• Aplastic Anemia Overview

    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. This can reduce the blood’s ability to carry oxygen (low red cells), make a child more susceptible to infection (low white cells), and impair the blood’s ability to clot (low platelets).

    About 80 to 85 percent of aplastic anemia cases in children are acquired and may include a history of infectious diseases, taking certain medications, exposure to toxins, or exposure to radiation therapy or chemotherapy. In about 15 to 20 percent of cases, children inherit a disorder that predisposes them to developing aplastic anemia, including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome, and amegakaryocytic thrombocytopenia.

    Aplastic Anemia Treatment at Dana-Farber/Boston Children's

    Children and young adults with aplastic anemia are treated at Dana-Farber/Boston Children's Cancer and Blood Disorders Center through our Bone Marrow Failure Program. Continue reading to learn more about aplastic anemia or visit the Bone Marrow Failure Program homepage to learn about our expertise with this condition.

    Symptoms & Diagnosis

    The symptoms of aplastic anemia may resemble those of other blood disorders or medical problems. The most common symptoms are:

    • Lack of energy or tiring easily
    • Pale skin, lips and hands, or paleness under the eyelids
    • Shortness of breath
    • Fever
    • Infection
    • Bleeding, such as bruising, bleeding gums, nosebleeds or blood in the stool
    • Irregular heartbeat
    • Dizziness or headache

    In addition to a complete medical history and physical examination, aplastic anemia can be diagnosed through a full evaluation of the blood and bone marrow, including:

    • Blood tests, including complete blood count
    • Bone marrow exam, including aspiration and biopsy

    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 all tests are completed, doctors will be able to outline the best treatment options.

    Treatment & Care Options

    The treatment of aplastic anemia has improved significantly due to innovative new therapies; however, it is a serious condition requiring extensive ongoing care. Treatment for aplastic anemia may include:

    • Stem cell transplant 
    • Treatment of an underlying disorder
    • Immunosuppressive drug therapy, which acts on the bone marrow and allows it to make blood cells again
    • Blood transfusion, including both red cells and platelets
    • Preventive antibiotic therapy

    Progressive or Recurrent Disease

    For some people with aplastic anemia, treatment improves blood counts, but the disease never completely goes away. If aplastic anemia recurs, it is often in the first year after treatment.

    Long-term Outlook

    Aplastic anemia was once fatal in many cases. Today, innovative therapies like stem cell transplant can cure aplastic anemia when blood stem cells come from a matched sibling donor. Children with aplastic anemia have a higher risk of developing complications from a stem cell transplant, including graft-versus-host disease.
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