollably. Acute myeloid leukemia (AML) is a type of leukemia that is a cancer of the blood-forming tissue in the bone marrow. AML may also be called acute nonlymphocytic leukemia or acute myelogenous leukemia.

About bone marrow and blood cells

Bone marrow is the spongy, red tissue in the inner part of the large bones and is where a person’s blood cells are made. Normal immature blood cells are called blasts. Blasts mature into one of three different types of blood cells:

  • White blood cells, which fight infection in the body
  • Red blood cells, which carry oxygen and other nutrients throughout the body
  • Platelets, which help the blood to clot

About AML

In AML, the bone marrow makes many abnormal cancerous cells, also called blasts or myeloblasts because they look similar to normal immature blast cells. Instead of becoming healthy mature blood cells, cancerous cells divide rapidly and uncontrollably. The cancerous cells are unable to mature and work like normal blast cells, and they do not die easily. Eventually, these myeloblasts fill up the bone marrow, preventing healthy cells from being made, and then build up in the bloodstream. They can also invade the lymph nodes, brain, skin, liver, kidneys, ovaries (in girls), testicles (in boys), and other organs. AML cells occasionally form a solid mass or tumor, called a chloroma.

Both children and adults can develop leukemia. This section is about AML that occurs in children, sometimes called pediatric AML. Learn more about AML in adults.

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If you would like additional introductory information, explore these related items. Please note these links will take you to other sections on Cancer.Net:

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Leukemia - Acute Myeloid - AML - Childhood - Statistics

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find information about how many children learn they have this type of cancer each year and some general survival information. Remember, survival rates depend on several factors. To see other pages, use the menu on the side of your screen.

In general, leukemia is the most common childhood cancer. AML is the second most common form of leukemia in children, after acute lymphoblastic leukemia (ALL). About 500 children and 230 adolescents in the United States are diagnosed with AML each year. Childhood AML is most common during the first two years of life and during the teenage years.

The five-year survival rate is the percentage of people who survive at least five years after the cancer is detected, excluding those who die from other diseases. Overall, the five-year survival rate for children with AML is between 60% and 70%. However, the survival rates for AML vary based on the subtype.

Cancer survival statistics should be interpreted with caution. These estimates are based on data from thousands of people with this type of cancer, but the actual risk for a particular individual may differ. It is not possible to tell a person how long he or she will live with AML. Because the survival statistics are measured in five-year intervals, they may not represent advances made in the treatment or diagnosis of this cancer. Learn more about understanding statistics.

Source: American Cancer Society and St. Jude Children’s Research Hospital.

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Leukemia - Acute Myeloid - AML - Childhood - Risk Factors

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find out more about what factors increase the chance of this type of cancer. To see other pages, use the menu on the side of your screen.

A risk factor is anything that increases a person’s chance of developing cancer. Although risk factors often influence the development of cancer, most do not directly cause cancer. Some people with several risk factors never develop cancer, while others with no known risk factors do.

Doctors and researchers don’t know what causes most childhood cancers, including AML. Sometimes, AML may be caused by certain genetic or environmental factors. For example, children who have Down syndrome have an increased risk of AML during the first four years of life. However, the reasons for this increased risk are not well understood.

AML occurs more often in children younger than two. The number of people with AML increases again in late childhood (during the teenage years) and continues to increase throughout later adulthood.

To continue reading this guide, use the menu on the side of your screen to select another section.  

Leukemia - Acute Myeloid - AML - Childhood - Symptoms and Signs

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find out more about body changes and other things that can signal a problem that may need medical care. To see other pages, use the menu on the side of your screen.

Children with AML may experience the following symptoms or signs. Sometimes, children with AML do not show any of these symptoms. Or, these symptoms may be caused by a medical condition that is not cancer. If you are concerned about a symptom or sign on this list, please talk with your child’s doctor.

The early signs and symptoms of AML can look very much like the flu or other common childhood illnesses. Most of the signs and symptoms of AML are caused by the bone marrow making fewer normal blood cells and the buildup of cancerous AML cells. Often a child with AML will have one or more of the following symptoms:

  • Fever
  • Chills
  • Aching bones and joints
  • Swollen lymph nodes
  • Bleeding and bruising easily
  • Paleness
  • Fatigue

The doctor will ask you questions about the symptoms your child is experiencing to help find out the cause of the problem, called a diagnosis. This may include how long your child has been experiencing the symptom(s) and how often.

If cancer is diagnosed, relieving symptoms and side effects remains an important part of cancer care and treatment. This may also be called symptom management, palliative care, or supportive care. Be sure to talk with your child’s health care team about symptoms your child experiences, including any new symptoms or a change in symptoms.

The next section helps explain what tests and scans may be needed to learn more about the cause of the symptoms. Use the menu on the side of your screen to select Diagnosis, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Diagnosis

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find a list of the common tests, procedures, and scans that doctors can use to find out what’s wrong and identify the cause of the problem. To see other pages, use the menu on the side of your screen.

Doctors use many tests to diagnose leukemia. Some tests may also help the doctor choose the treatment that may be the most effective. This list describes options for diagnosing this type of cancer, and not all tests listed will be used for every child. Your child’s doctor may consider these factors when choosing a diagnostic test:

  • Age and medical condition
  • Type of cancer suspected
  • Signs and symptoms
  • Previous test results

When a child has signs and symptoms of leukemia, the doctor will ask about the child’s medical history and perform a physical examination. In addition, the following tests may be used to diagnose AML:

Blood tests. Complete blood count (CBC) and cell type (called differential count) are blood tests done to count the number of each type of blood cell under a microscope and to check if they look abnormal.

Bone marrow aspiration and biopsy. A bone marrow aspiration is performed if the blood test shows abnormal blood counts. For this test, a sample of bone marrow is removed from the child’s hip with a needle and examined under a microscope. The child’s skin in that area will be numbed with medication beforehand, and other types of anesthesia (medication to block the awareness of pain) may be used. From this test, the doctor can find out whether the child has leukemia and, if so, what type of leukemia it is.

Molecular and genetic testing. Your doctor may recommend running laboratory tests to identify specific genes, proteins, and other factors involved in the leukemia. Examining the genes in the leukemia cells is important because AML can be caused by a buildup of mistakes (also called mutations) in the genes of a cell in the body. Identifying these mistakes helps diagnose the specific subtype of AML and choose treatment options. In addition, they can also be used to monitor how well treatment is working. Listed below are the more common molecular or genetic tests used for AML.

  • Cytochemical and immunohistochemical tests are laboratory tests that are used to find out the exact subtype of AML. In cytochemical tests, a special dye is used that stains the different types of leukemia cells differently based on the chemicals in the cells. For AML, immunohistochemical tests and a test called flow cytometry are used to find markers on the surface of the leukemia cells. The different subtypes of leukemia have different and unique combinations of cell surface markers.
  • Cytogenetics is a way to look at cell’s chromosomes (long strands of genes) through a microscope to analyze the number, size, shape, and the arrangement of the chromosomes to find genetic changes in the leukemia cells. Sometimes, a chromosome breaks off and reattaches to another chromosome, which is called a translocation. Other times, part of a chromosome is missing, called a deletion. A chromosome can also be made more than once, most often called a trisomy. Some subtypes of leukemia are caused by chromosome translocations, deletions, or trisomies. Knowing if there are certain translocations may help doctors determine the AML subtype and plan the best treatment. Flourescence-in-situ-hybridization (FISH) is a way to detect chromosome changes in cancer cells and is being used more often to help diagnose and determine the subtype of leukemia. This test is done on tissue removed during a biopsy or aspiration (see above).
  • The molecular genetics of leukemia cells can also be used to help find out if a person needs more or less chemotherapy and/or stem cell transplantation (see the Treatment section). The goal of this type of testing is to look for very small genetic mutations, called sub-microscopic mutations. People who have the Flt3 (pronounced flit 3) genetic mutation, called an internal tandem duplication (ITD), and specifically those with the mutation on both versions of the gene, have a high risk that the cancer will come back after treatment. For children with this type of AML, the use of stem cell transplantation may lengthen their lives when used after the first complete remission (see the Treatment section). There are also new drugs being tested that target Flt3-ITD positive cells to find out if the drugs can better treat the leukemia. Most recently, research has shown that children with two leukemia cell gene mutations called nucleophosmin-1 (NPM1) and CEBPα have a better prognosis (chance of recovery) than those without these mutations. If a child has these mutations, the doctor may recommend chemotherapy without stem cell transplantation.
  • Recently, highly advanced tools that can look at a person’s entire genetic make-up, called whole genome testing and whole exome testing, have been developed. These methods of testing are still early but have been used to look for genetic mistakes that can lead to cancer. However, these tests are still generally only available in research studies where they are being used to find out if using these tests improves diagnosis, treatment, and cure.

After these diagnostic tests are done, your child’s doctor will review all of the results with you. If the diagnosis is AML, these results also help the doctor describe the cancer and determine the subtype.

The next section helps explain the different subtypes for this type of cancer. Use the menu on the side of your screen to select Subtypes, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Subtypes

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will learn about how doctors determine the subtype of AML. To see other pages, use the menu on the side of your screen.

AML blasts are classified based on how much they look like normal immature bone marrow blast cells, and more recently, on the genetic testing of the leukemia cells (see the Diagnosis section). In the recent past, AML was divided into eight major subtypes according to a system called the FAB classification scheme (using levels M0 to M7). Now, a new method of subtyping AML is used that takes into consideration the cytogenetic causes of AML. This is called the World Health Organization (WHO) 2008 classification. These subtypes include:

  • Acute myeloid leukemia with recurrent genetic abnormalities
    • AML with t(8;21)(q22;q22); RUNX1-RUNX1T1
    • AML with inv(16)(p13.1q22) or t(16;16)(p13.1;q22); CBFB-MYH11
    • Acute promyelocytic leukemia (APL) with t(15;17)(q22;q12); PML-RARA
    • AML with t(9;11)(p22;q23); MLLT3-MLL
    • AML with t(6;9)(p23;q34); DEK-NUP214
    • AML with inv(3)(q21q26.2) or t(3;3)(q21;q26.2); RPN1-EVI1
    • AML (megakaryoblastic) with t(1;22)(p13;q13); RBM15-MKL1
    • AML with mutated NPM1
    • AML with mutated CEBPα
  • Acute myeloid leukemia with myelodysplasia-related changes
  • Therapy-related myeloid neoplasms
  • Acute myeloid leukemia, not otherwise specified
    • AML with minimal differentiation
    • AML without maturation
    • AML with maturation
    • Acute myelomonocytic leukemia
    • Acute monoblastic/monocytic leukemia
    • Acute erythroid leukemia
      • Pure erythroid leukemia
      • Erythroleukemia, erythroid/myeloid
    • Acute megakaryoblastic leukemia
    • Acute basophilic leukemia
    • Acute panmyelosis with myelofibrosis
  • Myeloid sarcoma
  • Myeloid proliferations related to Down syndrome
    • Transient abnormal myelopoiesis
    • Myeloid leukemia associated with Down syndrome

Information about the cancer’s subtype will help the doctor recommend a treatment plan.  The next section helps explain the treatment options for this type of cancer. Use the menu on the side of your screen to select Treatment Options, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Treatment Options

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will learn about the different ways doctors use to treat children with this type of cancer. To see other pages, use the menu on the side of your screen.

The following terms are used to describe the state of disease for children with AML:

Untreated AML. The child has not received any treatment except to relieve symptoms of the disease. The blood and/or bone marrow contains too many white blood cells, and the child has signs and symptoms of the disease.

Complete Remission AML. There are too few cancerous blast cells in the bone marrow to tell apart from normal blasts under the microscope (fewer than 5% blasts in the bone marrow). New monitoring methods, called minimal residual disease (MRD; cancer cells not destroyed by treatment) methods, are better able to find fewer cancerous blasts, as well as tell the difference between cancerous blasts and normal blasts. MRD methods are now being used more often to determine remission, and research studies are ongoing to find out if these methods are better able to predict the chance of curing the disease. The child usually does not have any signs or symptoms of the disease when in complete remission.

Partial Remission AML. The number of cancerous blast cells in the bone marrow is reduced but still can be seen under the microscope (or by MRD methods if appropriate). The child usually does not have any signs or symptoms of the disease. This happens only during initial therapy called induction (see below).

Recurrent AML. The disease has come back after the child had a period of remission (complete absence of symptoms; see below).

Refractory AML. The leukemia did not go into complete remission after treatment.

Treatment overview

In general, cancer in children is uncommon, so it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That’s why more than 60% of children with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare standard treatments (the best proven treatments available) with newer treatments that may be more effective. Studying new treatments involves careful monitoring using scientific methods, and all participants are followed closely to track their health and progress.

To take advantage of these newer treatments, all children with cancer should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children with cancer and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. In many cases, a team of doctors works with a child and the family to provide care; this is called a multidisciplinary team. Pediatric cancer centers often have extra support services for children and their families, such as child life specialists, nutritionists, physical and occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.

An increasing number of pediatric cancer centers also have services for teenagers and young adults. Sometimes, adult cancer centers also offer clinical trials for teens and young adults with cancer.

Descriptions of each treatment option for AML are listed below. Two types of treatment are commonly used to treat AML in children: chemotherapy and stem cell/bone marrow transplantation. Radiation therapy is used occasionally in specific situations. Sometimes, more than one treatment is used. Treatment options and recommendations depend on several factors, including the subtype of AML, possible side effects, and the patient’s preferences and overall health. Take time to learn about the treatment options and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your child's doctor and what you and your child can expect while receiving the treatment. Learn more about making treatment decisions

Chemotherapy

Chemotherapy is the most common treatment for AML. It is the use of drugs to kill cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. It is given by a pediatric oncologist or hematologist, a doctor who specializes in treating blood disorders. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.

Several treatment regimens have been developed that involve intensive use of several drugs. Following these regimens, about 85% of children will have an initial remission, and about 50% to 60% will be cured (meaning that the cancer never returns). During treatment, children with AML need to be watched very carefully and often spend many weeks in the hospital because very low blood cell counts increase the risk of developing infections.

Chemotherapy may be given by mouth, injected into a vein, or injected into the cerebrospinal fluid (CSF) that circulates around the brain and spinal cord. The choice of drugs depends on whether the child has previously been treated for AML, as well as other factors.

Chemotherapy for AML is usually divided into two phases of treatment: induction and intensification.

Induction chemotherapy uses chemotherapy to destroy as many of the cancer cells as possible to make the AML to go into remission.

Intensification chemotherapy is used to destroy any cancer cells that may be hiding (meaning that there are too few to detect by modern tests) after induction chemotherapy has led to remission. Stem cell transplantation also can be used for intensification therapy (see below).

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, low blood cell counts, risk of infection, hair loss, nausea and vomiting, hair loss, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished. The severity of side effects may also be affected by other factors, including genetic differences in the way the drugs are used by the body and the child’s overall health and well-being.

Doctors understand that everyone is different. Most children are initially treated similarly to other children with the same cancer. However, based on the side effects, doses or schedules may be changed. This is a constant balance between the effort to kill all the cancer cells and the need to avoid severe side effects. Your child’s doctor will discuss changes to the treatment plan with you as they are needed, and not all children will need to have their chemotherapy changed.

Learn more about chemotherapy and preparing for treatment. The medications used to treat cancer are continually being evaluated. Talking with the doctor is often the best way to learn about the medications prescribed for your child, their purpose, and their potential side effects or interactions with other medications. Learn more about your child’s prescriptions by using searchable drug databases.

Stem cell transplantation/bone marrow transplantation

Children with AML have different risks of recurrence depending on the subtype. For children with a higher risk of recurrence, it is more likely that the doctor will recommend stem cell transplantation to help prevent a recurrence. A stem cell transplant is a medical procedure in which bone marrow that contains cancer is replaced by highly specialized cells called hematopoietic stem cells that develop into healthy bone marrow. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant because it is the stem cells in the blood that are typically being transplanted, not the actual bone marrow tissue.

Before recommending stem cell transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of leukemia, results of any previous treatment, and patient’s age and general health.

Not all children with AML need stem cell transplantation. For instance, children with Down syndrome and the subtype called APL with t(15;17) do not need stem cell transplantation unless the AML recurs. AML with certain genetic changes (for example, chromosome abnormalities known as inv 16 and t[8;21], or molecular changes known as NPM1 or CEBPα) is not treated with stem cell transplantation unless the leukemia has recurred. Children with subtypes of AML that have lower risks of recurrence (and therefore higher chances of cure) can usually receive chemotherapy alone. Also, most children whose leukemia does not have highest risk genetic changes and who have experienced a remission quickly often do not need a stem cell transplant and can receive chemotherapy instead.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). ALLO transplantation is the type most often used for AML. In an ALLO transplant, the patient receives donor cells from another person. For children with a higher risk for recurrence, when a related donor is available, stem cell transplantation is the preferred treatment after a child has a first remission. For children with the highest risk of recurrence and the poorest chance of recovery, stem cell transplantation with either a related or unrelated donor is often used in addition to chemotherapy after the child has a first remission.

In the past, transplantations from unrelated donors were not done unless the AML had recurred. However, as the safety of using stem cells/bone marrow from unrelated donors has improved, these types of transplants are being used more often to prevent recurrence for children with high risk AML. Clinical trials are studying the use of unrelated donor transplants for patients with AML that has certain high-risk features (for example, a chromosome abnormality called monosomy 7 or a child who doesn’t experience a remission after their first induction chemotherapy).

The goal of stem cell transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. Learn more about bone marrow and stem cell transplantation.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. A radiation therapy regimen (schedule) usually consists of a specific number of treatments given over a set period of time.

Radiation therapy for AML is generally used only if the cancer has spread to the brain and does not respond to systemic chemotherapy and/or chemotherapy given into the spinal fluid. Radiation therapy may also be used to treat a chloroma when chemotherapy has not fully worked. As explained above, radiation therapy may also be used during a stem cell/bone marrow transplant.

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Radiation therapy can sometimes interfere with the normal growth and development of the child’s brain and body. Therefore, when possible, chemotherapy is used first to avoid radiation therapy. More information about long-term effects can be found in the After Treatment section. Learn more about radiation therapy.

Getting care for symptoms and side effects

Leukemia and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the disease, an important part of care is relieving a child’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care can help a child at any stage of illness. Children often receive treatment for the leukemia and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, and other therapies. Your child may also receive palliative treatments similar to those meant to eliminate the cancer, such as chemotherapy and radiation therapy. Talk with your child's doctor about the goals of each treatment in your child's treatment plan.

Before treatment begins, talk with your child’s health care team about the possible side effects of the specific treatment plan and supportive care options. And during and after treatment, be sure to tell your child’s doctor or another health care team member if you are experiencing a problem so it is addressed as quickly as possible. Learn more about palliative care.

Recurrent AML

A remission is when leukemia cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED. 

A remission can be temporary or permanent. This uncertainty leads to many families feeling worried or anxious that the cancer will come back. While many remissions are permanent, it’s important to talk with your child’s doctor about the possibility of the disease returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the leukemia does return. Learn more about coping with the fear of recurrence.   

If the cancer does return after the original treatment, it is called recurrent (or relapsed) cancer. Most often, AML comes back in the bone marrow, but sometimes it may come back in the brain or other parts of the body.

When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, you and your child’s doctor will talk about the treatment options. Treatment usually includes chemotherapy followed by stem cell transplantation. However, each child’s treatment is planned based on his or her specific circumstances. Your child’s doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent cancer. For some children, recurrent AML can be cured.

When cancer recurs, patients and their families often experience emotions such as disbelief or fear. Families are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

If treatment fails

Although treatment is successful for the majority of children with cancer, sometimes it is not. If a child’s cancer cannot be cured or controlled, this is called advanced or terminal cancer. This diagnosis is stressful, and it may be difficult to discuss. However, it is important to have open and honest conversations with your child’s doctor and health care team to express your family’s feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families.

Parents or guardians are encouraged to think about where the child would be most comfortable: at home, in a home-like setting elsewhere, in the hospital, or in a hospice environment. Hospice care is a type of palliative care for people who are expected to live less than six months. It is designed to provide the best possible quality of life for people who are near the end of life. Nursing care and special equipment can make staying at home a workable alternative for many families. Some children may be happier if they can arrange to attend school part-time or keep up other activities and social connections. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is extremely important as part of end-of-life care. Learn more about caring for a terminally ill child and advanced cancer care planning.

The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving. Learn more on grieving the loss of a child.

The next section helps explain clinical trials, which are research studies. Use the menu on the side of your screen to select About Clinical Trials, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - About Clinical Trials

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will learn more about clinical trials, which are the main way that new medical approaches are tested to see how well they work. To see other pages, use the menu on the side of your screen.

Doctors and scientists are always looking for better ways to treat children with AML. To make scientific advances, doctors create research studies involving volunteers, called clinical trials.

Many clinical trials are focused on new treatments, evaluating whether a new treatment is safe, effective, and possibly better than the current (standard) treatment. These types of studies evaluate new drugs, different combinations of existing treatments, new approaches to radiation therapy or surgery, and new methods of treatment. Children who participate in clinical trials are often among the first to receive new treatments before they are widely available. However, there is no guarantee that the new treatment will be safe, effective, or better than a standard treatment.

There are also clinical trials that study new ways to ease symptoms and side effects during treatment and manage the late effects that may occur after treatment. Talk with your child’s doctor about clinical trials regarding side effects. In addition, there are ongoing studies about ways to prevent the disease. 

Patients decide to participate in clinical trials for many reasons. For some children, a clinical trial is the best treatment option available. Because standard treatments are not perfect, patients are often willing to face the added uncertainty of a clinical trial in the hope of a better result. Other patients volunteer for clinical trials because they know that these studies are the only way to make progress in treating AML. Even if they do not benefit directly from the clinical trial, their participation may benefit future people with AML.

Sometimes people have concerns that, by participating in a clinical trial, they may receive no treatment by being given a placebo or a “sugar pill.” The use of placebos in cancer clinical trials is rare, and placebos are never used in clinical trials for the treatment of children with cancer. When a placebo is used in a study, it is done with the full knowledge of the participants. Find out more about placebos in cancer clinical trials.

To join a clinical trial, parents and children must participate in a process known as informed consent. During informed consent, the doctor should list all of the patient’s options so that the person understands how the new treatment differs from the standard treatment. The doctor must also list all of the risks of the new treatment, which may or may not be different from the risks of standard treatment. Finally, the doctor must explain what will be required of each patient in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.

For specific topics being studied for childhood AML, learn more in the Latest Research section.

Patients who participate in a clinical trial may stop participating at any time for any personal or medical reason. This may include that the new treatment is not working or there are serious side effects. Clinical trials are also closely monitored by experts who watch for any problems with each study. It is important that patients participating in a clinical trial talk with their doctor and researchers about who will be providing their treatment and care during the clinical trial, after the clinical trial ends, and/or if the patient chooses to leave the clinical trial before it ends. 

Cancer.Net offers a lot of information about cancer clinical trials in other areas of the website, including a complete section on clinical trials and places to search for clinical trials for a specific type of cancer.

The next section helps explain the areas of research going on today about this type of cancer. Use the menu on the side of your screen to select Latest Research, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Latest Research

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will read about the scientific research being done now to learn more about this type of cancer and how to treat it. To see other pages, use the menu on the side of your screen.

Doctors are working to learn more about AML, ways to prevent it, how to best treat it, and how to provide the best care to children diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the diagnostic and treatment options best for your child.

Genetic testing. The genetics of leukemia cells are being tested to see if they can help doctors decide which patients may need more or less intense treatment. These discoveries help doctors better understand the causes of leukemia, determine the prognosis for each child, and develop new drugs that target these specific genetic changes.

Better detection. New methods to measure minimal residual disease can find one leukemia cell in 10,000 normal cells, which can help determine the likelihood of recurrence. In the future, changes made to chemotherapy treatments and/or the use of stem cell transplantation may be partly based on these new types of tests.

New drugs and combinations of drugs. Targeted therapy is a treatment that targets the cancer’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to normal cells.

Sorafenib (Nexavar) is a type of targeted therapy called a protein kinase inhibitor that specifically targets AML blasts with the Flt3 mutation. It is being tested in combination with chemotherapy for children newly diagnosed with AML. Other new drugs that may be used for AML are being tested in clinical trials or are in development in research laboratories. Learn more about targeted treatments.

Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current leukemia treatments in order to improve patients’ comfort and quality of life.

Looking for More about the Latest Research?

If you would like additional information about the latest areas of childhood leukemia research, explore these related items that take you outside of this guide:

  • To find clinical trials specific to your child’s diagnosis, talk with the doctor or search online clinical trial databases now.

  • Visit ASCO’s CancerProgress.Net website to learn more about the historical pace of research for childhood cancer. Please note this link takes you to a separate ASCO website.

The next section addresses how to cope with the symptoms of the disease or the side effects of its treatment. Use the menu on the side of your screen to select Coping with Side Effects, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Coping with Side Effects

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find out more about steps to take to help your child cope with physical, social, and emotional side effects. This page includes several links outside of this guide to other sections of this website. To see other pages, use the menu on the side of your screen.

Fear of treatment side effects is common after a diagnosis of leukemia, but it may help to know that preventing and controlling side effects is a major focus of your child’s health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.

Common side effects from each treatment option for AML are described in detail within the Treatment section. Learn more about the most common side effects of leukemia and different treatments, along with ways to prevent or control them. Side effects depend on a variety of factors, including the subtype, the length and dosage of treatment(s), and your child’s overall health.

Before treatment begins, talk with your child’s doctor about possible side effects of each type of treatment your child will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of caregiving your child may need during treatment and recovery, as family members and friends often play an important role in the care of a child with AML. Learn more about caregiving.

In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies. Learn more about the importance of addressing such needs, including concerns about managing the cost of your child’s medical care

During and after treatment, be sure to tell the health care team about the side effects your child experiences, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care. Learn more by reading the After Treatment section or talking with your child’s doctor.

The next section helps explain medical tests and check-ups needed after finishing cancer treatment. Use the menu on the side of your screen to select After Treatment, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - After Treatment

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will read about your child’s medical care after cancer treatment is finished and why this follow-up care is important. To see other pages, use the menu on the side of your screen.

After treatment for AML ends, talk with your child’s doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests to monitor your child’s recovery for the coming months and years. All children treated for cancer, including AML, should have life-long follow-up care.

Based on the type of treatment your child received, the doctor will plan what examinations and tests are needed to check for long-term side effects, such as problems with the heart, lungs, or growth hormones, the development of a learning disability, and the possibility of a secondary cancer. This is a new type of cancer that develops after treatment for the first cancer. While this risk is generally low, your child should be closely monitored for their entire life for secondary cancers. Your child’s doctor can recommend the necessary screening tests. Follow-up care should also address your child’s quality of life, including any developmental or emotional concerns. Learn more about childhood cancer survivorship.

The child’s family is encouraged to organize and keep a record of the child’s medical information. That way, as the child enters adulthood, he or she has a clear, written history of the diagnosis, the treatment given, and the doctor’s recommendations about the schedule for follow-up care. The doctor’s office can help you create this. This information will be valuable to doctors who care for your child during his or her lifetime. ASCO offers cancer treatment summary forms to help keep track of the cancer treatment your child received and develop a survivorship care plan once treatment is completed.

Children who have had cancer can also enhance the quality of their future by following established guidelines for good health into and through adulthood, including not smoking, maintaining a healthy weight, eating a balanced diet, and participating in regular physical activity. Talk with the doctor about developing a plan that is best for your child’s needs.  Learn more about the next steps to take in survivorship.

The next section offers a list of questions you may want to ask. Use the menu on the side of your screen to select Questions to Ask the Doctor, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Questions to Ask the Doctor

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find some questions to ask your child's doctor or other members of your child's health care team, to help you better understand your child's diagnosis, treatment plan, and overall care. To see other pages, use the menu on the side of your screen.

Talking often with the doctor is important to make informed decisions about your child’s health care. These suggested questions are a starting point to help you learn more about your child’s cancer care and treatment. You are also encouraged to ask additional questions that are important to you and your child. You may want to print this list and bring it to your child's next appointment, or download Cancer.Net’s free mobile app for an e-list and other interactive tools to manage your child's care.

  • What type of leukemia has been diagnosed?
  • What subtype of AML has been diagnosed? What does this mean?
  • Can you explain my child’s pathology report (laboratory test results) to me?
  • What are the treatment options?
  • What clinical trials are open to my child?
  • What treatment plan do you recommend? Why?
  • What is the goal of each treatment? Is it to eliminate the cancer, help my child feel better, or both?
  • What are the chances that the AML will recur?
  • Who will be part of my child’s health care team, and what does each member do?
  • Who will be coordinating my child’s overall treatment and follow-up care?
  • What chemotherapy will my child receive?
  • Should my child receive stem cell transplantation?
  • Will my child receive radiation therapy?
  • How long will my child need to stay in the hospital?
  • What are the possible side effects of this treatment, both in the short-term and the long-term?
  • How will this treatment affect my child’s daily life? Will he or she be able to attend school and perform his or her usual activities?
  • Could this treatment affect my child’s ability to become pregnant or have children in the future? If so, should I talk with a fertility specialist before treatment begins?
  • If I’m worried about managing the costs related to my child’s cancer care, who can help me with these concerns?
  • What follow-up tests will my child need, and how often will he or she need them?
  • What support services are available to my child? To my family?
  • Whom should I call for questions or problems?

The next section offers some more resources that may be helpful to you. Use the menu on the side of your screen to select Additional Resources, or you can select another section, to continue reading this guide.  

Leukemia - Acute Myeloid - AML - Childhood - Additional Resources

This section has been reviewed and approved by the Cancer.Net Editorial Board, 01/2013

ON THIS PAGE: You will find some helpful links to other areas of Cancer.Net that provide information about cancer care and treatment. This is the final page of Cancer.Net’s Guide to Childhood AML. To go back and review other pages, use the menu on the side of your screen.

Cancer.Net includes many other sections about the medical and emotional aspects of cancer, both for the person diagnosed and their family members and friends. This website is meant to be a resource for you and your loved ones from the time of diagnosis, through treatment, and beyond. Here are a few sections that may get you started in exploring the rest of Cancer.Net:

This is the end of the Cancer.Net’s Guide to Childhood AML. Use the menu on the side of your screen to select another section, to continue reading this guide.