Peter Hillmen, Petra Muus, Alexander Röth, Modupe O Elebute, Antonio M Risitano, Hubert Schrezenmeier, Jeffrey Szer, Paul Browne, Jaroslaw P Maciejewski, Jörg Schubert, Alvaro Urbano-Ispizua, Carlos de Castro, Gérard Socié, Robert A Brodsky
Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by chronic, uncontrolled complement activation resulting in elevated intravascular haemolysis and morbidities, including fatigue, dyspnoea, abdominal pain, pulmonary hypertension, thrombotic events (TEs) and chronic kidney disease (CKD). The long-term safety and efficacy of eculizumab, a humanized monoclonal antibody that inhibits terminal complement activation, was investigated in 195 patients over 66 months. Four patient deaths were reported, all unrelated to treatment, resulting in a 3-year survival estimate of 97·6%...
July 2013: British Journal of Haematology