SEARCH

SEARCH BY CITATION

References

  • 1
    Mannucci PM, Tuddenham EG. The hemophilias–from royal genes to gene therapy. N Engl J Med 2001; 344: 17739.
  • 2
    Plug I, van der Bom JG, Peters M, Mauser-Bunschoten EP, de Goede-Bolder A, Heijnen L, Smit C, Zwart-van Rijkom JE, Willemse J, Rosendaal FR. Thirty years of hemophilia treatment in the Netherlands, 1972–2001. Blood 2004; 104: 3494500.
  • 3
    Adamson R. Design and operation of a recombinant mammalian cell manufacturing process for rFVIII. Ann Hematol 1994; 68(Suppl. 3): S914.
  • 4
    Toole JJ, Knopf JL, Wozney JM, Sultzman LA, Buecker JL, Pittman DD, Kaufman RJ, Brown E, Shoemaker C, Orr EC, Amphlett GW, Foster WB, Coe ML, Knutson GJ, Fass DN, Hewick RM. Molecular cloning of a cDNA encoding human antihaemophilic factor. Nature 1984; 312: 3427.
  • 5
    Kaufman RJ, Wasley LC, Furie BC, Furie B, Shoemaker CB. Expression, purification, and characterization of recombinant gamma-carboxylated factor IX synthesized in Chinese hamster ovary cells. J Biol Chem 1986; 261: 96228.
  • 6
    Mannucci PM. AIDS, hepatitis and hemophilia in the 1980s: memoirs from an insider. J Thromb Haemost 2003; 1: 20659.
  • 7
    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: 2532.
  • 8
    Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N Engl J Med 1993; 328: 4539.
  • 9
    Di Michele D. Inhibitor development in haemophilia B: an orphan disease in need of attention. Br J Haematol 2007; 138: 30515.
  • 10
    Ewenstein BM, Valentino LA, Journeycake JM, Tarantino MD, Shapiro AD, Blanchette VS, Hoots WK, Buchanan GR, Manco-Johnson MJ, Rivard GE, Miller KL, Geraghty S, Maahs JA, Stuart R, Dunham T, Navickis RJ. Consensus recommendations for use of central venous access devices in haemophilia. Haemophilia 2004; 10: 62948.
  • 11
    Santagostino E, Mancuso ME. Venous access in haemophilic children: choice and management. Haemophilia 2010; 16(Suppl. 1): 204.
  • 12
    Ryan SM, Mantovani G, Wang X, Haddleton DM, Brayden DJ. Advances in PEGylation of important biotech molecules: delivery aspects. Expert Opin Drug Deliv 2008; 5: 37183.
  • 13
    Gregoriadis G, Jain S, Papaioannou I, Laing P. Improving the therapeutic efficacy of peptides and proteins: a role for polysialic acids. Int J Pharm 2005; 300: 12530.
  • 14
    Yatuv R, Robinson M, Dayan-Tarshish I, Baru M. The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia. Int J Nanomedicine 2010; 5: 58191.
  • 15
    Huang C. Receptor-Fc fusion therapeutics, traps, and MIMETIBODY technology. Curr Opin Biotechnol 2009; 20: 6929.
  • 16
    Hilden I, Lauritzen B, Sorensen BB, Clausen JT, Jespersgaard C, Krogh BO, Bowler AN, Breinholt J, Gruhler A, Svensson LA, Petersen HH, Petersen LC, Balling KW, Hansen L, Hermit MB, Egebjerg T, Friederichsen B, Ezban M, Bjorn SE. Hemostatic effect of a monoclonal antibody mAb 2021 blocking the interaction between FXa and TFPI in a rabbit hemophilia model. Blood 2012; 119: 58718.
  • 17
    Waters EK, Genga RM, Schwartz MC, Nelson JA, Schaub RG, Olson KA, Kurz JC, McGinness KE. Aptamer ARC19499 mediates a procoagulant hemostatic effect by inhibiting tissue factor pathway inhibitor. Blood 2011; 117: 551422.
  • 18
    Toudjarska I, Cai Z, Racie T, Hettinger J, Milstein S, Bettencourt BR, Sah DW, Bumcrot D, Maraganore J, Vaishnaw A. RNAi-mediated inhibition of activated protein C- a new approach for hemophilia treatment. Blood (ASH Annual Meeting Abstracts) 2011; 118: 1204.
  • 19
    Akinc A, Sehgal A, Barros S, Hettinger J, Charisse K, Brodsky J, Qin J, Racie T. An RNAi therapeutic targeting antithrombin increases thrombin generation in nonhuman primates. ASH Annual Meeting Abstracts 2012; 120: 3370.
  • 20
    Abuchowski A, McCoy JR, Palczuk NC, van Es T, Davis FF. Effect of covalent attachment of polyethylene glycol on immunogenicity and circulating life of bovine liver catalase. J Biol Chem 1977; 252: 35826.
  • 21
    Veronese FM, Mero A. The impact of PEGylation on biological therapies. BioDrugs 2008; 22: 31529.
  • 22
    Gaberc-Porekar V, Zore I, Podobnik B, Menart V. Obstacles and pitfalls in the PEGylation of therapeutic proteins. Curr Opin Drug Discov Devel 2008; 11: 24250.
  • 23
    Caliceti P, Veronese FM. Pharmacokinetic and biodistribution properties of poly(ethylene glycol)-protein conjugates. Adv Drug Deliv Rev 2003; 55: 126177.
  • 24
    Pipe SW. Hemophilia: new protein therapeutics. Hematology Am Soc Hematol Educ Program 2010; 2010: 2039.
  • 25
    Pipe SW. The hope and reality of long-acting hemophilia products. Am J Hematol 2012; 87(Suppl. 1): S339.
  • 26
    Pasut G, Veronese FM. Polymer-drug conjugation, recent achievements and general strategies. Prog Polym Sci 2007; 32: 93361.
  • 27
    Capon DJ, Chamow SM, Mordenti J, Marsters SA, Gregory T, Mitsuya H, Byrn RA, Lucas C, Wurm FM, Groopman JE, Broder S, Smith DH. Designing CD4 immunoadhesins for AIDS therapy. Nature 1989; 337: 52531.
  • 28
    Kratz F. Albumin as a drug carrier: design of prodrugs, drug conjugates and nanoparticles. J Control Release 2008; 132: 17183.
  • 29
    Singh R, Lillard JW Jr. Nanoparticle-based targeted drug delivery. Exp Mol Pathol 2009; 86: 21523.
  • 30
    Baru M, Carmel-Goren L, Barenholz Y, Dayan I, Ostropolets S, Slepoy I, Gvirtzer N, Fukson V, Spira J. Factor VIII efficient and specific non-covalent binding to PEGylated liposomes enables prolongation of its circulation time and haemostatic efficacy. Thromb Haemost 2005; 93: 10618.
  • 31
    Spira J, Plyushch OP, Andreeva TA, Khametova RN. Evaluation of liposomal dose in recombinant factor VIII reconstituted with pegylated liposomes for the treatment of patients with severe haemophilia A. Thromb Haemost 2008; 100: 42934.
  • 32
    Powell JS, Nugent DJ, Harrison JA, Soni A, Luk A, Stass H, Gorina E. Safety and pharmacokinetics of a recombinant factor VIII with pegylated liposomes in severe hemophilia A. J Thromb Haemost 2008; 6: 27783.
  • 33
    Powell J, Martinowitz U, Windyga J, Di Minno G, Hellmann A, Pabinger I, Maas Enriquez M, Schwartz L, Ingerslev J. Efficacy and safety of prophylaxis with once-weekly BAY 79-4980 compared with thrice-weekly rFVIII-FS in haemophilia A patients. A randomised, active-controlled, double-blind study. Thromb Haemost 2012; 108: 91322.
  • 34
    Rostin J, Smeds AL, Akerblom E. B-Domain deleted recombinant coagulation factor VIII modified with monomethoxy polyethylene glycol. Bioconjug Chem 2000; 11: 38796.
  • 35
    Mei B, Pan C, Jiang H, Tjandra H, Strauss J, Chen Y, Liu T, Zhang X, Severs J, Newgren J, Chen J, Gu JM, Subramanyam B, Fournel MA, Pierce GF, Murphy JE. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood 2010; 116: 2709.
  • 36
    Coyle T, Reding M, Lin L, Michaelson L, Shah A, Powell J. An open-label phase I study to evaluate the pharmacokinetics and safety profile of Bay 94–9027, a PEGylated B-domain-deleted recombinant factor VIII, in previously treated patients with severe hemophilia A. Haemophilia 2012; 18: 22.
  • 37
    Agerso H, Stennicke HR, Pelzer H, Olsen EN, Merricks EP, Defriess NA, Nichols TC, Ezban M. Pharmacokinetics and pharmacodynamics of turoctocog alfa and N8-GP in haemophilia A dogs. Haemophilia 2012; 18: 9417.
  • 38
    Stennicke HR, Kjalke M, Karpf DM, Balling KW, Johansen PB, Elm T, Ovlisen K, Holmberg HL, Gudme CN, Persson E, Hilden I, Pelzer H, Rahbek-Nielsen H, Jespersgaard C, Bogsnes A, Pedersen AA, Kristensen AK, Peschke B, Kappers W, Rode F, et al. A novel B-domain O-glycoPEGylated FVIII (N8-GP) demonstrates full efficacy and prolonged effect in hemophilic mice models. Blood 2013; 121: 210816.
  • 39
    Tiede A, Brand B, Fischer R, Kavakli K, Lentz SR, Matsushita T, Rea C, Knobe K, Viuff D. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-man trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost 2013; 11: 6708.
  • 40
    Turecek PL, Bossard MJ, Graninger M, Gritsch H, Hollriegl W, Kaliwoda M, Matthiessen P, Mitterer A, Muchitsch EM, Purtscher M, Rottensteiner H, Schiviz A, Schrenk G, Siekmann J, Varadi K, Riley T, Ehrlich HJ, Schwarz HP, Scheiflinger F. BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation. Hamostaseologie 2012; 32(Suppl. 1): S2938.
  • 41
    Saenko EL, Pipe SW. Strategies towards a longer acting factor VIII. Haemophilia 2006; 12(Suppl. 3): 4251.
  • 42
    Rottensteiner H, Turecek PL, Pendu R, Meijer AB, Lenting P, Mertens K, Muchitsch E-M, Ehrlich H, Schwarz HP. PEGylation or polysialylation reduces FVIII binding to LRP resulting in prolonged half-life in murine models. Blood (ASH Annual Meeting Abstracts) 2007; 110: 3150.
  • 43
    Fogarty PF. Biological rationale for new drugs in the bleeding disorders pipeline. Hematology Am Soc Hematol Educ Program 2011; 2011: 397404.
  • 44
    Dumont JA, Liu T, Low SC, Zhang X, Kamphaus G, Sakorafas P, Fraley C, Drager D, Reidy T, McCue J, Franck HW, Merricks EP, Nichols TC, Bitonti AJ, Pierce GF, Jiang H. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood 2012; 119: 302430.
  • 45
    Peters RT, Toby G, Lu Q, Liu T, Kulman JD, Low SC, Bitonti AJ, Pierce GF. Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein. J Thromb Haemost 2013; 11: 13241.
  • 46
    Powell JS, Josephson NC, Quon D, Ragni MV, Cheng G, Li E, Jiang H, Li L, Dumont JA, Goyal J, Zhang X, Sommer J, McCue J, Barbetti M, Luk A, Pierce GF. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood 2012; 119: 30317.
  • 47
    Mahlangu J, Powell J, Ragni M, Chowdary P, Josephson N, Pabingerfasching I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi J, Krassova S, Jiang H, Li S, Neelakantan S, Greblikas F, Goyal J, Sommer J, et al. Phase 3 clinical study of recombinant FC fusion factor FVIII (RFVIIIFC) demonstrated safety, efficacy, and improved pharmacokinetics (A-LONG). Haemophilia 2013; 19: 70.
  • 48
    Ostergaard H, Bjelke JR, Hansen L, Petersen LC, Pedersen AA, Elm T, Moller F, Hermit MB, Holm PK, Krogh TN, Petersen JM, Ezban M, Sorensen BB, Andersen MD, Agerso H, Ahmadian H, Balling KW, Christiansen ML, Knobe K, Nichols TC, et al. Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide. Blood 2011; 118: 233341.
  • 49
    Negrier C, Knobe K, Tiede A, Giangrande P, Moss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood 2011; 118: 2695701.
  • 50
    Collins PW, Moss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost 2012; 10: 230512.
  • 51
    Peters RT, Low SC, Kamphaus GD, Dumont JA, Amari JV, Lu Q, Zarbis-Papastoitsis G, Reidy TJ, Merricks EP, Nichols TC, Bitonti AJ. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood 2010; 115: 205764.
  • 52
    Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallen B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF. Recombinant factor IX-Fc fusion protein (rFIX-Fc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood 2012; 119: 66672.
  • 53
    Diao L, Li S, Nestorov I, Jiang H. Population pharmacokinetic analysis of a longacting recombinant factor IX–FC fusion protein (RFIXFC) in patients with severe haemophilia B. Haemophilia 2013; 19: 33.
  • 54
    Metzner HJ, Weimer T, Kronthaler U, Lang W, Schulte S. Genetic fusion to albumin improves the pharmacokinetic properties of factor IX. Thromb Haemost 2009; 102: 63444.
  • 55
    Nolte MW, Nichols TC, Mueller-Cohrs J, Merricks EP, Pragst I, Zollner S, Dickneite G. Improved kinetics of rIX-FP, a recombinant fusion protein linking factor IX with albumin, in cynomolgus monkeys and hemophilia B dogs. J Thromb Haemost 2012; 10: 15919.
  • 56
    Santagostino E, Negrier C, Klamroth R, Tiede A, Pabinger-Fasching I, Voigt C, Jacobs I, Morfini M. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood 2012; 120: 240511.
  • 57
    Martinowitz U, Lubetsky A, Santagostino E, Jotov G, Luboshitz J, Lalezari S, Barazani-Brutman T, Voigt C, Moises T, Jacobs IC, Lissitchkov T. Efficacy, PK and safety results of a phase I/II clinical trial of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients with haemophilia B (PROLONG – 9FP). Blood (ASH Annual Meeting Abstracts) 2012; 120: 1121.
  • 58
    Yatuv R, Dayan I, Carmel-Goren L, Robinson M, Aviv I, Goldenberg-Furmanov M, Baru M. Enhancement of factor VIIa haemostatic efficacy by formulation with PEGylated liposomes. Haemophilia 2008; 14: 47683.
  • 59
    Spira J, Plyushch O, Zozulya N, Yatuv R, Dayan I, Bleicher A, Robinson M, Baru M. Safety, pharmacokinetics and efficacy of factor VIIa formulated with PEGylated liposomes in haemophilia A patients with inhibitors to factor VIII–an open label, exploratory, cross-over, phase I/II study. Haemophilia 2010; 16: 9108.
  • 60
    Ghosh S, Sen P, Pendurthi UR, Rao LV. Activity and regulation of glycoPEGylated factor VIIa analogs. J Thromb Haemost 2008; 6: 152533.
  • 61
    Johansen PB, Bjorn SE, Agerso H, Thorup I, Hermit MB, Sorensen B, Stennicke HR, Ezban M, Tranholm M. Prolonged effect of GlycoPEGylated rFVIIa (40k-PEG-rFVIIa) in rabbits correlates to activity in plasma. Thromb Haemost 2010; 104: 15764.
  • 62
    Karpf DM, Sorensen BB, Hermit MB, Holmberg HL, Tranholm M, Bysted BV, Groth AV, Bjorn SE, Stennicke HR. Prolonged half-life of glycoPEGylated rFVIIa variants compared to native rFVIIa. Thromb Res 2011; 128: 1915.
  • 63
    Moss J, Rosholm A, Lauren A. Safety and pharmacokinetics of a glycoPEGylated recombinant activated factor VII derivative: a randomized first human dose trial in healthy subjects. J Thromb Haemost 2011; 9: 136874.
  • 64
    Moss J, Stenmo C, Jimenez-Yuste V, Bottcher S, Fernandez I. Long-acting glycopegylated recombinant activated factor VII (LA-rFVIIa) is safe and shows a prolonged FVIIa half-life after single and multiple administrations in hemophilia A and B patients, making it suitable for development as prophylactic treatment of patients with hemophilia and inhibitors. Haemophilia 2010; 16: 31.
  • 65
    Liu T, Zhang X, Pan J, Sim D, McLean K, Cui Z, Sommer JM, Haaning J, Zhao X, Murphy JE, Pierce GF, Jiang H. Enhanced and prolonged efficacy of a novel recombinant FVIIa variant (BAY86-6150) for acute and prophylactic treatments in hemophilia A (HemA) mice. J Thromb Haemost 2009; 7: 222.
  • 66
    Mahlangu JN, Coetzee MJ, Laffan M, Windyga J, Yee TT, Schroeder J, Haaning J, Siegel JE, Lemm G. Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the recombinant factor VIIa variant BAY 86-6150 in hemophilia. J Thromb Haemost 2012; 10: 77380.
  • 67
    Salas J, Liu T, Kistanova E, Ashworth T, Slein M, Patel R, Kamphaus G, Correia A, Bitonti A, Dallabrida S, Jiang H, Peters R. Enhanced pharmacokinetics of factor VIIA as a monomeric FC fusion. J Thromb Haemost 2011; 9: 268.
  • 68
    Schulte S. Use of albumin fusion technology to prolong the half-life of recombinant factor VIIa. Thromb Res 2008; 122(Suppl. 4): S149.
  • 69
    Weimer T, Wormsbacher W, Kronthaler U, Lang W, Liebing U, Schulte S. Prolonged in-vivo half-life of factor VIIa by fusion to albumin. Thromb Haemost 2008; 99: 65967.
  • 70
    Chang JY, Chantrathammachart P, Monroe DM, Key NS. Studies on the mechanism of action of the aptamer BAX499, an inhibitor of tissue factor pathway inhibitor. Thromb Res 2012; 130: e1517.
  • 71
    Dockal M, Pachlinger R, Hartmann R, Knappe S, Sorensen B, Wong WY, Conlan M, Cecerle M, Ewenstein BM, Ehrlich HJ, Scheiflinger F. Biological explanation of clinically observed elevation of TFPI plasma levels after treatment with TFPI-antagonistic aptamer BAX 499. Blood (ASH Annual Meeting Abstracts) 2012; 120: 1104.
  • 72
    Kitazawa T, Igawa T, Sampei Z, Muto A, Kojima T, Soeda T, Yoshihashi K, Okuyama-Nishida Y, Saito H, Tsunoda H, Suzuki T, Adachi H, Miyazaki T, Ishii S, Kamata-Sakurai M, Iida T, Harada A, Esaki K, Funaki M, Moriyama C, et al. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med 2012; 18: 15704.
  • 73
    Wang W, Wang EQ, Balthasar JP. Monoclonal antibody pharmacokinetics and pharmacodynamics. Clin Pharmacol Ther 2008; 84: 54858.
  • 74
    Peyvandi F, Palla R, Menegatti M, Mannucci PM. Introduction. Rare bleeding disorders: general aspects of clinical features, diagnosis, and management. Semin Thromb Hemost 2009; 35: 34955.
  • 75
    Peyvandi F, Palla R, Menegatti M, Siboni SM, Halimeh S, Faeser B, Pergantou H, Platokouki H, Giangrande P, Peerlinck K, Celkan T, Ozdemir N, Bidlingmaier C, Ingerslev J, Giansily-Blaizot M, Schved JF, Gilmore R, Gadisseur A, Benedik-Dolnicar M, Kitanovski L, et al. Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders. J Thromb Haemost 2012; 10: 61521.
  • 76
    Carr ME Jr. Future directions in hemostasis: normalizing the lives of patients with hemophilia. Thromb Res 2010; 125(Suppl. 1): S7881.
  • 77
    Karimi M, Vafafar A, Haghpanah S, Payandeh M, Eshghi P, Hoofar H, Afrasiabi A, Gerdabi J, Ardeshiri R, Menegatti M, Peyvandi F. Efficacy of prophylaxis and genotype-phenotype correlation in patients with severe Factor X deficiency in Iran. Haemophilia 2012; 18: 2115.
  • 78
    Brenner B, Wiis J. Experience with recombinant-activated factor VII in 30 patients with congenital factor VII deficiency. Hematology 2007; 12: 5562.
  • 79
    Gonzalez-Boullosa R, Ocampo-Martinez R, Alarcon-Martin MJ, Suarez-Rodriguez M, Dominguez-Viguera L, Gonzalez-Fajo G. The use of activated recombinant coagulation factor VII during haemarthroses and synovectomy in a patient with congenital severe factor V deficiency. Haemophilia 2005; 11: 16770.
  • 80
    Divanon F, Hecquard C, Borel-Derlon A. Experience with use of recombinant activated factor VII. J Clin Pharm Ther 2002; 27: 1338.
  • 81
    Altisent C, Lozano M, Sol E, Valentìn P, Pico' M, Vila M, Puig LL. Dental extraction using rFVIIa in a patient with severe FV deficiency. Haemophilia 2000; 6: 408.
  • 82
    O'Connell NM. Factor XI deficiency–from molecular genetics to clinical management. Blood Coagul Fibrinolysis 2003; 14(Suppl. 1): S5964.
  • 83
    O'Connell NM, Riddell AF, Pascoe G, Perry DJ, Lee CA. Recombinant factor VIIa to prevent surgical bleeding in factor XI deficiency. Haemophilia 2008; 14: 77581.
  • 84
    Inbal A, Oldenburg J, Carcao M, Rosholm A, Tehranchi R, Nugent D. Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency. Blood 2012; 119: 51117.
  • 85
    Mannucci PM, Duga S, Peyvandi F. Recessively inherited coagulation disorders. Blood 2004; 104: 124352.
  • 86
    Werle M, Bernkop-Schnurch A. Strategies to improve plasma half life time of peptide and protein drugs. Amino Acids 2006; 30: 35167.
  • 87
    Mannucci PM. Evolution of the European guidelines for the clinical development of factor VIII products: little progress towards improved patient management. Haemophilia 2013; 19: 3448