Development of long-acting recombinant FVIII and FIX Fc fusion proteins for the management of hemophilia

Bibliography

• 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
• Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 1992;232:25-32
   PubMed Web of Science ®Google Scholar
• Raffini L, Manno C. Modern management of haemophilic arthropathy. Br J Haematol 2007;136:777-87
   PubMed Web of Science ®Google Scholar
• Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia a (the ESPRIT Study). J Thromb Haemost 2011;9:700-10
   PubMed Web of Science ®Google 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
• Royal S, Schramm W, Berntorp E, et al. Quality-of-life differences between prophylactic and on-demand factor replacement therapy in European haemophilia patients. Haemophilia 2002;8:44-50
   PubMed Web of Science ®Google Scholar
• Shapiro AD, Donfield SM, Lynn HS, et al. Defining the impact of hemophilia: the academic achievement in children with hemophilia study. Pediatrics 2001;108:E105
   PubMed Web of Science ®Google Scholar
• National Hemophilia Foundation, Medical and Scientific Advisory Council. MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). 2007. Available from: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007 [Last accessed 10 January 2013]
   Google Scholar
• World Federation of Hemophilia. Guidelines for the management of hemophilia. 2012. Available from: http://www1.wfh.org/publications/files/pdf-1472.pdf [Last accessed 14 January 2013]
   Google Scholar
• Collins PW. Personalized prophylaxis. Haemophilia 2012;18(Suppl 4):131-5
   PubMed Web of Science ®Google Scholar
• Collins PW, Fischer K, Morfini M, et al. Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia 2011;17:2-10
   PubMed Web of Science ®Google Scholar
• Lindvall K, Astermark J, Bjorkman S, et al. Daily dosing prophylaxis for haemophilia: a randomized crossover pilot study evaluating feasibility and efficacy. Haemophilia 2012;18:855-9
   PubMed Web of Science ®Google Scholar
• Fisher K. Optmimizing efficacy of factor VIII prophylaxis for severe haemophilia A. Haemophilia 2011;17(Suppl 3):9-15
   Google Scholar
• Bolton-Maggs PH, Pasi KJ. Haemophilias A and B. Lancet 2003;361:1801-9
   PubMed Web of Science ®Google Scholar
• Fijnvandraat K, Peters M, ten Cate JW. Inter-individual variation in half-life of infused recombinant factor VIII is related to pre-infusion von Willebrand factor antigen levels. Br J Haematol 1995;91:474-6
   PubMed Web of Science ®Google Scholar
• van Dijk K, van der Bom JG, Lenting PJ, et al. Factor VIII half-life and clinical phenotype of severe hemophilia A. Haematologica 2005;90:494-8
   PubMed Web of Science ®Google Scholar
• Fischer K, Pendu R, van Schooten CJ, et al. Models for prediction of factor VIII half-life in severe haemophiliacs: distinct approaches for blood group O and non-O patients. PLoS ONE 2009;4:e6745
   PubMed Web of Science ®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
• White GC II, Beebe A, Nielsen B. Recombinant factor IX. Thromb Haemost 1997;78:261-5
   PubMed Web of Science ®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
• Richards M, Altisent C, Batorova A, et al. Should prophylaxis be used in adolescent and adult patients with severe haemophilia? An European survey of practice and outcome data. Haemophilia 2007;13:473-9
   PubMed Web of Science ®Google Scholar
• Valentino LA, Ewenstein B, Navickis RJ, Wilkes MM. Central venous access devices in haemophilia. Haemophilia 2004;10:134-46
   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
• Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia 2009;15:1219-27
   PubMed Web of Science ®Google Scholar
• Lillicrap D. The improved factor concentrate. Hämostaseologie 2009;29:71-3
   PubMed Web of Science ®Google Scholar
• Walsh G, Jefferis R. Post-translational modifications in the context of therapeutic proteins. Nat Biotechnol 2006;24:1241-52
   PubMed Web of Science ®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
• Stennicke HR, Kjalke M, Karpf DM, et al. A novel B-domain O-glycoPEGylated FVIII (N8-GP) demonstrates full efficacy and prolonged effect in hemophilic mice models. Blood 2013;121:2108-16
   PubMed Web of Science ®Google Scholar
• Negrier C, Knobe K, Tiede A, et al. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood 2011;118:2695-701
   PubMed Web of Science ®Google Scholar
• Turecek PL, Bossard MJ, Graninger M, et al. BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation. Hamostaseologie 2012;32(Suppl 1):S29-38
   PubMed Web of Science ®Google Scholar
• Ivens IA, Baumann A, McDonald TA, et al. PEGylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregivers. Haemophilia 2013;19:11-20
   PubMed Web of Science ®Google Scholar
• Negrier C, Knobe K, Tiede A, et al. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood 2011;118:2695-701
   PubMed Web of Science ®Google Scholar
• Pipe SW, Kaufman RJ. Characterization of a genetically engineered inactivation-resistant coagulation factor VIIIa. Proc Natl Acad Sci USA 1997;94:11851-6
   PubMed Web of Science ®Google Scholar
• Bensen-Kennedy D. Bringing new therapy options to the hemophilia community. Thromb Res 2013;131(Suppl 2):S15-18
   PubMedGoogle Scholar
• Andersen JT, Sandlie I. The versatile MHC class I-related FcRn protects IgG and albumin from degradation: implications for development of new diagnostics and therapeutics. Drug Metab Pharmacokinet 2009;24:318-32
   PubMed Web of Science ®Google Scholar
• Chaudhury C, Mehnaz S, Robinson JM, et al. The major histocompatibility complex-related Fc receptor for IgG (FcRn) binds albumin and prolongs its lifespan. J Exp Med 2003;197:315-22
   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
• Schulte S. Half-life extension through albumin fusion technologies. Thromb Res 2009;124(Suppl 2):S6-8
   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. Submitted 2013
   Google Scholar
• Lencer WI, Blumberg RS. A passionate kiss, then run: exocytosis and recycling of IgG by FcRn. Trends Cell Biol 2005;15:5-9
   PubMed Web of Science ®Google Scholar
• Ghetie V, Ward ES. Multiple roles for the major histocompatibility complex class I-related receptor FcRn. Annu Rev Immunol 2000;18:739-66
   PubMed Web of Science ®Google Scholar
• McGarry T, Hough R, Rogers S, Rechsteiner M. Intracellular distribution and degradation of immunoglobulin G and immunoglobulin G fragments injected into HeLa cells. J Cell Biol 1983;96:338-46
   PubMed Web of Science ®Google Scholar
• Israel EJ, Taylor S, Wu Z, et al. Expression of the neonatal Fc receptor, FcRn, on human intestinal epithelial cells. Immunology 1997;92:69-74
   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;11:132-42
   PubMed Web of Science ®Google Scholar
• Peters RT, Low SC, Kamphaus GD, et al. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood 2010;115:2057-64
   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
• Durocher Y, Butler M. Expression systems for therapeutic glycoprotein production. Curr Opin Biotechnol 2009;20:700-7
   PubMed Web of Science ®Google Scholar
• Walsh G. Post-translational modifications of protein biopharmaceuticals. Drug Discov Today 2010;15:773-80
   PubMed Web of Science ®Google Scholar
• Swiech K, Picanco-Castro V, Covas DT. Human cells: new platform for recombinant therapeutic protein production. Protein Expr Purif 2012;84:147-53
   PubMed Web of Science ®Google Scholar
• Zhang M, Mezo A. Enzymatic release and detection of galactose-alpha-1,3-galactose (a-Gal) in recombinant FVIII products. Haemophilia 2012;18(Suppl S3):PO-MO-135
   Google Scholar
• Ghaderi D, Zhang M, Hurtado-Ziola N, Varki A. Production platforms for biotherapeutic glycoproteins. occurence, impact, and challenges of non-human sialylation. Biotechnol Genet Eng Rev 2012;28:147-76
   PubMed Web of Science ®Google Scholar
• Brinkhous KM, Sigman JL, Read MS, et al. Recombinant human factor IX: replacement therapy, prophylaxis, and pharmacokinetics in canine hemophilia B. Blood 1996;88:2603-10
   PubMed Web of Science ®Google Scholar
• Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001;98:3600-6
   PubMed Web of Science ®Google Scholar
• Poon M-C. Pharmacokinetics of factors IX, recombinant human activated factor VII and factor XIII. Haemophilia 2006;12(Suppl 4):61-9
   PubMedGoogle 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
• Shapiro AD, Ragni MV, Valentino LA, et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood 2012;119:666-72
   PubMed Web of Science ®Google Scholar
• BeneFIX. Prescribing Information. 2011. Available from: http://labelingpfizercom/showlabelingaspx?id=492 [Accessed 20 March 2013]
   Google Scholar
• Bjorkman S, Shapiro AD, Berntorp E. Pharmacokinetics of recombinant factor IX in relation to age of the patient: implications for dosing in prophylaxis. Haemophilia 2001;7:133-9
   PubMed Web of Science ®Google Scholar
• Kim HC, McMillan CW, White GC, et al. Purified factor IX using monoclonal immunoaffinity technique: clinical trials in hemophilia B and comparison to prothrombin complex concentrates. Blood 1992;79:568-75
   PubMed Web of Science ®Google Scholar
• Suiter T, Laffan M, Mannucci PM, et al. Recombinant human von Willebrand factor (rVWF): first-in-human study evaluating pharmacokinetics, demonstrating safety and tolerability in type 3 von Willebrand disease. Blood 2010;116:abstract 237
   Google Scholar
• Bjorkman S, Oh M, Spotts G, et al. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood 2012;119:612-18
   PubMed Web of Science ®Google Scholar
• Ljung R, Auerswald G, Benson G, et al. Novel coagulation factor concentrates: issues relating to their clinical implementation and pharmacokinetic assessment for optimal prophylaxis in haemophilia patients. Haemophilia 2013; [Epud ahead of print]
   PubMed Web of Science ®Google Scholar