Glaxo drug improves platelet count in study

Data from this study demonstrated that treatment with eltrombopag resulted in a significant increase in platelet count compared to placebo across a variety of typically poorly responding idiopathic thrombocytopenic purpura (ITP) patients including those with prior splenectomy or those having a very low baseline platelet count.

At doses of 50mg and 75mg, 70% and 81% of patients respectively, achieved the primary clinical endpoint of having a platelet count greater than 50,000/ml after up to 6 weeks of dosing compared to 11% of patients on placebo.

Based on these and previous clinical trial results, the company says that eltrombopag has the potential to be the first oral platelet growth factor for patients with thrombocytopenia, a disorder characterized by low platelet counts leaving patients at risk of episodes of spontaneous bruising, mucosal bleeding, and in severe cases intracranial hemorrhage.

“Results demonstrating the beneficial effect of an oral therapy such as eltrombopag, which may potentially work by stimulating the production of platelets, is very exciting and a major scientific advance,” says Adrian Newland, professor of hematology at Queen Mary, University of Londonand a principal investigator for this trial. “This is highly encouraging news for patients who are in real need of new therapies to combat ITP because current treatment options such as steroids or removal of the spleen all have significant limitations, particularly in terms of side effects.”

Eltrombopag is an investigational non-peptide small molecule that is administered orally and interacts with the thrombopoietin receptor. This receptor is located on the surface of specific cells in the bone marrow and its stimulation leads to the production of platelets.