Aplastic Anemia

What is aplastic anemia?

Aplastic anemia (AA) is a disorder that occurs when the bone marrow cannot make enough new blood cells for the body, including red blood cells (which carry oxygen throughout the body), white blood cells (which protect against infections), and platelets (which help the blood clot and stop bleeding).

What are the symptoms of aplastic anemia?

Common signs and symptoms of aplastic anemia include fevers/infections due to a low number of white blood cells, lack of energy (fatigue) due to anemia, and mucosal bleeding (bleeding in areas such as the mouth and nose) due to a low number of platelets in the blood. The reason for the low number of all of the blood cells is a decrease in the number of precursor blood cells in the bone marrow, which is usually a result of the immune system attacking the bone marrow, or the lack of a supportive environment in the bone marrow necessary to make new blood cells.

How do we treat aplastic anemia at Stanford Children’s Health?

Current treatment options for milder cases of aplastic anemia include immunosuppressive therapy (IST) to suppress the immune system, and/or red blood cell and platelet transfusions. Immunosuppressive therapy includes drugs like anti-thymocyte globulin (ATG) and cyclosporine to help restore the bone marrow’s ability to function normally.

Severe aplastic anemia (SAA) is defined as lower-than-average amounts of two or more types of blood cells. Stem cell transplantation (SCT) is the standard treatment for SAA. The goal of stem cell transplantation is to replace the patient’s damaged bone marrow with normal stem cells from a healthy donor. Stem cell transplantation can cure more than 95 percent of patients with SAA if a matched related donor is available. Stem cell transplants for SAA from unrelated donors have similar outcomes in children. It is better to perform the stem cell transplant as soon as possible when a matched donor is available.

Before a stem cell transplant, patients receive therapy to eliminate their immune system to permit the new donor stem cells to grow. Patients usually receive anti-thymocyte globulin (ATG) and cyclophosphamide to suppress their immune system. At Stanford, we use a tailored dose of ATG to maximize its benefit (decreasing the risks of graft rejection and graft-versus-host disease) while minimizing toxicity.

Patients are usually discharged from the hospital 40 to 50 days after transplantation when their blood and immune systems are functioning at healthy levels.