General considerations in treating aplastic anemia
Many years ago, aplastic anemia was considered a fatal disease unless the patient recovered without treatment (spontaneously). Now that we understand this is often an immune disease, treatments have been devised that have a high success rate.
Those with acquired aplastic anemia are treated with supportive care until their disease becomes severe (with very low blood counts). This is followed by immunosuppressant therapy (also called immunosuppressive therapy) or stem cell transplant. Stem cell transplant is preferred when the patient has a matched, related donor, and is young and otherwise healthy. Immune treatments may be the first option for older patients and those without a matched donor.
A small number of people with aplastic anemia will develop leukemia or other cancers after several years. But it isn’t clear if this is caused by the treatment or is just part of the disease itself. For example, many people with Fanconi anemia who don’t get aplastic anemia will still go on to develop leukemia.
Aplastic anemia is rare, and treating it is very specialized, so it may best be done at a major medical center where they have experience with this disease. This is particularly true for stem cell transplants.
Sometimes the first treatment isn’t successful and will need to be repeated. Often the second treatment will be effective, even if the first wasn’t. Also, the treatment may be only partially effective, and the blood counts may not return to normal. In this situation, many people can still lead a normal life and not need treatment. Their blood counts, although not normal, will be high enough for a normal life.
Sometimes, none of the treatments is successful. In this case a person with aplastic anemia might want to consider taking part in a clinical trial, that is, a study of a new therapy that hasn’t yet been proven successful.
Last Medical Review: 04/23/2013
Last Revised: 04/23/2013