Eculizumab is the first safe and effective targeted therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH), according to research presented at the 11th Congress of the European Hematology Association in Amsterdam.
Paroxysmal nocturnal hemoglobinuria is a rare form of hemolytic anemia that is characterized by destruction of red blood cells by the body's complement system. It is characterised by a triad of clinical features: hemoglobulinuria, with intravascular hemolysis that causes disabling symptoms of abdominal pain, dysphagia, erectile failure and severe lethargy; Budd-Chiari syndrome, the most feared PNH complication that can cause fatal thrombosis; and aplastic anemia, which results from bone marrow failure.