Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival.

Abstract	Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic disorder with increased mortality and morbidity resulting from intravascular hemolysis. Eculizumab, a monoclonal antibody against the complement protein 5, stops the intravascular hemolysis in PNH. We evaluated 79 consecutive patients treated with eculizumab in Leeds between May 2002 and July 2010. The survival of patients treated with eculizumab was not different from age- and sex-matched normal controls (P = .46) but was significantly better than 30 similar patients managed before eculizumab (P = .030). Three patients on eculizumab, all over 50 years old, died of causes unrelated to PNH. Twenty-one patients (27%) had a thrombosis before starting eculizumab (5.6 events per 100 patient-years) compared with 2 thromboses on eculizumab (0.8 events per 100 patient-years; P < .001). Twenty-one patients with no previous thrombosis discontinued warfarin on eculizumab with no thrombotic sequelae. Forty of 61 (66%) patients on eculizumab for more than 12 months achieved transfusion independence. The 12-month mean transfusion requirement reduced from 19.3 units before eculizumab to 5.0 units in the most recent 12 months on eculizumab (P < .001). Eculizumab dramatically alters the natural course of PNH, reducing symptoms and disease complications as well as improving survival to a similar level to that of the general population.
Authors	Richard J Kelly, Anita Hill, Louise M Arnold, Gemma L Brooksbank, Stephen J Richards, Matthew Cullen, Lindsay D Mitchell, Dena R Cohen, Walter M Gregory, Peter Hillmen
Journal	Blood (Blood) Vol. 117 Issue 25 Pg. 6786-92 (Jun 23 2011) ISSN: 1528-0020 [Electronic] United States
PMID	21460245 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
Chemical References	Antibodies, Monoclonal Antibodies, Monoclonal, Humanized eculizumab
Topics	Adolescent Adult Aged Aged, 80 and over Antibodies, Monoclonal (adverse effects, therapeutic use) Antibodies, Monoclonal, Humanized Blood Transfusion Female Hemoglobinuria, Paroxysmal (complications, drug therapy) Humans Male Middle Aged Survival Analysis Young Adult

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