The fraction of recombinant factor VIII:Ag unable to bind von Willebrand factor has no FVIII coagulant activity: Studies in vitro

Haemophilia

Volumn 18, Issue 6, 2012, Pages 917-925

  Ofosu, F A ^a   Tse, H ^a   Naqvi, A ^a   Bhakta, H ^a   Song, Y ^a  

^a MCMASTER UNIVERSITY   (Canada)

Author keywords

ELISA; Factor VIII; Gene therapy; VonWillebrand factor

Indexed keywords

BLOOD CLOTTING FACTOR 10A; CALCIUM CHLORIDE; COAGULATING AGENT; HIRUDIN; PHOSPHOLIPID; RECOMBINANT BLOOD CLOTTING FACTOR 8; VON WILLEBRAND FACTOR;

ARTICLE; ENZYME LINKED IMMUNOSORBENT ASSAY; GENE THERAPY; HUMAN; IN VITRO STUDY; NONHUMAN; PRIORITY JOURNAL; PROTEIN BINDING;

BLOOD COAGULATION TESTS; ENZYME-LINKED IMMUNOSORBENT ASSAY; FACTOR IXA; FACTOR VIII; FACTOR XA; PROTEIN BINDING; VON WILLEBRAND FACTOR;

EID: 84868212659     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2012.02861.x     Document Type: Article

References (32)

1. Lusher JM. First and second-generation recombinant factor VIII concentrates in previously untreated patients recovery, safety, efficacy, and inhibitor development. Semin Thromb Hemost 2002; 28: 273-6.
2. Manco-Johnson M, Abshire T, Shapiro A, Riske B. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia A. New England J Med 2007; 357: 535-44.
3. Goudemand J, Rothschild C, Demiguel V et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 2006; 12: 128-32.
4. Kreuz W, Ettingshausen CE, Auerswald G et al. Epidemiology of inhibitors and current treatment strategies. Haematologica 2003; 88: ERPO4.
5. Wright J, Paisley S.. The epidemiology of inhibitors in hemophilia A: a systematic review. Haemophilia 2003; 9: 418-35.
6. Gringeri A, Musso R, Mazzucconi MG et al. For the RITS-FITNHES Study Group Immune tolerance induction with a high purity von Willebrand factor/factor VIII complex concentrate in hemophilia A patients with inhibitors at high risk of a poor response. Haemophilia 2007; 13: 373-9.
7. Wang XF, Zhao YQ, Yang RC et al. The prevalence of factor VIII inhibitors and genetic aspects of inhibitor development in Chinese patients with haemophilia A. Haemophilia 2010; 16: 632-9.
8. Dimichele DM. Management of factor VIII inhibitors. Int J Hematol 2006; 83: 119-25.
9. Vlot AJ, Koppleman SJ, van den Berg H, Bouma BN, Sixma JJ. The affinity and stoichiometry of binding of human factor VIII to von Willebrand factor. Blood 1995; 85: 3150-7.
10. Hoyer LW. The factor FVIII complex: structure and function. Blood 1981; 58: 1-13.
11. Rapaport SI, Schiffman S, Patch MJ, Ames SB. The importance of activation of anti-haemophilic globulin and proaccelerin by traces of thrombin in the generation of intrinsic prothrombinase activity. Blood 1963; 21: 221-36.
12. Koedam JA, Meijers JCM, Sixma JJ, Bouma BN. Inactivation of human FVIII by activated protein C Cofactor activity of protein S and protective effect of von Willebrand factor. J Clin Invest 1988; 82: 1236-43.
13. Koedam JA, Hamer RJ, Beeser-Visser NH, Bouma BN, Sixma JJ. The effect of von Willebrand factor on activation of FVIII by factor Xa. Eur J Biochem 1990; 189: 229-34.
14. Fay PJ. Activation of factor VIII and mechanism of cofactor action. Blood Rev 2004; 18: 1-15.
15. Fujikawa K, Coan MH, Lagaz ME, Davie EW. The mechanism of activation of bovine factor X by intrinsic and extrinsic pathways. Biochem 1974; 13: 5290-9.
16. Lin Y, Yang X, Chevrier M et al. Relationships between factor VIII:Ag and factor VIII in recombinant and plasma-derived factor VIII concentrates. Haemophilia 2004; 10: 459-69.
17. Behrmann M, Pasi J, Saint-Remy JM, Kotitschke R, Kloft M. Von Willebrand factor modulates factor VIII immunogenicity: comparative study of different factor VIII concentrates in a hemophilia A mouse model. Thromb Haemost 2002; 88: 221-9.
18. Lacroix-Desmazes S, Represse Yohann, Karen S, Dasgupta S. The role of VWF in the immunogenicity of FVIII. Thrombosis Res 2008; 122: S3-6.
19. Lollar P, Knutson GJ, Fass DN. Stabilization of thrombin-activated porcine factor VIII by factor IXa and phospholipids. Blood 1984; 63: 1303-8.
20. Ofosu FA, Blajchman MA, Hirsh J. The inhibition by heparin of the intrinsic pathway of factor X activation in the absence of antithrombin-III. Thromb Res 1980; 20: 391-401.
21. Laemmli UK. Cleavage of structural proteins during the assembly of the head of bacteriophage T4. Nature 1970; 227: 680-5.
22. Mannucci PM, Gringeri A, Peyvandi E, Santagostino E. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers. Haemophilia 2007; 13(Suppl 5): 65-8.
23. Greninger DA, Saint-Rémy JM, Jacquemin M, Benhida A, Dimichele DM. The use of factor VIII/von Willebrand factor concentrate for immune tolerance induction in haemophilia A patients with high titer inhibitors: association with clinical outcome with inhibitor epitope profile. Haemophilia 2008; 14: 295-302.
24. Kreuz W. The role of VWF for the success of immune tolerance induction. Thromb Res 2008; 122(Suppl 2): S7-12.
25. Delignat S, Dasgupta S, Andre S et al. Comparison of the immunogenicity of different therapeutic preparations of factor VIII in the murine model of hemophilia A. Haematologica 2007; 92: 1423-6.
26. Dasgupta S, Repesse Y, Bayry J et al. VWF protects FVIII from endocytosis by dendritic cells and subsequent presentation to immune effectors. Blood 2007; 109: 610-2.
27. Leyte A, Van Schijndel HB, Niehrs C et al. Sulfation of Tyr 1680 of human blood coagulation factor VIII is essential for the interaction of factor VIII with von Willebrand factor. J Biol Chem 1991; 266: 749-6.
28. Pitman DD, Wang JH, Kaufman RJ. Identification and functional importance of tyrosine sulfate residues within recombinant factor VIII. Biochemistry 1992; 31: 3315-25.
29. Mechanic DA, Pittman DD, Wise RJ, Kaufman RJ. Identification of individual tyrosine sulfation sites within factor VIII required for optimal activity and efficient thrombin cleavage. J Biol Chem 1994; 269: 20095-102.
30. Grancha S, Navajas R, Maranon C, Paradela A, Albar JP, Jorquera JI. Incomplete tyrosine 1680 sulphation in recombinant FVIII concentrates. Haemophilia 2011; 17: 709-10.
31. Cannich C. The human cell line as a superior expression system- what are the benefits. A special symposium at the ISTH Congress 2009, pp 4-13
32. Christiansen MLS, Balling KW, Persson E et al. Functional characterization of N8, a new recombinant factor VIII. Haemophilia 2010; 16: 878-87.