KARACHI (PPI) - Patients suffering from a blood disorder that prevents proper clotting have the option of a new medication that may dramatically improve their health. Chronic immune thrombocytopenic purpura ITP is autoimmune disease that dramatically reduces number of platelets in their blood causing bruises, nosebleeds and, rarely, life threatening brain hemorrhages. Promacta (eltrombopag) was granted accelerated approval by U.S. Food & Drug Administration in November 2008 for treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Promising results of international, multi-centre Phase III clinical trial, led by New York Presbyterian/ Weill Cornell researchers, were basis of this approval and published in recent issue of The Lancet. "Findings from new study are very encouraging. I believe this treatment is effective option for all patients suffering from chronic ITP," says Dr James Bussel, principal investigator of study; director of Program for Platelet Disorders at New York Presbyterian Hospital/ Weill Cornell Medical Center. It follows previous Phase II study published 2007 in New England Journal of Medicine. This trial showed eltrombopag was effective in raising platelet counts and lowering bleeding in adult subjects with chronic ITP. Phase II study's results also determined most promising dose of 50mg, which was given to all of experimental subjects in Phase III study. Phase III study tested 114 subjects, who were all 18 years and older, with at least six months of history with ITP and with low-platelet counts of 30,000 per microliter (L) of blood (normal range is 150,000 to 400,000). The subjects were split into two groups: two-thirds were in experimental group that received standard of care with addition of 50mg of eltrombopag, and a control group received standard of care and a placebo pill. By end of 43-day testing period, 59% of subjects receiving Promacta achieved platelet counts at or over 50,000 per L of blood, compared with 16 percent of subjects in placebo group. A safe-level platelet count is between 30,000 and 50,000 per L of blood. Promacta subjects were almost 10 times more likely to reach target platelet counts as placebo group. Currently, patients are treated with corticosteroids, such as prednisone, which may have side effects like fatigue, mood swings, weight gain. Other common treatments include IV anti-D, IV gammaglobulin, and also rituximab. More drastic measures, likesurgical removal of spleen (splenectomy) are sometimes taken to prevent body from destroying platelet cells within organ. However, this may put a patient at risk for blood stream infections because of spleen's role as a filtering organ of immune system. Promacta works by stimulating production of cells in bone marrow that form platelet cells in blood. Past studies showed drug boosts platelet counts in both ITP and normal subjects. Ongoing and future studies will evaluate safety, efficacy of eltrombopag as a long-term treatment for ITP, and its efficacy and safety in populations with hepatitis-C related thrombocytopenia or patients receiving myelosuppressive chemotherapy.