Paroxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatment
Ilene Ceil Weitz
Jane Anne Nohl Division of Hematology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
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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