• Leukemia in Children

    Leukemia is cancer of the blood and develops in the bone marrow. It is the most common form of cancer in children.

    • Leukemia affects about 3,800 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 common in children between two and six years old.
    • The disease occurs slightly more frequently in boys than in girls, and is more common in Caucasian children than in children of other races.

    Types of Leukemia in Children

    • Acute lymphoblastic leukemia (ALL) (also called lymphocytic or lymphoid leukemia) is the type of leukemia that affects cells called lymphocytes. ALL most commonly affects children, most often between the ages of two and three, but it also affects adults. ALL accounts for about 80 percent of childhood leukemia cases each year in the U.S. It develops quickly, over a period of days or weeks.
    • Acute myeloid leukemia (AML) (also called myelogenous, granulocytic, myelocytic, or myeloblastic leukemia) affects cells called granulocytes (such as neutrophils or eosinophils). It is the second most common form of leukemia in children and accounts for about 20 percent of all childhood leukemia cases in the U.S. AML generally occurs by the age of two years and is not often seen in older children until the teenage years. AML is the most common type of acute leukemia in adults. Like ALL, it develops quickly, over days or weeks.
    • Chronic myeloid leukemia (CML) is very rare in children. Unlike ALL and AML, it develops slowly, over a period of months or years.
    • Juvenile myelomonocytic leukemia (JMML) is a rare type of blood cancer that occurs when bone marrow production of white blood cells becomes severely disregulated. It is sometime categorized as a myeloproliferative neoplasm (a blood disorder in which abnormal bone marrow cells produce too many blood cells) instead of a cancer. JMML accounts for only about one to two percent of childhood leukemia cases, and it mainly affects children younger than four years old.

    Leukemia Treatment at Dana-Farber/Boston Children's

    Children and teens with 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.

    What causes leukemia in children?

    Leukemia is cancer of the blood and develops in the bone marrow. The bone marrow is the soft, spongy center of the long bones that produces the three major blood cells: white blood cells to fight infection; red blood cells that carry oxygen; and platelets that help the blood clot and stop bleeding. When a child has leukemia, the bone marrow, for an unknown reason, begins to make white blood cells that do not mature correctly but continuously reproduce. Normal, healthy cells only reproduce when there is enough space for them to fit.

    The body can regulate the production of cells by sending signals when to stop. With leukemia, these cells do not respond to the signals to stop reproducing, regardless of space available. These abnormal cells reproduce very quickly and do not function as healthy white blood cells to help fight infection. When the immature white blood cells, called blasts, begin to crowd out other healthy cells in the bone marrow, the child experiences the symptoms of childhood leukemia.

    In nearly all cases, it is not known what causes leukemia. The majority of childhood leukemias are acquired genetic diseases. This means that gene mutations and chromosome abnormalities in leukemia cells occur sporadically (by chance). The abnormalities found in leukemia cells are not found in the other cells of the body.

    What role does the immune system play in leukemia?

    The immune system plays an important role in protecting the body from disease and possibly cancer. An alteration or defect in the immune system may increase the risk for developing leukemia. Factors such as exposure to certain viruses, environmental factors, chemical exposures, and infections have been associated with damage to the immune system, but none of these factors has been definitively linked as a cause of childhood leukemia.

    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 determines the type of leukemia my child has?

    The type of childhood leukemia (lymphoblastic or myeloid) is determined by the type of bone 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 chronic myelogenous leukemia (CML).

    Early in its development, the stem cell becomes either a lymphocyte precursor cell or a myeloid precursor cell. Acute lymphoblastic leukemia (ALL) is the type of cancer that develops from a lymphocyte precursor cell. Acute myelogenous leukemia (AML) is the type of cancer cell that develops from myeloid precursor cells.

    How is childhood leukemia diagnosed?

    In addition to a complete medical history and physical examination, leukemia diagnostic procedures may include:

    • 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. In a needle biopsy, marrow cells (not fluid) are removed. These methods are often used together. Most often samples are obtained from the bones of the pelvis (upper buttocks).
    • Complete blood count (CBC) – a measurement of size, number, and maturity of different blood cells in a specific volume of blood.
    • Additional blood tests – may include blood chemistries, evaluation of liver and kidney functions, and genetic studies.
    • Computerized tomography scan (also called a CT or CAT scan) – a diagnostic imaging procedure that uses a combination of x-rays and computer technology to produce cross-sectional images.
    • Magnetic resonance imaging (MRI) – a diagnostic procedure that uses a combination of large magnets, radiofrequencies, and a computer to produce detailed images of organs and structures within the body.
    • X-ray – a diagnostic test that uses invisible electromagnetic energy beams to produce images of internal tissues, bones, and organs onto film.
    • Ultrasound (also called sonography) – a diagnostic imaging technique that uses high frequency sound waves and a computer to create images of blood vessels, tissues, and organs. Ultrasounds are used to view internal organs as they function and to assess blood flow through various vessels.
    • Lymph node biopsy
    • 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 the spinal canal and brain can then be measured. A small amount of cerebrospinal fluid (CSF) can be removed and sent for testing to determine if there is an infection or other problems. CSF is the fluid that bathes your child's brain and spinal cord.

    What are leukemia treatment options for children?

    The specific leukemia treatment for your child will be determined by your child's physician based on:

    • Your child's age, overall health, and medical history
    • The extent of the disease and specific sub-type of leukemia
    • Your child's tolerance for certain medications, procedures, or therapies
    • Expectations for the course of the disease
    • Your opinion or preference

    Leukemia treatment usually begins by addressing the presenting symptoms such as anemia, bleeding, and/or infection. In addition, leukemia treatment may include (alone or in combination):

    • Chemotherapy/intrathecal medications (medications are inserted through a needle into the spinal cord into the area called the subarachnoid space)
    • Radiation therapy – although never used as part of induction therapy
    • Bone marrow (stem cell) transplantation – stem cell transplant is almost never used to treat ALL, except for certain rare subtypes or if the leukemia comes back after initial treatment with chemotherapy (and sometimes radiation)
    • Biological therapy – only rarely used for the treatment of ALL
    • Medications (to prevent or treat damage to other systems of the body caused by leukemia treatment; also for nausea and side effects of treatment)
    • Blood transfusions (red blood cells, platelets)
    • Antibiotics (to prevent/treat infections)
    • Continuous follow-up care (to determine response to treatment, detect recurrent disease, and manage late effects of treatment)

    What is relapse?

    Relapsed leukemia may occur even with aggressive therapy. Relapse is when the bone marrow begins making abnormal cells again. Relapse can occur during any of the stages of treatment or may occur months or years after treatment has ended. (Learn about relapsed acute lymphoblastic leukemia.)

    What are the various stages of leukemia treatment?

    There are various stages in the leukemia treatment, including the following:

    • Induction – a combination of chemotherapy and medications given to stop the process of abnormal cells being made in the bone marrow. Remission means the leukemia cells are no longer visible under the microscope when examining either the bone marrow or blood. However, even when remission is achieved, leukemia is still present in the body, which is why further treatment is necessary. The induction phase may last approximately one month and can be repeated if remission is not achieved.
    • Intensification or consolidation – continued treatment with chemotherapy to kill leukemia cells. Even though leukemia cells are not visible in a blood test or bone marrow examination, they are still present in the body. Therapy in this phase may include chemotherapy given to the central nervous system to kill any leukemia cells present there, or to prevent the spread of leukemia cells to the spinal cord or brain. Radiation therapy may also be given to the brain during this phase.
    • Maintenance or continuation – the goal of this phase is to eradicate all leukemia from the body. Usually, less intensive chemotherapy is used, much of which can be given at home. This phase can last months to several years. Regular visits to your child's physician are required in order to determine response to treatment, detect any recurrent disease, and manage any side effects of the treatment.

    What is the long-term outlook for children with leukemia?

    Prognosis greatly depends on:

    • The extent of the disease
    • Disease response to treatment
    • Genetic abnormalities of the leukemia
    • Age and overall health of your child
    • Your child's tolerance of specific medications, procedures, or therapies
    • New developments in treatment

    As with any cancer, prognosis and long-term survival can vary greatly from child to child. Prompt medical attention and aggressive therapy are important for the best prognosis. 

    Cancer survivorship

    Continuous follow-up care is essential for any child diagnosed with leukemia. Side effects of radiation and chemotherapy, as well as second malignancies, can occur in survivors of leukemia. New methods are continually being discovered to improve treatment and to decrease side effects of the treatment for the disease.

    Survivorship care is important for patients with leukemia in remission. Our weekly cancer 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.

  • Top Ranked for Cancer Care

    U.S. News Best Children's Hospitals logoWorld class cancer care at the #1 children's hospital

  • Video: Leukemia Treatment and Research

    Kimberly Stegmaier, MD, discusses current research aimed at finding new drug targets for children with leukemia and other cancers.

  • Leukemia Clinical Trials

    We offer promising experimental therapies for all types of leukemia.callout bg
  • Video: CAR T-Cell Therapy

    CAR (chimeric antigen receptor) T-cell therapy is a promising new treatment for some of the most challenging cases we face in pediatric acute lymphoblastic leukemia (ALL).