A New Investigational Drug for Treatment of Thrombocytopenia

A novel approach to treatment of thrombocytopenia involves stimulation of platelet production with agonists of thrombopoeitin receptors in the megakaryocyte lineage. One such drug, eltrombopag, is under investigation.

In an industry-sponsored randomized trial, 117 patients with idiopathic thrombocytopenic purpura (ITP) — all of whom had platelet counts below 30,000 and poor response to previous ITP treatment — received one of three oral doses of eltrombopag or placebo. During 6 weeks of treatment, 81% of patients receiving the highest dose of the drug achieved platelet counts of 50,000 or more, compared with 11% of placebo recipients. Within 2 weeks after eltrombopag treatment ended, platelet counts returned to baseline. Bleeding decreased d…