Emicizumab Prophylaxis in Hemophilia A with Inhibitors

• Published in: The New England journal of medicine Vol. 377; no. 9; pp. 809 - 818
• Main Authors: Oldenburg, Johannes, Mahlangu, Johnny N, Kim, Benjamin, Schmitt, Christophe, Callaghan, Michael U, Young, Guy, Santagostino, Elena, Kruse-Jarres, Rebecca, Negrier, Claude, Kessler, Craig, Valente, Nancy, Asikanius, Elina, Levy, Gallia G, Windyga, Jerzy, Shima, Midori
• Format: Journal Article
• Language: English
• Published: United States Massachusetts Medical Society 31.08.2017
• Subjects: Adolescent; Adult; Aged; Antibodies; Antibodies, Bispecific - adverse effects; Antibodies, Bispecific - pharmacokinetics; Antibodies, Bispecific - therapeutic use; Antibodies, Monoclonal, Humanized - adverse effects; Antibodies, Monoclonal, Humanized - pharmacokinetics; Antibodies, Monoclonal, Humanized - therapeutic use; Bleeding; Blood Coagulation Factors - adverse effects; Blood Coagulation Factors - therapeutic use; Child; Coagulation factors; Drug dosages; Drug Therapy, Combination; Factor VIIa - therapeutic use; Factor VIII - immunology; Factor VIII - therapeutic use; Factor VIII deficiency; Hematology; Hemophilia; Hemophilia A - drug therapy; Hemophilia A - immunology; Hemorrhage; Hemorrhage - prevention & control; Humans; Injections, Subcutaneous; Isoantibodies - blood; Male; Medicine; Middle Aged; Monoclonal antibodies; Prophylaxis; Prothrombin; Recombinant Proteins - therapeutic use; Recovery of function; Thrombosis; Thrombosis - chemically induced; Thrombotic microangiopathy; Young Adult
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMoa1703068

Inhibitors develop in many patients with hemophilia who receive recombinant factor VIII. Prophylaxis with emicizumab, an antibody that functionally replaces factor VIII in the clotting pathway, reduced the rate of bleeding events among patients with hemophilia with inhibitors.