Platelet Drug Helps Some Patients with Aplastic Anemia

An early phase clinical trial has shown promise in treating some cases of severe aplastic anemia that hasn’t responded to other treatments. Aplastic anemia is a disorder in which the bone marrow fails to make enough blood cells. Symptoms include fatigue, frequent infections, and abnormal bruising and bleeding. It is usually treated with stem cell transplants or drugs that suppress the immune system. However, about a third of patients with aplastic anemia do not get better with these treatments.

In the trial, 25 patients with aplastic anemia that hadn’t responded to treatment received a drug called Promacta (eltrombopag) that was designed to stimulate production of platelets from bone marrow. Researchers hoped it would also improve the number of blood stem cells and blood counts overall. After 12 weeks, 11 of the patients showed improved production of at least 1 kind of blood cell. Blood counts rose enough for 9 patients to stop having platelet transfusions. The drug is given orally and patients had few side effects. The researchers say that even though the drug is currently available for other diseases, it should be limited to aplastic anemia patients in clinical trials until more research is done.

There are about 300 to 600 new cases of aplastic anemia in the U.S. each year. It is not a type of cancer, but may be associated with certain cancers or cancer treatments. A small number of patients with aplastic anemia may develop leukemia. Aplastic anemia can be inherited or acquired. Acquired aplastic anemia is much more common than the inherited type. Most cases of acquired aplastic anemia are caused by the immune system attacking the bone marrow. Patients with this type of aplastic anemia were included in the study of Promacta.

The study, conducted by researchers at the National Institutes of Health, was published in the July 5, 2012 issue of The New England Journal of Medicine.

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Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia. Published in the July 5, 2012 issue of The New England Journal of Medicine (Vol. 367, No. 1). First author: Mathew J. Olnes, MD, PhD, National Institutes of Health, Bethesda, Md.

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