New treatment for chronic Immune Thrombocytopenic Purpura

Data from a Phase II study of eltrombopag, an oral investigational drug that increases the production of platelets, demonstrate that the compound was effective in increasing platelet counts in patients with chronic Immune Thrombocytopenic Purpura (ITP), a disorder characterized by episodes of frequent spontaneous bruising, mucosal bleeding, and in severe cases intracranial hemorrhage, and acute episodes of severe bleeding.

The data were presented by James B. Bussel, M.D. -- director of the Platelet Disorders Center, Children's Blood Foundation Division at NewYork-Presbyterian Hospital/Weill Cornell Medical Center -- at the "Seventh Review of Immune Thrombocytopenic Purpura: Current Issues in Pathogenesis, Diagnosis and Management." The annual symposium is held in connection with the 47th Annual Meeting of the American Society of Hematology (ASH).

In the current double-blind randomized study, 104 adult ITP patients who had failed one prior therapy and had a platelet count <30,000/µL, were randomly assigned to take oral eltrombopag 30 mg, 50 mg, 75 mg, or placebo once daily for six weeks. The study found that eltrombopag at doses of 50 mg and 75 mg was significantly better than placebo (P < 0.001) at increasing and maintaining platelet counts to a level that would reduce the risk of bleeding and bruising. The drug increased platelet levels quickly, with up to 87 percent of responders raising their counts by day 15. Response was defined as a platelet count of 50,000/µL. At the end of six weeks of dosing, there were no safety and tolerability events that would preclude the advancement to future studies.

"The results of this trial are very encouraging, given that there are currently no oral treatment options available for ITP that increase the platelet count in such a high percentage of often difficult patients with apparently so little toxicity," said Dr. Bussel, who is the principal investigator of the study and professor of pediatrics at Weill Medical College of Cornell University. "Eltrombopag may offer physicians an effective and convenient treatment alternative because current therapies, such as steroids and platelet transfusions, can have unwanted side effects, must be given IV, or are not sustainable over the long-term."