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.
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.
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:
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:
In addition to a complete medical history and physical examination, diagnostic procedures for aplastic anemia may include:
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.
Specific treatment for aplastic anemia will be determined by your child’s physician based on:
Treatment options include, but are not limited to the following:
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.
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.