Overview of Pediatric Aplastic Anemia

Aplastic anemia is a disorder where the bone marrow is not able to make the blood cells that it normally is responsible for producing. As a consequence, patients with aplastic anemia have low numbers of red blood cells, neutrophils (a kind of white blood cell), and platelets. Because of this, patients with aplastic anemia often feel fatigued, and are at risk of infections, bleeding and bruising.

There are many potential causes of impaired bone marrow function. In children, aplastic anemia is most often the result of an inappropriate immune system attack on the bone marrow. However, some children with low blood cell counts may have an underlying genetic syndrome that leads to bone marrow failure. Additionally, certain chemicals, medications, and very high doses of radiation can also impair marrow function.

Treatment for aplastic anemia includes temporarily supporting the patients with transfusions and antibiotics to treat infections. However, currently, the only proven therapies to restore the ability of the bone marrow to make the blood cells are either medications to intensely suppress the immune system or a bone marrow transplant (also known as a hematopoietic stem cell transplant).

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Immune-mediated aplastic anemia resembles other bone marrow disorders such as myelodysplastic syndrome, certain infections, and leukemia. Because treatment for aplastic anemia differs from management of other diseases affecting the bone marrow, it is critically important to distinguish between these disorders when a diagnosis of aplastic anemia is suspected. Therefore, children with persistently low blood counts should be evaluated by pediatric hematologists with expertise in diagnostics and treatment of these uncommon disorders.