Recombinant factor VIII Fc (Biogen/Swedish Orphan Biovitrium) for the treatment of hemophilia A

Bibliography

• Peyvandi F, Jayandharan G, Chandy M, et al. Genetic diagnosis of haemophilia and other inherited bleeding disorders. Haemophilia 2006;12(Suppl 3):82-9
   PubMedGoogle Scholar
• Mannucci PM, Tuddenham EG. The hemophilias – from royal genes to gene therapy. N Engl J Med 2001;344:1773-9
   PubMed Web of Science ®Google Scholar
• Peters RT, Toby G, Lu Q, et al. Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein. J Thromb Haemost 2013;11:132-41
   PubMed Web of Science ®Google Scholar
• Nilsson IM, Blomback M, Ahlberg A. Our experience in Sweden with prophylaxis on haemophilia. Bibl Haematol 1970;34:111-24
   PubMedGoogle Scholar
• 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
   PubMed Web of Science ®Google Scholar
• Collins P, Faradji A, Morfini M, et al. Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study. J Thromb Haemost 2010;8:83-9
   PubMed Web of Science ®Google Scholar
• Manco-Johnson MJ, Kempton CL, Reding MT, et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost 2013;11:1119-27
   PubMed Web of Science ®Google Scholar
• Collins PW, Blanchette VS, Fischer K, et al. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost 2009;7:413-20
   PubMed Web of Science ®Google Scholar
• Rath T, Baker K, Dumont JA, et al. Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics. Crit Rev Biotechnol 2013. [Epub ahead of print]
   PubMed Web of Science ®Google Scholar
• World Federation of Haemophilia Report on the Annual Global Survey 2012. World Federation of Hemophilia2013
   Google Scholar
• Freydin M. A review of treatment options for hemophilia. J Young Invest 2007;17:236-43
   Google Scholar
• Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia 2001;7:392-6
   PubMed Web of Science ®Google Scholar
• Fogarty PF. Biological rationale for new drugs in the bleeding disorders pipeline. Hematology Am Soc Hematol Educ Program 2011;2011:397-404
   PubMed Web of Science ®Google Scholar
• Lillicrap D. Improvements in factor concentrates. Curr Opin Hematol 2010;17:393-7
   PubMed Web of Science ®Google Scholar
• Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol 2007;7:715-25
   PubMed Web of Science ®Google Scholar
• Schultze HE, Heremans JF. Molecular biology of human proteins:with special reference to plasma proteins. Elselvier, New York;1996
   Google Scholar
• Peters T Jr. Serum albumin: recent progress in the understanding of its structure and biosynthesis. Clin Chem 1977;23:5-12
   PubMed Web of Science ®Google Scholar
• Watson D. Albumin and total globulin fractions of blood. Adv Clin Chem 1965;8:237-303
   PubMedGoogle Scholar
• Roopenian DC, Christianson GJ, Sproule TJ, et al. The MHC class I-like IgG receptor controls perinatal IgG transport, IgG homeostasis, and fate of IgG-Fc-coupled drugs. J Immunol 2003;170:3528-33
   PubMed Web of Science ®Google Scholar
• Waldmann TA, Terry WD. Familial hypercatabolic hypoproteinemia. A disorder of endogenous catabolism of albumin and immunoglobulin. J Clin Invest 1990;86:2093-8
   PubMed Web of Science ®Google Scholar
• Wani MA, Haynes LD, Kim J, et al. Familial hypercatabolic hypoproteinemia caused by deficiency of the neonatal Fc receptor, FcRn, due to a mutant beta2-microglobulin gene. Proc Natl Acad Sci USA 2006;103:5084-9
   PubMed Web of Science ®Google Scholar
• Dumont JA, Liu T, Low SC, et al. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood 2012;119:3024-30
   PubMed Web of Science ®Google Scholar
• Dumont JA, Low SC, Peters RT, Bitonti AJ. Monomeric Fc fusions: impact on pharmacokinetic and biological activity of protein therapeutics. BioDrugs 2006;20:151-60
   PubMed Web of Science ®Google Scholar
• van der Flier A, Liu Z, Light D, et al. Immunohistochemical staining of rFVIIIFc and rFVIII in liver cells differentiates between a VWF-dependent and independent clearance pathway in mice. Blood 2013;122(21):2331
   Google Scholar
• Liu Z, Goodman A, Peters R, et al. Characterization of a panel of Anti-FVIII Antibodies by domain specificity assignment, pairwise epitope overlap analysis and determination of affinities for FVIII and rFVIIIFc. [Abstract: PB 3.55-3]. International Society on Thrombosis and Haemostasis XXIV Congress, Amsterdam, The Netherlands; 2013. Available from: http://www.eventure-online.com/eventure/publicAbstractView.do?id=217968&congressId=6839
   Google Scholar
• Krishnamoorthy SBIH. Mechanisms of immune tolerance induction by rFVIIIFc in hemophilia A mice. [Abstract: OC 56.4]. International Society on Thrombosis and Haemostasis XXIV Congress 2013, Amsterdam, The Netherlands: 2014. Available from: http://www.eventure-online.com/eventure/publicAbstractView.do?id=217970&congressId=6839
   Google Scholar
• Lenting PJ, VAN Schooten CJ, Denis CV. Clearance mechanisms of von Willebrand factor and factor VIII. J Thromb Haemost 2007;5:1353-60
   PubMed Web of Science ®Google Scholar
• Terraube V, O’Donnell JS, Jenkins PV. Factor VIII and von Willebrand factor interaction: biological, clinical and therapeutic importance. Haemophilia 2010;16:3-13
   PubMed Web of Science ®Google Scholar
• Powell JS, Josephson NC, Quon D, et al. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood 2012;119:3031-7
   PubMed Web of Science ®Google Scholar
• Mahlangu J, Powell JS, Ragni J, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood 2014;123:317-25
   PubMed Web of Science ®Google Scholar
• Young G, Mahlangu J, Nolan B, et al. Pharmacokinetics Of recombinant factor VIII Fc fusion protein (rFVIIIFc) in pediatric subjects with hemophilia A: an interim analysis of the Kids A-LONG study. Blood 2013;122:3609
   Google Scholar
• Mei B, Pan C, Jiang H, et al. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood 2010;116:270-9
   PubMed Web of Science ®Google Scholar
• Shapiro AD. Long-lasting recombinant factor VIII proteins for hemophilia A. Hematology Am Soc Hematol Educ Program 2013;2013:37-43
   PubMedGoogle Scholar
• Carcao M. Changing paradigm of prophylaxis with longer acting factor concentrates. Haemophilia 2014;20(Suppl 4):99-105
   PubMed Web of Science ®Google Scholar
• Available from: http://www.abdserotec.com/immunoglobulin-antibody.html 2014
   Google Scholar