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
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 ..
View full abstract