Increasing Platelet Count in ITP

A new drug under investigation significantly increases blood platelet counts in patients with chronic immune thrombocytopenic purpura (ITP).

Data from a Phase II study of eltrombopag, an investigational small-molecule thrombopoietin receptor being developed by GlaxoSmithKline (London, UK), demonstrated that the compound was effective in increasing platelet counts in ITP. The disorder is marked by platelet destruction, episodes of frequent spontaneous bruising, mucosal bleeding, and in severe cases, intracranial hemorrhage and acute episodes of severe bleeding.

Eltrombopag is thought to stimulate the proliferation and differentiation of megakaryocytes, the bone marrow cells that give rise to blood platelets, and thus is considered a platelet growth factor. Because it is a small molecule, the drug has less potential than large protein molecules for causing an immune system reaction.

The data were presented by Dr. James B. Bussel, professor of pediatrics and director of the platelet disorders center, children's blood foundation division, at New York-Presbyterian Hospital/Weill Cornell Medical Center (New York, NY, USA), at the annual meeting of the American Society of Hematology (ASH) in Atlanta (GA, USA) in December 2005.

"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, principal investigator of the study.






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