Paroxysmal nocturnal haemoglobinuria: to prednisone or not to prednisone? - a case report of a patient previously treated with steroids for 15yrs and significant response on eculizumab

摘要

BackgroundParoxysmal nocturnal haemoglobinuria (PNH) is a rare acquired haematopoietic stem cell disorder characterised by persistent haemolysis and platelet activation, severe end-organ damage, an increased risk of thrombosis and early mortality. We present the case of a 56-year-old male with long-standing PNH and significant disease-related morbidity who underwent steroid therapy for approximately 15yrs before treatment with eculizumab, a humanized monoclonal antibody that blocks the terminal phase of the complement cascade at the C5 level.