Bibliography
- Mannucci PM, Tuddenham EG. The hemophilias--from royal genes to gene therapy. N Engl J Med 2001;344:1773-9
- Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia 2013;19:e1-47
- Pipe S. Antihemophilic factor (recombinant) plasma/albumin-free method for the management and prevention of bleeding episodes in patients with hemophilia A. Biologics 2009;3:117-25
- Coppola A, Di Capua M, Di Minno MN, et al. Treatment of hemophilia: a review of current advances and ongoing issues. J Blood Med 2010;1:183-95
- Coppola A, Tagliaferri A, Di Capua M, et al. Prophylaxis in children with hemophilia: evidence-based achievements, old and new challenges. Semin Thromb Hemost 2012;38:79-94
- Roosendaal G, Lafeber FP. Pathogenesis of haemophilic arthropathy. Haemophilia 2006;12(Suppl 3):117-21
- Martinowitz U, Bjerre J, Brand B, et al. Bioequivalence between two serum-free recombinant factor VIII preparations (N8 and ADVATE®)-an open-label, sequential dosing pharmacokinetic study in patients with severe haemophilia A. Haemophilia 2011;17:854-9
- Christiansen ML, Balling KW, Persson E, et al. Functional characteristics of N8, a new recombinant FVIII. Haemophilia 2010;16:878-87
- Petrini P. Identifying and overcoming barriers to prophylaxis in the management of haemophilia. Haemophilia 2007;13(Suppl 2):16-22
- Coppola A, Franchini M, Tagliaferri A. Prophylaxis in people with haemophilia. Thromb Haemost 2009;101:674-81
- 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
- MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Available from: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=57&contentid=1007 [Last accessed 4 November 2013]
- Berntorp E, Boulyjenkov V, Brettler D, et al. Modern treatment of haemophilia. Bull World Health Organ 1995;73:691-701
- Geraghty S, Dunkley T, Harrington C, et al. Practice patterns in haemophilia A therapy -- global progress towards optimal care. Haemophilia 2006;12:75-81
- Skinner MW. Haemophilia: provision of factors and novel therapies: world federation of hemophilia goals and achievements. Br J Haematol 2011;154:704-14
- Stonebraker JS, Brooker M, Amand RE, et al. A study of reported factor VIII use around the world. Haemophilia 2010;16:33-46
- Tiede A, Brand B, Fischer R, et al. 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 2013;11:670-8
- Thim L, Vandahl B, Karlsson J, et al. Purification and characterization of a new recombinant factor VIII (N8). Haemophilia 2010;16:349-59
- Burke RL, Pachl C, Quiroga M, et al. The functional domains of coagulation factor VIII:C. J Biol Chem 1986;261:12574-8
- Pittman DD, Alderman EM, Tomkinson KN, et al. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood 1993;81:2925-35
- Butler M, Meneses-Acosta A. Recent advances in technology supporting biopharmaceutical production from mammalian cells. Appl Microbiol Biotechnol 2012;96:885-94
- Omasa T, Onitsuka M, Kim WD. Cell engineering and cultivation of chinese hamster ovary (CHO) cells. Curr Pharm Biotechnol 2010;11:233-40
- Michnick DA, Pittman DD, Wise RJ, et al. Identification of individual tyrosine sulfation sites within factor VIII required for optimal activity and efficient thrombin cleavage. J Biol Chem 1994;269:20095-102
- Pittman DD, Wang JH, Kaufman RJ. Identification and functional importance of tyrosine sulfate residues within recombinant factor VIII. Biochemistry 1992;31:3315-25
- Leyte A, van Schijndel HB, Niehrs C, et al. Sulfation of Tyr1680 of human blood coagulation factor VIII is essential for the interaction of factor VIII with von Willebrand factor. J Biol Chem 1991;266:740-6
- Kannicht C, Ramström M, Kohla G, et al. Characterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIII. Thromb Res 2013;131:78-88
- Nielsen PF, Bak S, Vandahl B. Characterization of tyrosine sulphation in rFVIII (turoctocog alfa) expressed in CHO and HEK-293 cells. Haemophilia 2012;18:e397-8
- NovoEight® Prescribing Information. U S Food and Drug Administration. Available from: http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM371094.pdf [Last accessed 17 January 2014]
- Hansen JJ, Bolt G, Kjærgaard K, et al. Generation of recombinant FVIII purification antibody for manufacturing of N8. J Thromb Haemost 2009;7:820-1
- Newell JL, Fay PJ. Acidic residues C-terminal to the A2 domain facilitate thrombin-catalyzed activation of factor VIII. Biochemistry 2008;47:8786-95
- Burnouf T, Radosevich M. Nanofiltration of plasma-derived biopharmaceutical products. Haemophilia 2003;9:24-37
- Winkler CJ, Jorba N, Shitanishi KT, et al. Protein sieving characteristics of sub-20-nm pore size filters at varying ionic strength during nanofiltration of Coagulation Factor IX. Biologicals 2013;41:176-83
- Koenderman AH, ter Hart HG, Prins-de Nijs IM, et al. Virus safety of plasma products using 20 nm instead of 15 nm filtration as virus removing step. Biologicals 2012;40:473-81
- Soluk L, Price H, Sinclair C, et al. Pathogen safety of intravenous Rh immunoglobulin liquid and other immune globulin products: enhanced nanofiltration and manufacturing process overview. Am J Ther 2008;15:435-43
- Stuckey J, Strauss D, Venkiteshwaran A, et al. A novel approach to achieving modular retrovirus clearance for a parvovirus filter. Biotechnol Prog 2013. [Epub ahead of print]
- Casademunt E, Martinelle K, Jernberg M, et al. The first recombinant human coagulation factor VIII of human origin: human cell line and manufacturing characteristics. Eur J Haematol 2012;89:165-76
- Furuya K, Murai K, Yokoyama T, et al. Implementation of a 20-nm pore-size filter in the plasma-derived factor VIII manufacturing process. Vox Sang 2006;91:119-25
- Kim IS, Choi YW, Kang Y, et al. Improvement of virus safety of an antihemophilc factor IX by virus filtration process. J Microbiol Biotechnol 2008;18:1317-25
- Monroe DM, Hoffman M, Roberts HR. Platelets and thrombin generation. Arterioscler Thromb Vasc Biol 2002;22:1381-9
- Elm T, Karpf DM, Øvlisen K, et al. Pharmacokinetics and pharmacodynamics of a new recombinant FVIII (N8) in haemophilia A mice. Haemophilia 2012;18:139-45
- Karpf DM, Kjalke M, Thim L, et al. Pharmacokinetics and ex vivo whole blood clot formation of a new recombinant FVIII (N8) in haemophilia A dogs. Haemophilia 2011;17:e963-8
- Pastoft AE, Lykkesfeldt J, Ezban M, et al. A sensitive venous bleeding model in haemophilia A mice: effects of two recombinant FVIII products (N8 and Advate®). Haemophilia 2012;18:782-8
- Ingerslev J, Jankowski MA, Weston SB, et al. Collaborative field study on the utility of a BDD factor VIII concentrate standard in the estimation of BDDr Factor VIII:C activity in hemophilic plasma using one-stage clotting assays. J Thromb Haemost 2004;2:623-8
- Viuff D, Barrowcliffe T, Saugstrup T, et al. International comparative field study of N8 evaluating factor VIII assay performance. Haemophilia 2011;17:695-702
- Barrowcliffe TW, Raut S, Sands D, et al. Coagulation and chromogenic assays of factor VIII activity: general aspects, standardization, and recommendations. Semin Thromb Hemost 2002;28:247-56
- Lentz SR, Misgav M, Ozelo M, et al. Results from a large multinational clinical trial (guardian1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy. Haemophilia 2013;19:691-7
- Kulkarni R, Karim FA, Glamocanin S, et al. Results from a large multinational clinical trial (guardian3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety, efficacy and pharmacokinetics. Haemophilia 2013;19:698-705
- European Medicines Agency. Committee for medicinal products for human use (CHMP). Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2011/08/WC500109692.pdf [Last accessed 4 November 2013]
- Abshire TC, Brackmann HH, Scharrer I, et al. Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy--International Kogenate-FS Study Group. Thromb Haemost 2000;83:811-16
- Lusher JM, Lee CA, Kessler CM, et al. The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A. Haemophilia 2003;9:38-49
- Recht M, Nemes L, Matysiak M, et al. 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 2009;15:869-80
- Tarantino MD, Collins PW, Hay CR, et al. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Haemophilia 2004;10:428-37
- Blanchette VS, Shapiro AD, Liesner RJ, et al. Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008;6:1319-26
- Smith MP, Giangrande P, Pollman H, et al. A postmarketing surveillance study of the safety and efficacy of ReFacto (St Louis-derived active substance) in patients with haemophilia A. Haemophilia 2005;11:444-51
- Valentino LA, Mamonov V, Hellmann A, et al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost 2012;10:359-67
- Santagostino E, Lentz SR, Misgav M, et al. Surgery with turoctocog alfa: efficacy in bleeding prevention during surgical procedures-results from the guardian™ trials. Blood 2013;120:2228
- Bolton-Maggs PH, Pasi KJ. Haemophilias A and B. Lancet 2003;361:1801-9
- Peyvandi F, Garagiola I, Seregni S. Future of coagulation factor replacement therapy. J Thromb Haemost 2013;11(Suppl 1):84-98
- Pipe SW. Hemophilia: new protein therapeutics. Hematology Am Soc Hematol Educ Program 2010;2010:203-9
- 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
- Evatt BL. The tragic history of AIDS in the hemophilia population, 1982-1984. J Thromb Haemost 2006(4):2295-301
- Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011;365:2357-65
- High KA. The gene therapy journey for hemophilia: are we there yet? Blood 2012;120:4482-7
- Björkman 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
- Gomperts ED. The need for previously untreated patient population studies in understanding the development of factor VIII inhibitors. Haemophilia 2006;12:573-8
- Gouw SC, van der Bom JG, Ljung R, et al. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med 2013;368:231-9
- Franchini M, Mengoli C. RODIN and the pitfalls of observational studies. Haemophilia 2013;19:e315-16
- Kessler CM, Iorio A. The rodin (Research of determinants of inhibitor development among PUPs with haemophilia) study: the clinical conundrum from the perspective of haemophilia treaters. Haemophilia 2013;19:351-4
- Skinner MW. WFH: closing the global gap--achieving optimal care. Haemophilia 2012;18(Suppl 4):1-12