Hemophilia therapy-navigating speed bumps on the innovation highway

New England Journal of Medicine

Volumn 374, Issue 21, 2016, Pages 2087-2089

  DiMichele, Donna M ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALLOANTIBODY; BISPECIFIC ANTIBODY; BLOOD CLOTTING FACTOR 8; MONOCLONAL ANTIBODY; NEUTRALIZING ANTIBODY; VON WILLEBRAND FACTOR;

ANTAGONISTS AND INHIBITORS; BLOOD; HEMOPHILIA A; HEMORRHAGE; HUMAN; IMMUNOLOGY; MALE;

ANTIBODIES, BISPECIFIC; ANTIBODIES, MONOCLONAL, HUMANIZED; ANTIBODIES, NEUTRALIZING; FACTOR VIII; HEMOPHILIA A; HEMORRHAGE; HUMANS; ISOANTIBODIES; MALE; VON WILLEBRAND FACTOR;

EID: 84971324494     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMe1603419     Document Type: Editorial

References (10)

1. Ragni MV. New and emerging agents for the treatment of hemophilia: focus on extended half-life recombinant clotting proteins. Drugs 2015; 75: 1587-600.
2. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
3. Gouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood 2013; 121: 4046-55.
4. Hay CRM, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335-44.
5. Dargaud Y, Pavlova A, Lacroix-Desmazes S, et al. Achievements, challenges and unmet needs for haemophilia patients with inhibitors: report from a symposium in Paris, France on 20 November 2014. Haemophilia 2016; 22: Suppl 1: 1-24.
6. Lai JD, Georgescu MT, Hough C, Lillicrap D. To clear or to fear: an innate perspective on factor VIII immunity. Cell Immunol 2016; 301: 82-9.
7. Oldenburg J, Lacroix-Desmazes S, Lillicrap D. Alloantibodies to therapeutic factor VIII in hemophilia A: the role of von Willebrand factor in regulating factor VIII immunogenicity. Haematologica 2015; 100: 149-56.
8. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med 2016; 374: 2054-64.
9. Shima M, Hanabusa H, Taki M, et al. Factor VIII-mimetic function of humanized bispecific antibody in hemophilia A. N Engl J Med 2016; 374: 2044-53.
10. Uchida N, Sambe T, Yoneyama K, et al. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood 2016; 127: 1633-41.