A Global Registry of Patients with Paroxysmal Nocturnal Hemoglobinuria.
Robert Brodsky, Hubert Schrezenmeier, Petra Muus, Monica Bessler, Jeffrey Szer, Bruno Rotoli, Jaroslaw P Maciejewski, Gerard Socie, A Urbano-Ispizua, Wendell F Rosse, Yuzuru Kanakura, Anders Karnell, Camille Bedrosian, Peter Hillmen
Blood | American Society of Hematology | Published : 2009
Abstract Abstract 3007 Poster Board II-983 Paroxysmal nocturnal hemoglobinuria (PNH) is a rare clonal hematopoietic stem cell disease characterized by complement-mediated hemolysis which can lead to life-threatening complications including thrombosis, kidney disease, and pulmonary hypertension. The natural history of PNH is highly variable and has previously been captured by retrospective assessment. However, the clinical presentation and prognosis of the disease has changed with the increased awareness of PNH, the increased use of more sensitive diagnostic tests, and the availability of new treatment. Specifically, the development of targeted but..View full abstract