Journal article
Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria
P Hillmen, P Muus, A Röth, MO Elebute, AM Risitano, H Schrezenmeier, J Szer, P Browne, JP Maciejewski, J Schubert, A Urbano-Ispizua, C de Castro, G Socié, RA Brodsky
British Journal of Haematology | Published : 2013
DOI: 10.1111/bjh.12347
Abstract
Summary: 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%. All patients showed a reduction in lactate dehydrogenase levels, which was sustained over the course of ..
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Funding Acknowledgements
P.H. served on an advisory committee for and received honoraria from Alexion Pharmaceuticals. P.M. served on an advisory committee for Alexion Pharmaceuticals. A.R. served on an advisory committee for and received honoraria and research funding from Alexion Pharmaceuticals. M.O.E. served on an advisory committee for and received honoraria from Alexion Pharmaceuticals. A.M.R. served on an advisory committee for and received honoraria from Alexion Pharmaceuticals. H.S. served on an advisory committee for and received honoraria and research funding from Alexion Pharmaceuticals and received honoraria from Genzyme. J.S. served as a consultant for and received honoraria from Alexion Pharmaceuticals, Ortho Biotech and Roche. P.B. has nothing to disclose. J.P.M. served as a consultant for Alexion Pharmaceuticals and received research funding from Celgene and Eisai. J.S. served on an advisory committee for and received honoraria from Alexion Pharmaceuticals and served on advisory committees for Celgene, Novartis and Pfizer. A.U.-I. served on an advisory committee for Alexion Pharmaceuticals. C.D. served on a speakers bureau for Alexion Pharmaceuticals. G.S. served as a consultant for Alexion Pharmaceuticals and served on advisory committees for and received honoraria from Therakos and Fressenius. R.A.B. served on an advisory committee for Alexion Pharmaceuticals.