Leukemia - Acute Myeloid - AML - Childhood

Treatment Options

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.

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. 

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.

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. 

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. 

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. 

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. 

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. 

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. 

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. 

Clinical Trials

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.

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. 

After Treatment

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. 

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. 

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. 

Coping with Side Effects

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.  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. 

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. 

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.

Latest Research

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. 

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.