Journal article

Long Term Safety and Efficacy of Sustained Eculizumab Treatment In Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Robert A Brodsky, Carlos de Castro, Hubert Schrezenmeier, Antonio M Risitano, Joerg Schubert, Jaroslaw P Maciejewski, Ulrich Duehrsen, Lucio Luzzatto, Petra Muus, Jeffrey Szer, Gérard Socié, Peter Hillmen

Blood | American Society of Hematology | Published : 2010

Abstract

Abstract Abstract 4237 PNH is a chronic, life-threatening, acquired disease associated with deficiency of GPI-anchored complement inhibitory proteins on blood cells. The resulting defective regulation of terminal complement activation is responsible for hemolysis and can lead to thromboembolism (TE), chronic kidney disease (CKD) and pulmonary hypertension. The risk of TE is high, with an observed 6.24 venous TE events per 100 patient years, or approximately 62-fold higher compared to the general population: in fact, TE accounts for 40–67% of PNH related deaths. The effectiveness of anticoagulation (AC) in PNH patients (pts) is uncertain, as AC treated PNH may st..

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