2015•Orphan Drugs Research and ReviewsOpen access

Paroxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatment

Ilene Weitz

Abstract

Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder characterized by hemolysis, cytopenias, bone marrow dysfunction, and thrombosis. Over the last 20 years, we have seen a dramatic unraveling of the pathophysiology of this rare and complex disorder. As a result, new therapies involving complement inhibition have been developed, changing our understanding of the disease and improving outcomes for affected patients. Keywords: paroxysmal nocturnal hemoglobinuria, pathophysiology, treatment

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Key concepts: Paroxysmal nocturnal hemoglobinuria, Pathophysiology, Hemoglobinuria, Eculizumab, Medicine, Hematology, Bone marrow failure, Internal medicine