Abstract
Paroxysmal 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 15 yrs before treatment with eculizumab, a humanized monoclonal antibody that blocks the terminal phase of the complement cascade at the C5 level. The patient presented with a severely impaired quality of life in 1997 and was diagnosed with PNH 8 months later, soon after which he was commenced on steroid therapy with prednisone. During long-term steroid therapy with progressive increases in prednisone dose, the patient had frequent haemolytic episodes as well as thrombosis and renal complications. He also experienced Cushing's syndrome with arterial hypertension, insulin-dependent diabetes mellitus, osteoporosis, acne and portal fibrosis. Eculizumab therapy was started in late-2009 and led to rapid improvements in haemoglobin and lactate dehydrogenase levels with a complete cessation of haemolytic episodes. Eculizumab has been well tolerated....Continue Reading
References
May 1, 1987·American Journal of Hematology·S IssaragrisilY Tang-naitrisorana
Nov 9, 1995·The New England Journal of Medicine·P HillmenJ V Dacie
Jan 1, 1994·Acta Haematologica·K Bourantas
Aug 31, 1996·Lancet·G SociéE Gluckman
Feb 6, 2004·The New England Journal of Medicine·Peter HillmenRussell P Rother
Jul 30, 2005·Blood·Charles ParkerUNKNOWN International PNH Interest Group
Sep 23, 2006·The New England Journal of Medicine·Peter HillmenLucio Luzzatto
Dec 7, 2007·Blood·Robert A BrodskyPeter Hillmen
Apr 25, 2008·Current Protocols in Immunology·C J Parker
Jul 9, 2008·Blood·Antonio M RisitanoBruno Rotoli
Jan 16, 2009·Lancet·Charles Parker
Apr 18, 2009·Blood·Robert A Brodsky
Jun 1, 2008·Biologics : Targets & Therapy·Antonio M Risitano, Bruno Rotoli
Jul 27, 2010·American Journal of Hematology·Peter HillmenWendell Rosse
Sep 3, 2011·European Journal of Haematology·Alexander Röth, Ulrich Dührsen
Mar 8, 2012·Current Opinion in Hematology·Charles J Parker
Jan 18, 2013·Cytometry. Part B, Clinical Cytometry·Iuri MarinovAndrea Illingworth
Apr 24, 2013·Blood·Anita HillPeter Hillmen
Apr 27, 2013·British Journal of Haematology·Peter HillmenRobert A Brodsky
May 3, 2013·International Journal of Hematology·Jong Wook LeeSang Kyun Sohn