Journal
BLOOD REVIEWS
Volume 27, Issue -, Pages S1-S6Publisher
CHURCHILL LIVINGSTONE
DOI: 10.1016/S0268-960X(13)00080-5
Keywords
Paroxysmal nocturnal haemoglobinuria (PNH); Chronic uncontrolled complement activation; Eculizumab; Thrombosis; Premature mortality; Nitric oxide scavenging
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Paroxysmal nocturnal haemoglobinuria (PNH) is a progressive and life-threatening disease that causes thrombosis, end organ damage and impaired quality of life. Chronic uncontrolled complement activation leads to chronic haemolysis, causing progressive morbidities and early mortality. Hence, early diagnosis is essential for improved patient management and prognosis. Eculizumab (SOLIRIS (R)) specifically inhibits chronic, uncontrolled complement activation and is the first-in-class, humanised, monoclonal antibody targeting C5 within the terminal complement pathway. Eculizumab is the first and only approved treatment for PNH in adults and children. (C) 2013 Elsevier Ltd. All rights reserved.
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