Eltrombopag, a drug that was designed to stimulate production of platelets from the bone marrow and thereby improve blood clotting, can raise blood cell levels in some people with severe aplastic anemia who have failed all standard therapies.
About one-third of aplastic anemia cases do not respond to standard therapy, a combination of immune-suppressing drugs. Although bone marrow stem cell transplantation is an option for some, patients without a matched donor have few treatment options. The findings of this new clinical study, carried out by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health, suggest eltrombopag could be a second-line therapeutic option for them.
"Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia," will be published online July 5 in the New England Journal of Medicine.
Aplastic anemia is a rare blood disorder, with about 600 new cases in the U.S. each year. Aplastic anemia results from the destruction of bone marrow stem cells, which mature into red blood cells that carry oxygen, white blood cells that fight infection, and platelets that prevent excess bleeding. Symptoms of the disorder include fatigue, frequent infections, and hemorrhaging. In severe cases unresponsive to treatment, death can occur.