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For the full abstracts of these presentations, please visit the European Hematology Association Web site at http://congress.ehaweb.org/12th.

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. Hemolysis can cause one or more of the following symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life and blood clots (thromboses).(4)(5)

PNH 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) Ten percent of all patients first develop symptoms at 21 years of age or younger.(5) 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.(8) The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.(6)(8)

PNH has been identified more commonly among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndrome (MDS).(9)(10)(11)(12) In patients with thrombosis of unknown origin, PNH may be an underlying cause.(5)(13)

There are currently no therapies available in Europe specifically for the treatment of PNH. PNH treatment has been limited to symptom management through periodic blood transfusions, non-specific immunosuppressive therapy and, infrequently, bone marrow transplantations - a procedure that carries considerable mortality risk.(5)(13)

About Soliris
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