How is aplastic anemia treated?

Although there are both mild and severe cases, aplastic anemia is a serious illness and treatment usually depends on the underlying cause. For certain causes, recovery can be expected after treatment, however, relapses can occur. To treat the low blood counts in aplastic anemia patients, initial treatment is usually supportive, meaning that it is necessary to treat the symptoms first before treating disease itself. Supportive therapy may include:

• blood transfusion (both red blood cells and platelets)
• preventative antibiotic therapymeticulous hand washing
• special care to food preparation (such as only eating cooked foods)
• avoiding construction sites which may be a source of certain fungi
• medications (to stimulate the bone marrow to produce cells)
• immunosuppressive therapy
• hormone therapy

Current treatments for aplastic anemia include immunosuppressive drugs such as antithymocyte globulin (ATG), cyclosporine, and prednisone. More than 65% of patients treated with immunosuppressive drugs experience improvement in their blood counts. This usually occurs two to three months after treatment. Relapse may occur in up to 30% of patients, but many will respond to a second course of treatment.

High doses of the chemotherapy drug cyclophosphamide can lead to long-lasting, complete remission in some patients. This type of therapy rids the body of the cells that cause aplastic anemia without destroying key blood and bone marrow-forming stem cells. Stem cells contain high levels of an enzyme called aldehyde dehydrogenase, which makes them resistant to the toxic effects of cyclophosphamide.

Bone marrow transplantation - where the patient's own non-functioning bone marrow is destroyed with drugs and/or radiation and replaced with donor bone marrow from a compatible donor, usually a sibling or other family member - is also a treatment option for some. When successful, bone marrow transplant is often a cure for aplastic anemia with few incidences of relapse. However, it is a very intensive therapy, and serious immune complications and infections are not unusual. Other non-life-threatening effects of bone marrow transplantation may include sterility and temporary hair loss. Patients under age 40 are the best candidates. Cure rates decline significantly in those over 40.