Platelet drug shows clinical benefits for severe, unresponsive aplastic anemia

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," was published on-line July 5 in the New England Journal of Medicine. The drug is marketed by UK pharma giant GlaxoSmithKline (LSE: GSK), which was not involved in the research, under the brand names Promacta and Revolade.