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Aplastic Anemia

  • Normally, bone marrow produces all of the blood cells your child’s body needs. Aplastic anemia is a blood disorder that occurs when the bone marrow fails to make enough of all types of blood cells: red cells, white cells and platelets. Aplastic anemia was once fatal in many cases. However, treatment for children with the condition has improved significantly due to innovative new therapies, including stem cell transplantation and effective supportive care.

    How Dana-Farber/Boston Children's approaches aplastic anemia

    At Dana-Farber/Boston Children's Cancer and Blood Disorders Center, our Bone Marrow Failure Program provides comprehensive treatment for children with aplastic anemia. We are a national pediatric hematology oncology referral center, with one of the most experienced, multidisciplinary teams in the diagnosis and treatment of aplastic anemia.

    • We offer specialized diagnostic and treatment options, including hematopoietic stem cell transplantation.
    • We offer direct referral to Dana-Farber/Boston Children’s Pediatric Stem Cell Transplant Program, one of the nation’s oldest and most experienced pediatric stem cell transplant programs.
    • Our pediatric hematology experts provide your child with ongoing, comprehensive follow-up care to manage the symptoms of aplastic anemia.

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  • What is aplastic anemia?

    Aplastic anemia is a blood disorder that affects three out of every 1 million people in the United States each year. It occurs when the bone marrow produces too few of all types of blood cells: red cells, white cells and platelets.

    • A low number of red blood cells reduces the blood’s ability to carry oxygen.
    • A reduced number of white blood cells makes the child more susceptible to infection.
    • A low number of platelets reduces the blood’s ability to clot.


    What causes aplastic anemia?

    Aplastic anemia in children has multiple causes. Usually it is “idiopathic,” occurring with no known reason. It also can be the result of a previous illness, existing problem or can be caused by an inherited genetic disorder.

    About 80 to 85 percent of cases of aplastic anemia in children are acquired and may include a history of:

    • specific infectious diseases, such as hepatitis, Epstein-Barr virus or cytomegalovirus
    • taking certain medications, including some antibiotics and arthritis drugs
    • exposure to certain toxins, such as benzene, pesticides and insecticides
    • exposure to radiation or chemotherapy

    In about 15 to 20 percent of cases, children inherit a disorder that predisposes them to developing aplastic anemia, such as:


    What are the symptoms of aplastic anemia?

    Each child may experience symptoms differently, but the most common symptoms of aplastic anemia are:

    • lack of energy or tiring easily
    • pale skin, lips and hands or paleness under the eyelids
    • shortness of breath
    • fevers or infections bleeding, such as bruising, bleeding gums, nosebleeds or blood in the stool
    • irregular heartbeat
    • dizziness or headache

    The symptoms of aplastic anemia may resemble other blood disorders or medical problems, some of which are very common and easy to treat, while others are more serious. The symptoms listed above are common presentations of the disease, but do not include all possible symptoms.

    It is important to be evaluated by a physician to obtain an accurate diagnosis. Always consult your child's physician if you have concerns.

  • Your child’s physician may order a number of different tests to make a diagnosis of aplastic anemia.

    In addition to a complete medical history and physical examination, aplastic anemia can only be diagnosed accurately by a full evaluation of the blood and bone marrow, which includes some or all of the following tests:

    • blood tests—A complete blood count (CBC) can help your child’s doctor to understand the cause of the anemia. In aplastic anemia, this test shows an abnormally low number of all blood cells.
    • bone marrow exam—Using a needle, samples of the fluid (aspiration) and solid (biopsy) portions of bone marrow are withdrawn under local or general anesthesia. This allows your child’s doctor to look for marrow abnormalities. This test is necessary to make a definitive diagnosis of aplastic anemia.
    • other tests—Additional blood tests and genetic testing may be ordered to rule out certain inherited types of anemia and other disorders, such as myelodysplastic syndrome.

    After we complete all necessary tests, our experts meet to review and discuss what they have learned about your child's condition. Then, we will meet with you and your family to discuss the results and outline the best treatment options for your child’s aplastic anemia

  • Children with aplastic anemia are treated through the Bone Marrow Failure Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, an integrated pediatric hematology and oncology partnership between Dana-Farber Cancer Institute and Boston Children’s Hospital. Working together, we provide more specialists, more programs, and more clinical trials than almost any other pediatric center treating cancer and blood disorders in the world.

    Your child’s physician will determine a specific course of aplastic anemia treatment based on several factors, including:

    • your child's age, overall health and medical history
    • your child’s severity of symptoms
    • your child's tolerance for specific medications, procedures or therapies
    • how your child's doctors expect the disease to progress
    • the availability of a suitable stem cell donor

    What are the treatments available for aplastic anemia in children?

    Treatment for pediatric aplastic anemia may include:

    Stem cell transplant:

    • A hematopoietic stem cell transplant can be a cure when the transplanted stem cells come from a matched sibling donor. However, a matched sibling donor may not be available in many cases.
    • Hematopoietic stem cells from a matched unrelated donor or cord blood also may be considered under certain circumstances, such as when immunosuppressive drug therapy is ineffective.

    Treating an underlying disorder:

    • Some inherited causes of bone marrow failure must be ruled out as they are treated in different ways.
    • In the rare case that aplastic anemia is caused by an underlying disorder, we identify and treat that condition.

    Immunosuppressive drug therapy:

    • These drugs act on the bone marrow, allowing it to make blood cells again.
    • This is the standard drug therapy used when a matched sibling donor is not available.

    Supportive care:

    Our physicians also use supportive care in conjunction with treatment to help relieve a child’s symptoms, such as:

    • blood transfusion, including both red cells and platelets
    • preventive antibiotic therapy
    • medications that stimulate the body to make more white blood cells

    If your child receives blood transfusions, she may eventually require additional medication to prevent iron from building up in the blood. This is called chelation therapy.

    Lifestyle and alternative therapies

    • We teach your child and family behavioral interventions—including thorough hand-washing and avoiding large crowds and people who have fevers or infections—because children with aplastic anemia are more susceptible to infection.
    • Patients should avoid excessive exercise or contact sports because low platelet counts can increase the risk of trauma-induced bleeding.
    • Aplastic anemia is not caused by vitamin or nutrient deficiencies and, as a result, supplements and herbal treatments are not effective in treating the condition. Some may even be harmful.

    Resources & support

    In addition to providing medical care, we strive to also meet all of your family’s emotional and quality-of-life needs. Our family support services will help address many of your needs and concerns. Read about our resources and support services.

  • Dana-Farber/Boston Children's Cancer and Blood Disorders Center researchers are actively collecting voluntary samples of blood and bone marrow from patients with aplastic anemia and other bone marrow failure syndromes to better understand genetic contributions and molecular aspects of the diseases. 

    Currently, researchers are exploring possible genetic factors that put children at risk of developing aplastic anemia. Using skin cells and bone marrow cells from patients with aplastic anemia, they will use specialized techniques to compare their genetic material and look for genetic abnormalities in the bone marrow cells.

    More about clinical trials

    For many children with rare or hard-to-treat conditions, clinical trials provide new options.

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Dana-Farber/Boston Children's Cancer and Blood Disorders Center

The future of pediatrics will be forged by thinking differently, breaking paradigms and joining together in a shared vision of tackling the toughest challenges before us.”
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