PNH Patients With Lower Levels of Hemolysis, Mild Anemia and Minimal Transfusion Support Have Significant Disease Burden; Soliris(R) Therapy Provided Clinical Improvements in PNH Patients Regardless of Diseas...(more points is considered clinicallymean...),Health

Date:12/11/2007

more points is considered clinically meaningful in this instrument. (5) In patients with mild baseline anemia, fatigue scores improved by 5 points during the first six months and by 6 points during the most recent six months of therapy (P<0.001 for each comparison to baseline). Even in patients with minimal transfusion support prior to Soliris therapy, fatigue scores improved by 9 points during the first six months and by 15.5 points during the most recent six months of treatment (P<0.001 for each comparison to baseline).

In addition, in patients with history of bone marrow failure, units transfused were reduced from mean 9.2 +/- 1.2 units per patient prior to treatment to 4.4 +/- 1.4 units per patient during the most recent six months of Soliris treatment (P<0.001).

About PNH

PNH is an acquired genetic blood disorder defined by hemolysis, in which patients' red blood cells are destroyed by complement, a component of the body's immune system. PNH is a rare disease that affects an estimated 8,000 to 10,000 people in North America and Europe. (6) PNH often strikes people in the prime of their lives, with an average age of onset in the early 30's. (7) Approximately ten percent of all patients first develop symptoms at 21 years of age or younger. (4) PNH develops without warning and can occur in men and women of all races, backgrounds and ages. PNH often goes unrecognized, with delays in diagnosis often ranging from one to more than 10 years. (3) The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis. (3,7)

PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndrome (MDS). (8,9,10,11) In patients with thrombosis of unknown origin, PNH may be an underlying cause. (4,12)

Prior to approval of Soliris, there were no therapies specifically available for the treatment of PNH. PNH treatment was limited to sympt
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SOURCE Alexion Pharmaceuticals, Inc. Copyright©2007 PR Newswire. All rights reserved

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