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 contain too many white blood cells, and the child has signs and symptoms of the disease.
Complete Remission AML. The number of cancerous blast cells in the bone marrow is too few to be distinguished from normal blasts under the microscope (fewer than 5% blasts in the bone marrow). The child usually does not have any signs or symptoms of the disease.
Partial Remission AML. The number of cancerous blast cells in the bone marrow is reduced (between 5% and 15% in the bone marrow) but still recognizable under the microscope. The child usually does not have any signs or symptoms of the disease. This applies only during the initial course of therapy called induction (see below).
Recurrent AML. The disease has come back after the child experienced a period of remission (complete absence of symptoms).
Refractory AML. The leukemia did not go into complete remission after treatment.
Treatments for children with AML
Two types of treatment are commonly used to treat AML in children: chemotherapy and stem cell transplantation/bone marrow transplantation; both are described below in detail. Radiation therapy is occasionally used in specific situations. Sometimes, more than one treatment is used.
Clinical Trials are the standard of care for the treatment of children with cancer. In fact, 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 treatments available) with newer treatments that may be more effective. Cancer in children is rare, so it can be hard for doctors to plan treatments unless they know what has been most effective in other children. Investigating new treatments involves careful monitoring using scientific methods, and all participants are followed closely to track 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. Many times, a team of doctors treats a child with cancer. Pediatric cancer centers often have extra support services for children and their families, such as nutritionists, social workers, and counselors. Special activities for kids with cancer 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.
Chemotherapy
Chemotherapy is the use of drugs to kill cancer cells. Systemic chemotherapy is delivered through the bloodstream, targeting cancer cells throughout the body. Chemotherapy primarily kills cancer cells because they are the most rapidly dividing cells. Chemotherapy is the most common treatment for AML.
Several treatment plans have been developed that involve intensive use of several drugs. Following these plans, about 85% of children will have an initial remission, and about 50% will be cured. During treatment, children with AML need to be monitored very carefully and often spend many weeks in the hospital because of very low blood counts and the possibility of developing infections.
Chemotherapy may be given by mouth, injected into a vein, or injected into the cerebrospinal fluid (CSF). The choice of drugs depends on whether the child has previously been treated for AML and other factors.
Chemotherapy for AML is usually divided into two phases of treatment: induction and intensification.
Induction chemotherapy uses chemotherapy to kill as many of the cancer cells as possible and cause the AML to go into remission.
Intensification chemotherapy is used to kill any cancer cells remaining after induction chemotherapy. Stem cell transplantation also can be used for intensification therapy.
The side effects of chemotherapy depend on the individual and the dose used, but can include fatigue, low blood counts, risk of infection, hair loss, nausea and vomiting, 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 processed by the body and the child’s overall health and well-being. Doctors understand that everyone is unique. 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 complications. Your doctor will discuss these changes with you as they become necessary. Not everyone needs to have their chemotherapy changed.
The medications used to treat cancer are continually being evaluated. Talking with your 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 through Cancer.Net's Drug Information Resources, which provides links to searchable drug databases.
Stem cell transplantation/bone marrow transplantation
Children with AML have different risks of recurrence depending on the subtype. The higher a risk for recurrence, the greater the consideration to use a stem cell transplantation.
Not all children with AML need stem cell transplantation. For instance, children with Down syndrome and M3 acute promyelocytic leukemia 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]) is not treated with stem cell transplantation unless the leukemia has recurred. For children with subtypes of AML that have lower risks of recurrence and therefore higher chances of cure, most can be treated with chemotherapy alone.
A stem cell transplant is a medical procedure in which diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are the cells that normally become bone marrow. They are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant as stem cells from either the blood or the bone marrow may be used.
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 used mainly for AML. AUTO stem cell transplantation is not a standard treatment for AML in the United States except for patients with recurrent M3 acute promyelocytic leukemia.
In an ALLO transplant, stem cells are obtained from a donor whose tissue matches the patient’s on a genetic level; this testing is called HLA-typing. Most often, a patient’s brother or sister serves as the donor, although unrelated donors can serve as the donor too. Millions of people worldwide have volunteered to donate stem cells for patients who do not have matched family members; matches can be made by searching a computer registry. In addition, a donation of stem cells derived from umbilical cord blood is sometimes considered if family donors are not available.
For children with an average (also called intermediate) risk for recurrence and survival, when a related donor is available, stem cell transplantation is the preferred choice of treatment after a child has a first remission. For children with the highest risk of recurrence and the poorest chance for survival, 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 general, transplantations from unrelated donors are not performed unless the AML has recurred. 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 have a complete or partial remission after their first course of induction chemotherapy).
In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are then frozen until they are needed, usually after the high-dose treatment (explained below) is completed.
In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and have replacement blood stem cells 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. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system so that, in an ALLO transplant, the donor cells are not rejected by the body. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts from donor cells. Sometimes, ALLO transplants can also be performed after giving lower doses of chemotherapy and/or radiation therapy that are still sufficient to suppress the immune system and allow growth of the donor cells. (These transplants, sometimes termed “mini-transplants” or “reduced intensity transplants” have less immediate side effects, allowing the procedure to be used for older patients.)
Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and patient’s age and general health.
For both ALLO and AUTO transplant types, the replacement cells engraft (begin to make new blood cells) and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the patient’s immune system is back to normal, patients may need antibiotics and blood transfusions.
In an ALLO transplant, another major risk is that the donor’s cells will recognize the patient’s body as foreign, causing graft-versus-host disease (GVHD). GVHD may be a serious complication of allogeneic transplants and can be fatal. Other side effects may include liver problems, diarrhea, infections, and rashes. However, GVHD can also be a benefit, in that the donor cells can recognize the cancer cells as foreign and destroy these cells, a mechanism that is one of the major reasons why ALLO transplantation generally works so well over the long term. The risk of GVHD can be reduced with exact HLA-type matching and the use of preventative drugs.
In an AUTO transplant, there is little risk of GVHD because the replacement stem cells are the patient’s own cells. However, there is a risk in an autologous transplant that some of the cells that are put back into the patient could still be cancerous.
Learn more by reading the Cancer.Net Feature series Understanding 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. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. 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. 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 can be found in the After Treatment section.
Recurrent AML
For some children who received treatment for AML, the disease comes back after treatment. Most recurrences occur in the bone marrow, but sometimes it may come back in the brain or other parts of the body. Treatment usually includes chemotherapy followed by stem cell transplantation. Each child’s treatment is planned individually depending on the circumstances. Clinical trials testing new targeted therapies for AML are an option for some children with recurrent AML. For some children, recurrent AML can be cured.
To learn about the terms used in this section, read the Cancer.Net Feature: Cancer Terms to Know: During Treatment.
Printer-Friendly
E-mail this page