Journal article

Clinical Characteristics of Classic Paroxysmal Nocturnal Hemoglobinuria (PNH) in Pediatric Patients: A Comparison with Classic PNH in Adults. An International PNH Registry Study

Alvaro Urbano-Ispizua, Hubert Schrezenmeier, Petra Muus, Jaroslaw P Maciejewski, Gérard Socié, Jeffrey Szer, Robert A Brodsky, Monica Bessler, Wendell F Rosse, Yuzuru Kanakura, Gus Khursigara, Camille L Bedrosian, Peter Hillmen

Blood | American Society of Hematology | Published : 2011

DOI: 10.1182/blood.v118.21.2102.2102

Abstract

Abstract Abstract 2102 Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, life-threatening hematopoietic stem cell disorder characterized by deficiency of the GPI-anchored complement inhibitory proteins CD55 and CD59. Uncontrolled complement activation is responsible for chronic hemolysis that leads to the serious clinical morbidities including thromboembolism (TE) and chronic kidney disease, which increase risk of mortality. PNH is infrequent in children and as such, there is a paucity of data in the literature examining their burden of disease. This is the largest series of pediatric patients with PNH, and ..

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