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Rare Diseases: Aplastic Anemia
Alternate Names: None
Definition
Aplastic anemia is a rare, non-contagious and often life-threatening disorder that results from the unexplained failure of the bone marrow to produce red blood cells, white blood cells and platelets. A decrease in the production of blood cells means that patients are more susceptible to bleeding, fatigue and infections.
Incidence
Aplastic anemia affects two to six people per million worldwide. The disease is more prevalent in Asia than in the Western world. Most cases occur in older adults, but a significant number of children are affected.
Influencing Factors
The cause of the disease in an individual patient is usually unknown, although the disease has been linked to environmental toxins such as benzene derivatives, some drugs, pesticides and chemicals.
Aplastic anemia can be caused by a virus in some individuals.
There is evidence that parts of the immune system play a role in the disease process.
Treatment Strategies
Patients with severe aplastic anemia require immediate hospital treatment.
Initial treatment often includes blood and platelet transfusions and intravenous antibiotics.
Bone marrow transplantation is recommended for patients who are good candidates and who have matched donors. Most young patients who have matched donors are successfully treated by bone marrow transplantation.
Drug therapy, which suppresses the immune system, is another treatment choice that is very successful.
Current Research
St. Jude is preparing for a study for patients without a matched donor and in whom drug therapy was only partially successful. Investigators are planning to use a therapy that modifies the immune system in a different way than drug therapy. This therapy is expected to interrupt the disease process and allow the bone marrow to recover and produce blood cells again.
St. Jude is also developing a new bone marrow transplant regimen for patients who do not have a matched sibling donor and who do not respond to the drug therapy.
Source: http://www.stjude.org/
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