• Childhood Aplastic Anemia

    What is Aplastic Anemia?

    All blood cells are produced by the bone marrow, and aplastic anemia occurs when the bone marrow produces too few of these cells. The bone marrow produces red blood cells, white blood cells and platelets. Red blood cells contain the protein hemoglobin (Hb) that carries oxygen from the lungs to tissues. Too few red blood cells leads to a decrease in Hb and symptoms of tiredness. White blood cells fight infection. A reduced number of neutrophils, a major type of white blood cell that are important for defense against bacterial and fungal infections, causes increased susceptibility of infection. Platelets are important for blood to clot, and a reduced number leads to increased risk of bleeding or bruising.

    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. Dana-Farber/Boston Children's is also home to one of the largest and most experienced pediatric stem cell transplant centers in the world. Stem cell (bone marrow) transplant is currently the only cure for aplastic anemia.

    What causes aplastic anemia?

    Aplastic anemia in children has multiple causes, but many causes are “idiopathic”, meaning they occur for no known reason. Other causes are secondary, resulting from a previous illness or disorder.

    Acquired causes may include: a history of specific infectious diseases such as hepatitis, Epstein-Barr virus (EBV), cytomegalovirus (CMV), or human immunodeficiency virus (HIV), a history of taking certain medications, exposure to certain toxins such as heavy metals, exposure to radiation, or a history of an autoimmune disease such as lupus.

    Children may also inherit a disorder that predisposes them to developing aplastic anemia. Some examples include:

    What are the symptoms of aplastic anemia?

    The symptoms of aplastic anemia may resemble those of other blood disorders or medical problems. Each child may experience symptoms differently, but the most common symptoms of aplastic anemia include:

    • Lack of energy or tiring easily (fatigue)
    • Shortness of breath
    • Pale skin, lips, and hands, or paleness under the eyelids
    • Bruising, or a red or purple pinpoint rash on the face or body
    • Bleeding (for example bleeding gums, nosebleeds, blood in the stool)
    • Fevers/infections

    How is aplastic anemia diagnosed?

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

    • Blood tests, including a complete blood count
    • Bone marrow aspiration and biopsy — marrow may be removed by aspiration and a needle biopsy under local anesthesia. In a bone marrow aspiration, 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.

    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 meet with you and your family to discuss the results and outline the best treatment options for your child’s aplastic anemia.

    What is the treatment for aplastic anemia?

    Specific treatment for aplastic anemia will be determined by your child’s physician based on:

    • Your child’s age, overall health, and medical history
    • Extent of the disease
    • Your child’s tolerance for specific medications, procedures, or therapies
    • Expectations for the course of bone marrow failure
    • Your opinion or preference

    Treatment options include, but are not limited to the following:

    • Stem Cell (Bone Marrow) Transplant: At present, this is the only cure for aplastic anemia. Bone marrow transplant involves the replacement of diseased bone marrow with another person’s healthy bone marrow. Unfortunately it may not be an option for everyone. The decision to proceed with bone marrow transplant should be discussed with your child’s hematologist and a stem cell transplant team
    • Immunosuppressive Therapy: This is the standard drug therapy for aplastic anemia. It is based on the theory that aplastic anemia is caused by the immune system reacting against the bone marrow. Therefore, immunosuppressive drugs “put down” the immune response and allow the bone marrow to make blood again.
    • Treating an underlying disorder: In rare cases aplastic anemia is caused from a previous illness or disorder. If this problem can be identified and treated, there are cases in which the aplastic anemia will improve.
    • Additional treatment alternatives are currently being studied.
    • Supportive Care: In addition to treatment, supportive care helps the symptoms of aplastic anemia (fatigue, bleeding, infections, etc.). Examples include blood transfusion (both red cells and platelets), preventative antibiotic therapy, medications that stimulate the body to make more white blood cells, and behavioral interventions like thorough hand washing and avoiding large crowds or people who are known to have fevers or illness.

    What is the latest research on aplastic anemia?

    The Bone Marrow Failure Program at Dana-Farber/Boston Children's
    In addition to providing information and access to local and national research initiatives, our clinic offers multidisciplinary care (physician specialists, dentists, nurse practitioners, social workers) and consultative services for patients with inherited (genetic) and acquired bone marrow failure syndromes, including aplastic anemia.

    Our researchers are actively collecting samples of blood and bone marrow (voluntary) from patients with aplastic anemia and other bone marrow failure syndromes in order to better understand genetic and molecular aspects of the diseases, and clinical outcomes of patients.

    Aplastic Anemia & MDS International Foundation, Inc.
    This foundation, formed in 1983 by concerned parents and medical professionals, maintains a patient registry of children, adolescents, and adults with aplastic anemia. Enrollment is voluntary and serves to help researchers compile data about the disease. For more information, including information about clinical trials, visit www.aplastic.org.

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

    What is the long-term outlook for children with aplastic anemia?

    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.

    Aplastic anemia was once fatal in many cases. Today, innovative therapies like stem cell transplant can cure aplastic anemia, although stem cell transplant carries risks, including graft-versus-host disease.

  • Treatment Questions and New Patient Appointments

    Call 1-855-320-2091. We can answer your questions about treatment, second opinions, and new patient scheduling. In urgent cases, we typically can see new patients within 24 hours.
  • Anemia Clinical Trials

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  • Stem Cell Transplant

    Dana-Farber/Boston Children’s has one of the largest and most experienced pediatric stem cell transplant programs in the U.S. Stem cell transplants can provide effective treatment for cancers, blood disorders, and other conditions.