There are estimated to be between 50,000 -- 100,000 individuals in the U.S. diagnosed with chronic Immune Thrombocytopenic Purpura (ITP), an autoimmune disease that dramatically reduces the number of platelets in their blood -- causing bruises, nosebleeds and, sometimes, life-threatening brain hemorrhages.
Now, the results of an international multicenter clinical research study led by NewYork-Presbyterian Hospital/Weill Cornell Medical Center demonstrate that an investigational oral platelet growth factor called eltrombopag successfully increased platelet counts and decreased bleeding in patients with the condition.
Published in today's New England Journal of Medicine and representing the largest ever clinical trial of its kind for ITP patients, the Phase 2 study found that eltrombopag doses of 50 and 75 mg daily elevated platelet counts to a safe level (= 50,000 per cubic millimeter) in more than 75 percent of chronic ITP patients within two weeks -- compared to 11 percent of patients receiving placebo. As long as the therapy was administered, platelet counts continued to rise or remain increased, and bleeding symptoms decreased. Incidence and severity of adverse effects was similar in the placebo and eltrombopag treated groups.