The bone marrow produces all blood cells: red blood cells,
white blood cells and platelets. Red blood cells contain the protein hemoglobin
that carries oxygen from the lungs to tissues. White blood cells fight
infection. Platelets are important for blood to clot.
Aplastic anemia occurs when the bone marrow produces too few
of these cells. Too few red blood cells lead to a decrease in hemoglobin and
symptoms of fatigue. A reduced number of neutrophils (a major type of white
blood cell important for defense against bacterial and fungal infections) increases
susceptibility to infection. Too few platelets leads to increased risk of
bleeding or bruising.
Children and young adults with aplastic anemia are treated
at Dana-Farber/Boston Children's through our Bone Marrow Failure Program, recognized as one of the nation’s best pediatric treatment
and research programs for bone marrow failure and related conditions. Our
patients have access to advanced treatments and diagnosis, including DNA mutation identification and ongoing clinical trials investigating new treatments. 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 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 benzine, or exposure
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
The Bone Marrow Failure Program at Dana-Farber/Boston
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
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.
We are also interested in studying new drugs that may help children
with aplastic anemia. For instance, a new drug called eltrombopag has shown
promise in treating adults with aplastic anemia. We are considering a trial to
investigate its use in the treatment of children.
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.
Contact us: If you’re not
sure which clinical trials might be right for your child, email us at firstname.lastname@example.org.
We can help you navigate your options.
Aplastic anemia was once fatal in many cases. Today,
therapies like stem cell transplant can
cure aplastic anemia in the great majority of children with either a
tissue-matched sibling donor or, in many patients, a matched unrelated donor.
However, stem cell transplant carries risks such as infection and
For children with aplastic anemia who do not have suitable
donors, medical treatment can bring blood counts back to normal in
approximately two thirds of cases. While their blood counts may remain normal
for many years—allowing them to return to all normal activities—laboratory
tests show that the disease may never completely go away. If aplastic anemia
recurs, other treatments are available.