title: Learnings from over 25 years of PNH experience: the era of targeted complement inhibition.
abstract: 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®) 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.
authors: Bahnhofstrasse 17, 6403 Küssnacht am Rigi, Switzerland. Electronic address: ellen.heitlinger@hando.ch.
authors: Ellen
authors: E
authors: Heitlinger
mesh_terms: D000818:Animals; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D003167:Complement Activation / Q000187:drug effects*; D003169:Complement Inactivator Proteins / Q000627:therapeutic use*; D003165:Complement System Proteins / Q000276:immunology; D006457:Hemoglobinuria, Paroxysmal / Q000150:complications* / Q000188:drug therapy* / Q000276:immunology; D006461:Hemolysis; D006801:Humans; D058990:Molecular Targeted Therapy; D013927:Thrombosis / Q000209:etiology*
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D003169:Complement Inactivator Proteins; D003165:Complement System Proteins; C481642:eculizumab
keywords: Chronic uncontrolled complement activation; Eculizumab; Nitric oxide scavenging; Paroxysmal nocturnal haemoglobinuria (PNH); Premature mortality; Thrombosis