Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
Tóm tắt
A novel recombinant factor VIII with prolonged half-life, rFVIIIFc, was developed to reduce prophylactic injection frequency. rFVIIIFc was well-tolerated in patients with severe hemophilia A, and resulted in low bleeding rates when dosed 1 to 2 times per week.
Từ khóa
Tài liệu tham khảo
Fogarty
Rath, Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics [published online ahead of print October 24, 2013]., Crit Rev Biotechnol
National Hemophilia Foundation MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Available at:http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007. Accessed July 18, 2013
Lillicrap, 2010, Improvements in factor concentrates., Curr Opin Hematol, 17, 393, 10.1097/MOH.0b013e32833c06c6
Hacker, 2001, Barriers to compliance with prophylaxis therapy in haemophilia., Haemophilia, 7, 392, 10.1046/j.1365-2516.2001.00534.x
Peters, 2013, Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein., J Thromb Haemost, 11, 132, 10.1111/jth.12076
Roopenian, 2007, FcRn: the neonatal Fc receptor comes of age., Nat Rev Immunol, 7, 715, 10.1038/nri2155
Powell, 2012, Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients., Blood, 119, 3031, 10.1182/blood-2011-09-382846
European Medicines Agency Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2011/08/WC500109692.pdf. Accessed July 18, 2013
United States Food and Drug Administration Guidance for industry: assay development for immunogenicity testing of therapeutic proteins. Available at: http://www.fda.gov/downloads/Drugs/.../Guidances/UCM192750.pdf. Accessed July 18, 2013
Clopper, 1934, The use of confidence or fiducial limits illustrated in the case of binomial., Biometrika, 26, 404, 10.1093/biomet/26.4.404
Gouw, 2012, F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis., Blood, 119, 2922, 10.1182/blood-2011-09-379453
Margaglione, 2008, The Italian AICE-Genetics hemophilia A database: results and correlation with clinical phenotype., Haematologica, 93, 722, 10.3324/haematol.12427
Miller, 2012, F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicity., Haemophilia, 18, 375, 10.1111/j.1365-2516.2011.02700.x
Valentino, 2012, A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management., J Thromb Haemost, 10, 359, 10.1111/j.1538-7836.2011.04611.x
Recht, 2009, Clinical evaluation of moroctocog alfa (AF-CC), a new generation of B-domain deleted recombinant factor VIII (BDDrFVIII) for treatment of haemophilia A: demonstration of safety, efficacy, and pharmacokinetic equivalence to full-length recombinant factor VIII., Haemophilia, 15, 869, 10.1111/j.1365-2516.2009.02027.x
Manco-Johnson, 2013, Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose formulated recombinant factor VIII-FS in adults with severe hemophilia A (SPINART)., J Thromb Haemost, 11, 1119, 10.1111/jth.12202
Terraube, 2010, Factor VIII and von Willebrand factor interaction: biological, clinical and therapeutic importance., Haemophilia, 16, 3, 10.1111/j.1365-2516.2009.02005.x
Gallinaro, 2008, A shorter von Willebrand factor survival in O blood group subjects explains how ABO determinants influence plasma von Willebrand factor., Blood, 111, 3540, 10.1182/blood-2007-11-122945
Dumont, 2012, Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs., Blood, 119, 3024, 10.1182/blood-2011-08-367813
Mei, 2010, Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment., Blood, 116, 270, 10.1182/blood-2009-11-254755
Tiede, 2013, Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A., J Thromb Haemost, 11, 670, 10.1111/jth.12161
Kumar, 2013, Effect of unstructured polypeptide insertions on the recombinant expression of human factor VIII. International Society on Thrombosis and Haemostasis - 24th Congress; 2013; Amsterdam, Netherlands., J Thromb Haemost, 11, 711
Gouw, 2013, Factor VIII products and inhibitor development in severe hemophilia A., N Engl J Med, 368, 231, 10.1056/NEJMoa1208024
Ingerslev, 2004, FDA Workshop on factor VIII inhibitors held at the NIH, Bethesda, MA on 21 November 2003., Haemophilia, 10, 288, 10.1111/j.1365-2516.2004.00900.x
Whelan, 2013, Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients., Blood, 121, 1039, 10.1182/blood-2012-07-444877
Klintman, 2013, Antibody formation and specificity in Bethesda-negative brother pairs with haemophilia A., Haemophilia, 19, 106, 10.1111/j.1365-2516.2012.02903.x
Klintman, 2013, Long-term anti-FVIII antibody response in Bethesda-negative haemophilia A patients receiving continuous replacement therapy., Br J Haematol, 163, 385, 10.1111/bjh.12540