Aplastic Anemia

What is aplastic anemia?

Aplastic anemia occurs when the bone marrow produces too few platelets, leading to a decrease in hemoglobin and symptoms of fatigue. A reduced number of a major type of white blood cell important for defense against bacterial and fungal infections, called neutrophils, increases susceptibility to infection. Too few platelets leads to increased risk of bleeding or bruising.

The role of bone marrow in aplastic anemia

Bone marrow produces all blood cells: red blood cells, white blood cells, and platelets.

  • Red blood cells contain a protein called hemoglobin that carries oxygen from the lungs to tissues.
  • White blood cells fight infection.
  • Platelets are important for blood to clot.

What are the symptoms of aplastic anemia?

Each child may experience symptoms differently, but the most common symptoms of aplastic anemia are:

  • lack of energy or tiring easily
  • pale skin, lips, and hands, or paleness under the eyelids
  • shortness of breath
  • fevers or infections bleeding, such as bruising, bleeding gums, nosebleeds, or blood in the stool
  • irregular heartbeat
  • dizziness or headache

It is important to understand that some symptoms of anemia may resemble those of other more common medical problems or other blood disorders. Because some of these symptoms can also point to other conditions, and because anemia itself can be a symptom of another medical problem, it’s important to have your child evaluated by a qualified medical professional for an accurate diagnosis and prompt treatment.

What causes aplastic anemia?

Aplastic anemia in children has multiple causes. Usually it is “idiopathic,” occurring with no known reason. It also can be the result of a previous illness, existing problems, or can be caused by an inherited genetic disorder.

About 80 to 85 percent of cases of aplastic anemia in children are acquired and may include a history of:

  • specific infectious diseases, such as hepatitis, Epstein-Barr virus, or cytomegalovirus
  • taking certain medications, including some antibiotics and arthritis drugs
  • exposure to certain toxins, such as benzene, pesticides, and insecticides
  • exposure to radiation or chemotherapy

In 15 to 20 percent of cases, children inherit a disorder that predisposes them to developing aplastic anemia such as:

How we care for aplastic anemia

Children and young adults with aplastic anemia are treated at The Bone Marrow Failure and Myelodysplastic Syndrome (MDS) Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, 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.

Our areas of research

In addition to providing information and access to local and national research initiatives, our clinic offers multidisciplinary care and consultative services for patients with inherited and acquired bone marrow failure syndromes, including aplastic anemia.

Our researchers are actively collecting samples of blood and bone marrow from volunteer 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 our clinical trial finder.