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Paroxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatment
Authors Ilene Weitz I
Received 18 May 2015
Accepted for publication 21 July 2015
Published 1 September 2015 Volume 2015:5 Pages 75—81
DOI https://doi.org/10.2147/ODRR.S73641
Checked for plagiarism Yes
Review by Single-blind
Peer reviewer comments 4
Editor who approved publication: Dr Lise Aagaard
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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