• Acute Myeloid Leukemia (AML) in Children

    Acute myeloid leukemia, also called acute myelogenous leukemia or AML, is a type of blood cancer. It is a quickly progressing disease in which too many abnormal white blood cells are found in the bone marrow, the soft, spongy center of long bones. In AML, myeloid stem cells (a type of blood stem cell) become immature white blood cells called myeloblasts or “blasts.” These blasts do not become healthy white blood cells. Instead, they build up in the bone marrow, so there is less room for healthy white blood cells, red blood cells and platelets. In addition, these abnormal cells are unable to fight off infection.

    Acute Myeloid Leukemia (AML) Treatment Dana-Farber/Boston Children's

    Children and teens with acute myeloid leukemia are treated at Dana-Farber/Boston Children's through our Hematologic Malignancy Center's Leukemia Program. Dana-Farber/Boston Children’s has played a key role in refining treatment for childhood leukemia, and we continue to be a world leader in leukemia clinical trials designed to increase cure rates, decrease treatment-related side effects and improve care for long-term survivors. The Leukemia Program also offers families the chance to have their child's leukemia cells molecularly profiled, which may help identify opportunities for targeted treatment.

    The Leukemia Program also partners closely with Dana-Farber/Boston Children's Stem Cell Transplant Center, one of the most experienced stem cell transplant centers in the world, to make stem cell transplantation available to children with AML.

    What is leukemia?

    Leukemia is a blood cancer. It develops in the bone marrow—the soft, spongy center of the long bones that produces the three major blood cells: 

    • white blood cells (fight infection)
    • red blood cells (carry oxygen)
    • platelets (help blood clot and stop bleeding)

    When your child has leukemia, two things happen:

    1. His bone marrow makes white blood cells that are abnormal.
    2. These abnormal cells do not respond to the signals to stop, and keep reproducing regardless of space available.
    3. The abnormal/immature white cells, called “blasts,” reproduce quickly, and—unlike normal white blood cells—do not help fight infection.
    4. When blasts begin to crowd out the healthy cells in the bone marrow, your child begins to experience symptoms of leukemia (e.g., infections, anemia, bleeding).

    How common is leukemia?

    Leukemia is the most common form of cancer in children.

    • It affects about 3,000 children each year in the United States, accounting for about 30 percent of childhood cancers.
    • Leukemia can occur at any age, although it is most commonly seen in children who are between 2 and 6 years old.
    • The disease occurs slightly more frequently in males than in females, and it is more common in Caucasian children than in children of other races.

    It’s important to understand that leukemias most often occur for no known reason, and in nearly all cases, its cause cannot be determined. There’s nothing that you could have done or avoided doing that would have prevented the disease from developing. In the majority of childhood leukemias, gene mutations and chromosome abnormalities in the leukemia cells occur by chance. The abnormalities found in leukemia cells are not found in the other cells of the body.

    What are the different types of leukemia?

    In addition to acute myelogenous leukemia (AML), there are two other types of leukemia seen in children:

    What’s the difference between ALL, AML and CML?

    Acute lymphoblastic leukemia (ALL)

    • is also called acute lymphocytic or lymphoid leukemia
    • accounts for about 80 percent of childhood leukemias
    • can occur over a short period of days to weeks
    • affects cells called lymphocytes

    Acute myeloid leukemia (AML)

    • is also called myelogenous, granulocytic, myelocytic, or myeloblastic leukemia
    • accounts for about 20 percent of childhood leukemias
    • can occur over a short period of days to weeks
    • affects cells called granulocytes (such as neutrophils or eosinophils)

    Chronic myelogenous leukemia (CML)

    • is very uncommon in children
    • can develop over a period of months or years

    These three types all have different leukemia treatments.

    What determines what kind of leukemia my child has?

    The type of leukemia (lymphoblastic or myelogenous) is determined by the type of marrow cell from which the cancer develops.

    • All blood cells start out as hematopoietic stem cells, which means they can develop into any type of blood cell. When leukemia develops from a stem cell, it is typically CML.
    • Early on, the stem cell becomes either a lymphocyte precursor cell or a myeloid precursor cell. ALL is the type of cancer that develops from a lymphocyte precursor cell. AML is the type of cancer cell that develops from myeloid precursor cells.

    What is acute myeloid leukemia (AML)?

    • In AML, the white blood cells that are affected are a specific kind called myeloid cells, or “myeloblasts.”
    • AML may be also called myelogenous, granulocytic, myelocytic, or myeloblastic leukemia.
    • It accounts for about 20 percent of the childhood leukemias.
    • With AML, these cells are most commonly found in the bone marrow and blood, but can also appear in the spinal fluid and the skin.
    • Rarely, they can form into solid tumors called chloromas.
    • The symptoms associated with AML usually occur over a short period of days to weeks.

    What causes AML in children?

    In nearly all cases, it’s not known what causes leukemia. In the majority of childhood leukemias, gene mutations and chromosome abnormalities in the leukemia cells occur sporadically (by chance). The abnormalities found in leukemia cells are not found in the other cells of the body.

    Are there any risk factors associated with AML?

    If your child has certain genetic syndromes, including Fanconi anemia, Bloom syndrome, Kostmann syndrome, and Down syndrome, he may be at a higher risk of developing AML than other children. Myelodysplastic syndrome (MDS) or other forms of bone marrow disease can also place your child at risk of developing AML, as can treatment for a previous cancer (though this is rare in children).

    What are the symptoms of AML in children?

    Because leukemia is cancer of the blood-forming bone marrow, the initial symptoms are often related to abnormal bone marrow function. While each child may experience symptoms differently, some of the most common include:

    • anemia – When your child’s bone marrow is too crowded for red blood cells to be produced, anemia is present. Your child may appear tired, pale and may breathe faster to compensate for the decrease in his cells’ ability to carry oxygen. A blood count will show fewer than normal red blood cells.
    • bleeding and/or bruising – When your child’s marrow is too crowded to allow platelets to be produced, bleeding can occur, and your child may begin to bruise more easily. You might notice petechia—tiny red dots often seen on the skin when a child has a low number of platelets. These are very small blood vessels that have "leaked" or bled. A blood count will show thrombocytopenia, a decreased number of platelets.
    • recurrent infections – Although there may be an unusually high number of white blood cells on your child’s blood count, these white blood cells are immature and do not fight infection. Your child may have had several viral or bacterial infections over the past few weeks and may show symptoms of an infection such as fever, runny nose and cough.
    • bone and joint pain – Your child may also experience pain in his bones and joints. This pain is usually a result of the bone marrow being overcrowded and "full."
    • abdominal pain – stomach aches may also be a symptom of leukemia. Leukemia cells can collect in your child’s kidney, liver and spleen, causing enlargement of these organs. Pain in the abdomen may cause your child to lose his appetite and lose weight.

    With AML, these symptoms usually occur over a number of days or weeks.
    It’s important to understand that the symptoms of leukemia may resemble other blood disorders or medical problems. These are common symptoms of the disease, but do not include all possible symptoms. Always consult your child's physician for a diagnosis.

    How is childhood AML diagnosed?

    There are many diagnostic procedures that may be used to determine whether your child has AML. In addition to a complete medical history and physical examination, some of these may include:

    • complete blood count (CBC) – a measurement of size, number and maturity of different blood cells in a specific volume of your child’s blood
    • additional blood tests – may include blood chemistries, evaluation of liver and kidney functions, and genetic studies
    • bone marrow aspiration and biopsy – Marrow may be removed by aspiration or a needle biopsy under local anesthesia. In aspiration biopsy, a fluid specimen is removed from the bone marrow. During a needle biopsy, marrow cells are removed. These methods are often used together. Most often, samples are obtained from the bones of your child’s pelvis. Doctors perform several additional tests on these samples (flow cytometry, chromosomal analysis, etc.) that help determine the exact kind of leukemia your child has and guide how we tailor our treatment approach.
    • spinal tap/lumbar puncture – A special needle is placed into the lower back, into the spinal canal. This is the area around the spinal cord. The pressure in your child’s spinal canal and brain can then be measured. A small amount of cerebral spinal fluid (called CSF—the fluid that bathes your child’s brain and spinal cord)—can be removed and tested.

    What type of childhood AML treatment should we expect?

    There are a number of treatments that your child’s physician may recommend. Some of them help to treat the leukemia, while others are intended to address complications of the disease or side effects of the treatment.

    Your child’s physician will determine a specific course of treatment for childhood leukemia based on several factors, including:

    • your child's age, overall health and medical history
    • the type of AML
    • certain aspects of the AML, such as the number of white blood cells in the blood when the leukemia is diagnosed, whether or not leukemia cells are seen in the spinal fluid, and whether or not specific chromosomal abnormalities are found in the leukemia cells
    • how well the leukemia responds to the first few weeks of treatment
    • your child's tolerance for certain medications, procedures or therapies
    • whether your child has Down syndrome
    • how your child's physician expects the disease to progress

    Treatment usually begins by addressing your child’s symptoms, such as anemia, bruising and/or fever. In addition, treatment may include the following (alone or in combination):

    Chemotherapy is a drug treatment that interferes with the cancer cell's ability to grow or reproduce. For some types of cancer, chemotherapy is used alone, while in others it is used in conjunction with other therapy, such as radiation or surgery.

    Stem cell transplant
    A stem cell (bone marrow) transplant involves the replacement of diseased bone marrow with another person’s healthy bone marrow.

    The success of a transplant depends on many factors, such as how close the match is between child and donor (matched siblings provide the closest match; if your child has a sibling, there is a 1 in 4 chance they will be a match) and the patient's age. The degree to which your child's bone marrow has failed also factors into the discussion about whether to pursue a transplant.

    The decision to proceed with bone marrow transplant should be discussed with your child’s doctor and a stem cell transplant team.

    Other medications
    Other medications may be given to prevent or treat damage to other systems of your child’s body caused by leukemia treatment or for nausea and side effects of treatment.

    Blood transfusions
    Blood transfusions replace or supplement your child’s red blood cells and platelets.

    Your child may be given antibiotics to prevent or treat infections.

    Continuous follow-up care
    Your child’s health will be closely monitored to determine response to treatment, detect recurrent disease and manage late effects of treatment.

    What are the phases of AML treatment in children?
    In general, phases in the treatment of AML include:

    1. Induction
    Induction is the first phase of treatment. It is a combination of chemotherapy and medications given to stop abnormal cells from being made in the bone marrow.

    The goal of this phase is to induce the cancer to go into remission. This means leukemia cells are no longer visible under the microscope when examining either the bone marrow or blood.

    The induction phase may last approximately one month and is usually followed by a second course of induction treatment regardless of whether a remission is achieved. It can be repeated if remission is not achieved.

    However, even when remission is achieved, leukemia is still present in the body, which is why further therapy is necessary.

    2. Intensification or consolidation
    Intensification or consolidation is continued treatment to kill any leukemia cells remaining after induction. In some cases, further chemotherapy is recommended, while in others we recommend a stem cell transplant.

    Your care team will work with you to determine the best course of treatment.

    How is AML treatment personalized for my child?

    Our multidisciplinary Leukemia Program ensures in-depth discussion of each case and personalized treatment plans for every patient. Your child will receive integrated care from a team that includes the following specialists:

    In addition, our cancer center offers the following services:

    • Expert diagnosis by pathologists using advanced molecular diagnostic testing to identify your child’s type of tumor. Knowing the molecular composition of a tumor helps predict which treatments are more likely to work.
    • Access to unique Phase I clinical trials—from our own investigators and from the Children’s Oncology Group.

    Support services to address all of your child and family’s needs.

    What is the long-term outlook for my child with AML?

    Your child’s AML prognosis greatly depends on:

    • genetic abnormalities and other features of the leukemia
    • the cancer’s response to treatment
    • his age and overall health
    • his tolerance of specific medications, procedures or therapies
    • new developments in treatment

    Relapse can occur during any stage of treatment, even with aggressive therapy, or may occur months or years after treatment has ended. Overall, between 60 and 70 percent of children treated for AML will be long-term survivors; for some specific types, the survival rate exceeds 80 percent.

    As with any cancer, prognosis and long-term survival can vary greatly. Prompt medical attention and aggressive therapy are important for the best prognosis, and continuous follow-up care is essential. New methods are continually being discovered to improve treatment and decrease side effects of the treatment for the disease.

    Survivorship care is important for patients with AML in remission. Our weekly survivorship clinic, which set the national standard for childhood cancer survivorship care, offers ongoing care to manage late effects caused by your child’s cancer or the treatment they received.

    What is the latest research on childhood AML?

    Research is a top priority at Dana-Farber/Boston Children's, and our physicians work continuously to translate laboratory findings into clinical therapies.

    New research from Dana-Farber/Boston Children's and the Broad Institute of Harvard and MIT integrates sophisticated interdisciplinary approaches to solve a molecular mystery that may lead to alternative therapeutic strategies for AML. The study identifies a previously unrecognized AML target that responds well to pharmacological inhibition and may be an excellent candidate for use in future clinical trials.

    In the area of stem cell transplantation, the Dana-Farber/Boston Children's Stem Cell Transplant Center is investigating the use of umbilical cord transplantation.

    Also under study is a novel method for preventing graft versus host disease, a serious complication that occurs when transplanted cells do not recognize the tissues and organs of the recipient's body and react against the recipient's tissue. The result of this treatment approach, if it continues to be as successful, will be that the degree of match between donor and recipient will not need to be particularly close, greatly increasing the pool of potential donors for each patient. This could also eliminate the need for long-term drug therapy traditionally needed to treat graft versus host disease.

    Clinical trials

    It's possible that your child may be eligible to participate in one of our Hematologic Malignancy Program's pediatric leukemia clinical trials. We also offer access to trials through collaborative groups such as the Children's Oncology Group (COG), the Pediatric Oncology Experimental Therapeutics Investigators' Consortium (POETIC) and the Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL) consortium. Should you have questions or need advice on whether a particular trial would be appropriate for your child, email our clinical trials team at clinicaltrials@danafarberbostonchildrens.org. We can help you navigate your options.

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