Recent advances in the development of coagulation factors and procoagulants for the treatment of hemophilia

Biochemical Pharmacology

Volumn 82, Issue 2, 2011, Pages 91-98

  Schaub, Robert G ^a  

^a Archemix Corporation   (United States)

Author keywords

Coagulation; Factor IX; Factor VIII; Hemophilia

Indexed keywords

ACTIVATED PROTEIN C; ARC 19499; AV 513; BAY 794980; BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; NN 1731; PROCOAGULANT; PROTEIN C ANTAGONIST; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG;

BLOOD CLOTTING; BLOOD CLOTTING FACTOR DEFICIENCY; CARBOXY TERMINAL SEQUENCE; DRUG EFFICACY; DRUG HALF LIFE; DRUG MECHANISM; DRUG SYNTHESIS; GENE MUTATION; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; MOLECULAR CLONING; NONHUMAN; PARTICLE SIZE; PRIORITY JOURNAL; PROTEIN DOMAIN; PROTEIN MODIFICATION; REVIEW; SEQUENCE HOMOLOGY;

EID: 79958144891     PISSN: 00062952     EISSN: 18732968     Source Type: Journal    
DOI: 10.1016/j.bcp.2011.03.015     Document Type: Note

References (62)

1. J.J. Toole, J.L. Knopf, J.M. Wozney, L.A. Sultzman, J.L. Buecker, and D.D. Pittman Molecular cloning of a cDNA encoding human antihaemophilic factor Nature 312 1984 342 347 (Pubitemid 15209311)
2. G.A. Vehar, B. Keyt, D. Eaton, H. Rodriguez, D.P. O'Brien, and F. Rotblat Structure of human factor VIII Nature 312 1984 337 342 (Pubitemid 15209310)
3. A. Poustka, A. Dietrich, G. Langenstein, D. Toniolo, S.T. Warren, and H. Lehrach Physical map of human Xq27-qter: localizing the region of the fragile X mutation Proc Natl Acad Sci USA 88 1991 8302 8306 (Pubitemid 21915344)
4. D. Freije, and D. Schlessinger A 1.6-Mb contig of yeast artificial chromosomes around the human factor VIII gene reveals three regions homologous to probes for the DXS115 locus and two for the DXYS64 locus Am J Hum Gene 51 1992 66 80
5. K.L. Wion, D. Kelly, J.A. Summerfield, E.G. Tuddenham, and R.M. Lawn Distribution of factor VIII mRNA and antigen in human liver and other tissues Nature 317 1985 726 729 (Pubitemid 16223638)
6. W.H. Kane, A. Ichinose, F.S. Hagen, and E.W. Davie Cloning of cDNAs coding for the heavy chain region and connecting region of human factor V, a blood coagulation factor with four types of internal repeats Biochemistry 26 20 1987 6508 6514 (Pubitemid 17155516)
7. A.R. Thompson Structure and function of the factor VIII gene and protein Semin Thromb Hemost 29 2003 11 22 (Pubitemid 36250259)
8. C.M. Jackson, and Y. Nemerson Blood coagulation Ann Rev Biochem 49 1980 765 811
9. R.A. Marlar, A.J. Kleiss, and J.H. Griffin Mechanism of action of human activated protein C, a thrombin-dependent anticoagulant enzyme Blood 59 1982 1067 1072 (Pubitemid 12125601)
10. C.A. Fulcher, J.E. Gardiner, J.H. Griffin, and T.S. Zimmerman Proteolytic inactivation of human factor VIII procoagulant protein by activated human protein C and its analogy with factor V Blood 63 1984 486 489 (Pubitemid 14188903)
11. G.C. White, F. Rosendaal, L.M. Aledort, J.M. Lusher, C. Rothschild, and J. Ingerslev Factor VIII and factor IX subcommittee definitions in hemophilia. Recommendations of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis Thromb Haemost 85 2001 560 (Pubitemid 32233035)
12. P.M. Mannucci, and E.D.G. Tuddenham The hemophilias - from royal genes to gene therapy N Engl J Med 344 2001 1773 1779 (Pubitemid 32497033)
13. K. Kurachi, and E.W. Davie Isolation and characterization of a cDNA coding for human factor IX Proc Natl Acad Sci USA 79 1982 6461 6464 (Pubitemid 13207514)
14. K.H. Choo, K.G. Gould, D.J. Rees, and G.G. Brownlee Molecular cloning of the gene for human anti-haemophilia factor IX Nature 299 1982 178 180 (Pubitemid 12056800)
15. P.F. Chance, K.A. Dyer, K. Kurachi, S. Yoshitake, H.H. Ropers, and P. Wieacker Regional localization of the human factor IX gene by molecular hybridization Hum Genet 65 1983 207 208 (Pubitemid 14210648)
16. M. Jaye, H. de la Salle, F. Schamber, A. Balland, V. Kohli, and A. Findeli Isolation of a human anti-haemophilia factor IX cDNA clone using a unique 52-base synthetic oligonucleotide probe deduced from the amino acid sequence of bovine factor IX Nucleic Acids Res 11 1983 2325 2335
17. P. Jagadeeswaran, D.E. Lavelle, R. Kaul, T. Mohandas, and S.T. Warrant Isolation and characterization of human factor IX cDNA: identification of taq I polymorphism and regional assignment Somat Cell Mol Genet 10 1984 465 473 (Pubitemid 14039229)
18. D.L. Aronson Factor IX complex Semin Thromb Hemost 6 1979 28 43 (Pubitemid 10169659)
19. K. Kurachi, S. Kurachi, M. Furukawa, and S.N. Yao Biology of factor IX Blood Coagul Fibrinolysis 4 1993 953 973
20. B. Osterud, and S.I. Rapaport Activation of factor IX by the reaction product of tissue factor and factor VII: additional pathway for initiating blood coagulation Proc Natl Acad Sci USA 74 1977 5260 5264
21. S.W. Herring, C. Abildgaard, K.T. Shitaniski, J. Harrison, S. Gendler, and C.M. Heldebrant Human coagulation factor IX: assessment of thrombogenicity in animal models and viral safety J Lab Clin Med 121 1993 394 405 (Pubitemid 23105702)
22. D. Doyle Thomas Addis of Edinburgh (1881-1949) and the coagulation cascade: 'for the greatest benefit done to practical medicine' Br J Haematol 132 2005 268 276 (Pubitemid 43381533)
23. A.J. Patek, F.H. Taylor, and I.I. Hemophilia Some properties of a substance obtained form normal human plasma effective in accelerating the coagulation of hemophilic blood J Clin Invest 16 1937 113 124
24. K.M. Brinkhous Clotting defect in hemophilia: deficiency in a plasma factor required for platelet utilization Proc Soc Exp Biol Med 66 1947 117 120
25. P.M. Aggeler, S.G. White, M.B. Glendening, E.W. Page, T.B. Leake, and G. Bates Plasma thromboplastin component (PTC) deficiency; a new disease resembling hemophilia Proc Soc Exp Biol Med 79 1952 692 694
26. R. Biggs, A.S. Douglas, R.G. MacFarlane, J.V. Dacie, W.R. Pitney, and C. Merskey Christmas disease: a condition previously mistaken for haemophilia Br Med J 2 1952 1378 1382
27. J.G. Pool, E.J. Hershgold, and A.R. Pappenhagen High-potency antihaemophilic factor concentrate prepared from cryoglobulin precipitate Nature 203 1964 312
28. M. Lee, M. Morfini, C. Negrier, and V. Chamouard The pharmacokinetics of coagulation factors Haemophilia 12 Suppl. 3 2006 1 7
29. P. Bailon, and C.-Y. Wong PEG-modified biopharmaceuticals Expert Opin Drug Deliv 6 2009 1 16
30. J. Spira, O.P. Plyushch, A. Tatyana, A. Andreev, and Y. Andreev Prolonged bleeding-free period following prophylactic infusion of recombinant factor VIII reconstituted with pegylated liposomes Blood 108 2006 3668 3673 (Pubitemid 44864543)
31. J.S. Powell, D.J. Nugent, J.A. Harrison, A. Soni, A. Luk, and H. Stass Safety and pharmacokinetics of a recombinant factor VIII with pegylated liposomes in severe hemophilia A J Thromb Haemost 6 2008 277 283
32. J. Pan, T. Liu, J.-Y. Yun Kim, D. Zhu, C. Patel, and Z.-H. Cui Enhanced efficacy of recombinant FVIII in noncovalent complex with PEGylated liposome in hemophilia a mice Blood 114 2009 2802 2811
33. G. DiMinno, A.M. Cerbone, A. Coppola, E. Cimino, M. Di Capua, and F. Pamparana Longer-acting factor VIII to overcome limitations in haemophilia management: the PEGylated liposomes formulation issue Haemophilia 16 Suppl. 1 2010 2 6
34. B. Mei, C. Pan, H. Jiang, H. Tjandra, J. Strauss, and Y. Chen Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment Blood 116 2010 270 279
35. M. Recht, L. Nemes, M. Matysiak, M. Manco-Johnson, J. Lusher, and M. Smith Clinical evaluation of moroctocog alfa [AF-CC], a new generation of b-domain deleted recombinant factor VIII (BDDrFVIII) for treatment of hemophilia A: demonstration of safety, efficacy, and pharmacokinetic equivalence to full-length recombinant factor VIII Haemophilia 15 2009 869 880
36. M.L.S. Christiansen, K.W. Balling, E. Presson, I. Hilden, A. Bagger-Sorensen, and B.B. Borensen Functional characteristics of N8, a new recombinant FVIII Haemophilia 16 2010 878 887
37. L. Thim, B. Vandahl, J. Karlsson, N.K. Klausen, J. Pedersen, and T.N. Krough Purification and characterization of a new recombinant factor VIII [N8] Haemophilia 16 2010 349 359
38. J.A. Dumont, G.D. Kamphaus, C. Fraley, T. Ashworth, H. Franck, and E.P. Merricks Factor VIII-Fc fusion protein shows extended half-life and hemostatic activity in hemophilia A dogs Blood 114 2009 545
39. R.T. Peters, S.C. Low, G.D. Kamphaus, J.A. Dumont, J.V. Amari, and Q. Lu Prolonged activity of factor IX as a monomeric Fc fusion protein Blood 115 2010 2057 2064
40. G.C. White, A. Beebe, and B. Nielsen Recombinant factor IX Thromb Haemost 78 1997 261 265 (Pubitemid 27289251)
41. R. Hartmann, M. Dockal, W. Kammlander, E. Panholzer, G.A. Nicolaes, and C. Fiedler Factor IX mutants with enhanced catalytic activity J Thromb Haemost 7 2009 1656 1662
42. P. Simioni, D. Tormene, G. Tognin, S. Gavasso, C. Bulato, and N.P. Iacobelli X-linked thrombophilia with a mutant Factor IX [Factor IX Padua] N Engl J Med 361 2009 1671 1675
43. H.R. Roberts, D.M. Monroe, and G.C. White The use of recombinant factor VIIa in the treatment of bleeding disorders Blood 104 2004 3858 3864 (Pubitemid 39620130)
44. A. Srivistava WFH Treatment Guidelines Working Group Guidelines for the management of haemophilia 2005 World Federation of Haemophilia
45. G.A. Allen, E. Persson, R.A. Campbell, M. Ezban, U. Hedner, and A.S. Wolberg A variant of recombinant factor VIIa with enhanced procoagulant and antifibrinolyitc activities in an in vitro model of hemophilia Arterioscler Thromb Vasc Biol 27 2007 683 689
46. B. Sorensen, E. Persson, and J. Ingerslev Factor VIIa analogue (V158D/E296V/M298Ql-FVIIa) normalises clot formation in whole blood from patients with severe haemophilia A Br J Haematol 137 2007 158 165 (Pubitemid 46474712)
47. B. Lauritzen, M. Tranholm, and M. Esban rFVIIa and a new enhanced rFVIIa-analogue NN1731, reduce bleeding in clopidogrel-treated and in thrombocytopenic rats J Thromb Haemost 7 2009 651 657
48. T. Liu, X. Zhang, J. Pan, D. Sim, K. McLean, and Z. Cui Enhanced and prolonged efficacy of a novel recombinant FVIIa variant (BAY86-6150) for acute and prophylactic treatments in hemophilia A (Hem A) mice J Thromb Haemost 7 Suppl. 2 2009 222
49. P.B. Johansen, S.E. Bjorn, H. Agerso, I. Thorup, M.B. Hermit, and B.B. Sorensen Prolonged effect of glycoPEGylated rFVIIa (40k-PEG-rFVIIa) in rabbits correlates to activity in plasma Thromb Haemost 104 2010 157 164
50. M.W. Bunce, R. Toso, and R.M. Camire Zymogen-like factor Xa variants restore thrombin generation and effectively bypass the intrinsic pathway in vitro Blood 117 2011 290 298
51. J.H. Griffin, J.A. Fernandez, A.J. Gale, and L.O. Mosnier Activated protein C J Thromb Haemost 5 Suppl. 1 2007 73 80 (Pubitemid 46958820)
52. K. van Dijk, J.G. van der Bom, K. Fischer, D.E. Grobbee, and H.M. van den Berg Do prothrombotic factors influence clinical phenotype of severe haemophilia? A review of the literature Thromb Haemost 92 2004 305 310 (Pubitemid 39172311)
53. A. Schlachterman, J. Schuettrumpf, J.-H. Liu, C.F. Freguia, R. Toso, and M. Poncz Factor V Leiden improves in vivo hemostasis in murine hemophilia models J Thromb Haemost 3 2003 2730 2737 (Pubitemid 41819802)
54. S. Butenas, T. Orfeo, M. Kalafatas, and K. Mann Peptidomimetic inhibitors for activated protein C: implications for hemophilia management J Thromb Haemost 4 2006 2411 2416 (Pubitemid 44575437)
55. H. Kato Regulation of functions of vascular wall cells by tissue factor pathway inhibitor: basic and clinical aspects Arterioscler Thromb Vasc Biol 22 2002 539 548 (Pubitemid 34408731)
56. G.J. Broze Jr. The rediscovery and isolation of TFPI Thromb Haemost 1 2003 1671 1675
57. J.T.B. Crawley, and D.A. Lane The haemostatic role of tissue factor pathway inhibitor Arterioscler Thromb Vasc Biol 28 2008 233 242 (Pubitemid 351161194)
58. T. Liu, C.D. Scallan, G.J. Broze Jr., S. Patarroyo-White, G.F. Pierce, and K.W. Johnson Improved coagulation in bleeding disorders by non-anticoagulant sulfated polysaccharides (NASP) Thromb Haemost 95 2006 68 76
59. E. Erhardtsen, M. Ezban, M.T. Madsen, V. Diness, S. Glazer, and U. Hedner Blocking of tissue factor pathway inhibitor (TFPI) shortens the bleeding time in rabbits with antibody induced haemophilia A Blood Coagul Fibrinolysis 6 1995 388 394
60. S. Prasad, D. Lillicrap, A. Labelle, S. Knappe, T. Keller, and E. Burnett Efficacy and safety of a new-class hemostatic drug candidate AV513, in dogs with hemophilia A Blood 111 2008 672 679
61. Emily K. Waters, Ryan M. Genga, Michael C. Schwartz, Jennifer A. Nelson, Robert G. Schaub, Karen A. Olson, et al., Aptamer ARC19499 mediates a procoagulant hemostatic effect by inhibiting tissue factor pathway inhibitor. Blood 2011. First Edition Paper, prepublished online March 9, 2011.
62. P.R. Bouchard, R.M. Hutabarat, and K.M. Thompson Discovery and development of therapeutic aptamers Annu Rev Pharmacol Toxicol 50 2010 237 257