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Paroxysmal nocturnal hemoglobinuria: new concepts in pathophysiology and treatment

Authors Weitz IC

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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