Stem Cell Transplantation
Aplastic Anemia
On this page:
- What is Aplastic Anemia?
- What causes Aplastic Anemia?
- What are the symptoms of Aplastic Anemia?
- How is Aplastic Anemia diagnosed?
- What is the treatment for Aplastic Anemia?
- What is the latest research on Aplastic Anemia?
What is Aplastic Anemia?
Aplastic anemia occurs when the bone marrow produces too few of all types of blood cells: red cells, white cells, and platelets. A reduced number of red blood cells causes the hemoglobin (a type of protein in the red blood cells that carries oxygen to the tissues of the body) to drop. A reduced number of white blood cells cause the patient to be susceptible to infection. A reduced number of platelets can cause the blood not to clot as easily as normal.
What causes Aplastic Anemia?
Aplastic anemia in children has multiple causes, but many causes are "idiopathic," meaning they occur sporadically 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:
- Fanconi anemia
- Dyskeratosis congenita
- Shwachman-Diamond syndrome
- Reticular dysgenesis
- Familial aplastic anemias
What are the symptoms of Aplastic Anemia?
The following are the most common symptoms of aplastic anemia. However, each child may experience symptoms differently. Symptoms may include:
- lack of energy or tiring easily (fatigue)
- pale skin, lips, and hands, or paleness under the eyelids
- fevers/infections
- shortness of breath
- bleeding (bleeding gums, nosebleeds, blood in the stool)
How is Aplastic Anemia diagnosed?
In addition to a complete medical history and physical examination, diagnostic procedures for aplastic anemia may include:
- blood tests
- bone marrow aspiration and biopsy - marrow may be removed by aspiration or 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.
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 the bone marrow failure
- your opinion or preference
Treatment options include, but are not limited to the following:
- 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 Bone Marrow 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 Children's Hospital Boston
In addition to providing information and access to local and national research initiatives, this outpatient 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. This clinic is part of the outpatient Hematology service and functions in conjunction with Dana-Farber Cancer Institute and the Bone Marrow Transplant Service.
Children's Hospital and Dana-Farber Cancer Institute 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 material, 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, please go to www.aplastic.org.
