An early phase clinical trial has shown promise in treating some cases of severe aplastic anemia that hasn’t responded to other 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. Read the news report here.
Study mentioned: Olnes MJ. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. PMID: 22762314
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