Eltrombopag shows efficacy in severe, refractory aplastic anemia

by Bruce Sylvester – Emerging uses of FDA Approved Drugs –

In some patients severe aplastic anemia who have failed standard therapies, eltrombopag appears to raise blood cell levels, researchers reported  online on July 5, 2012 in the New England Journal of Medicine.

As background, the authors noted that even though  bone marrow stem cell transplantation is an option for some patients with aplastic anemia, patients without a matched donor have few treatment options. The new findings suggest eltrombopag might become a therapeutic option for these patients.

In the phase 2 study, the investigators found that 11 of 25 patients showed improvement in production of at least 1 type of blood cell — red blood cell, white blood cell, or platelet — after 12 weeks of oral eltrombopag therapy.

Among 7 subjects who volunteered to continue on eltombopag long-term (8-32 months), 6 showed an improvement in all 3 types of blood cells, and maintained safe blood counts without red blood cell or platelet transfusions.

Overall the drug was well tolerated, with few side effects.