Preclinical testing of human recombinant von willebrand factor: ADAMTS13 cleavage capacity in animals as criterion for species suitability

Seminars in Thrombosis and Hemostasis

Volumn 36, Issue 5, 2010, Pages 522-528

  Muchitsch, Eva Maria ^a   Dietrich, Barbara ^a   Rottensteiner, Hanspeter ^a   Auer, Wilfried ^a   Nehrbass, Dirk ^a   Gritsch, Herbert ^a   Ehrlich, Hartmut J ^a   Turecek, Peter L ^a   Peter Schwarz, Hans ^a  

^a BAXTER BIOSCIENCE   (Austria)

Author keywords

ADAMTS13 cleavage; Recombinant von Willebrand factor; rVWF; species suitability

Indexed keywords

BLOOD CLOTTING FACTOR; HUMAN RECOMBINANT VON WILLEBRAND FACTOR; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR CLEAVING PROTEINASE; ADAM PROTEIN; RECOMBINANT PROTEIN; VON WILLEBRAND FACTOR;

C57BL 6 MOUSE; DRUG EFFECT; DRUG SCREENING; HEMOSTASIS; HUMAN; IN VITRO STUDY; IN VIVO STUDY; KNOCKOUT MOUSE; MONKEY; MOUSE STRAIN; NONHUMAN; PRIORITY JOURNAL; RABBIT; REVIEW; SINGLE DRUG DOSE; THROMBOCYTOPENIA; WILD TYPE; ANIMAL; METABOLISM;

ADAM PROTEINS; ANIMALS; HUMANS; RECOMBINANT PROTEINS; VON WILLEBRAND FACTOR;

EID: 77954881627     PISSN: 00946176     EISSN: 10989064     Source Type: Journal    
DOI: 10.1055/s-0030-1255446     Document Type: Review

References (26)

1. Ruggeri Z M. Von Willebrand factor: looking back and looking forward. Thromb Haemost 2007 98 (1) 55-62
2. Sadler J E. von Willebrand factor: two sides of a coin. J Thromb Haemost 2005 3 (8) 1702-1709
3. Mannucci P M. Treatment of von Willebrand's disease. N Engl J Med 2004 351 (7) 683-694
4. Turecek P L., Mitterer A, Matthiessen H P. et al. Development of a plasma-and albumin-free recombinant von Willebrand factor. Hamostaseologie 2009 29 (Suppl 1) S32-S38
5. Turecek P L., Schrenk G, Rottensteiner H et al. Structure and function of a recombinant von Willebrand factor (VWF) drug candidate. Semin Thromb Hemost 2010 36 (5) 522-528
6. Fischer B, Mitterer A, Schlokat U, DenBouwmeester R, Dorner F. Structural analysis of recombinant von Willebrand factor: identification of hetero- and homo-dimers [published correction appears in FEBS Lett 1994;353:337]. FEBS Lett 1994 351 (3) 345-348
7. Fischer B E., Schlokat U, Mitterer A et al. Structural analysis of recombinant von Willebrand factor produced at industrial scale fermentation of transformed CHO cells co-expressing recombinant furin. FEBS Lett 1995 375 (3) 259-262
8. Schlokat U, Fischer B E., Herlitschka S et al. Production of highly homogeneous and structurally intact recombinant von Willebrand factor multimers by furin-mediated propeptide removal in vitro. Biotechnol Appl Biochem 1996 24 (Pt 3) 257-267
9. Turecek P L., Váradi K, Schlokat U, Pichler L, Dorner F, Schwarz H P. In vivo and in vitro processing of recombinant pro-von Willebrand factor. Histochem Cell Biol 2002 117 (2) 123-129
10. Fischer B E., Schlokat U, Reiter M, Mundt W, Dorner F. Biochemical and functional characterization of recombinant von Willebrand factor produced on a large scale. Cell Mol Life Sci 1997 53 (1112) 943-950
11. Plaimauer B, Schlokat U, Turecek P L. et al. Recombinant von Willebrand factor: preclinical development. Semin Thromb Hemost 2001 27 (4) 395-403
12. Turecek P L., Gritsch H, Pichler L et al. In vivo characterization of recombinant von Willebrand factor in dogs with von Willebrand disease. Blood 1997 90 (9) 3555-3567
13. Roussi J, Turecek P L., André P et al. Effects of human recombinant, plasma-derived and porcine von Willebrand factor in pigs with severe von Willebrand disease. Blood Coagul Fibrinolysis 1998 9 (4) 361-372
14. Schwarz H P., Dorner F, Mitterer A et al. Evaluation of recombinant von Willebrand factor in a canine model of von Willebrand disease. Haemophilia 1998 4 (suppl 3) 53-62
15. Schwarz H P., Dorner F, Mitterer A et al. Preclinical evaluation of recombinant von Willebrand factor in a canine model of von Willebrand disease. Wien Klin Wochenschr 1999 111 (5) 181-191
16. Turecek P L., Pichler L, Auer W et al. Evidence for extracellular processing of pro-von Willebrand factor after infusion in animals with and without severe von Willebrand disease. Blood 1999 94 1637-1647
17. Schwarz H P., Schlokat U, Mitterer A et al. Recombinant von Willebrand factorinsight into structure and function through infusion studies in animals with severe von Willebrand disease. Semin Thromb Hemost 2002 28 (2) 215-226
18. Levy G G., Nichols W C., Lian E C. et al. Mutations in a member of the ADAMTS gene family cause thrombotic thrombocytopenic purpura. Nature 2001 413 (6855) 488-494
19. Plaimauer B, Zimmermann K, Völkel D et al. Cloning, expression, and functional characterization of the von Willebrand factor-cleaving protease (ADAMTS13). Blood 2002 100 (10) 3626-3632
20. Tsai H M. Physiologic cleavage of von Willebrand factor by a plasma protease is dependent on its conformation and requires calcium ion. Blood 1996 87 (10) 4235-4244
21. Moake J. Thrombotic thrombocytopenia purpura (TTP) and other thrombotic microangiopathies. Best Pract Res Clin Haematol 2009 22 (4) 567-576
22. Varadi K, Rottensteiner H, Vejda S et al. Species-dependent variability of ADAMTS13-mediated proteolysis of human recombinant von Willebrand factor. J Thromb Haemost 2009 7 (7) 1134-1142
23. Laje P, Shang D, Cao W et al. Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell-mediated gene therapy. Blood 2009 113 (10) 2172-2180
24. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use. ICH Harmonised Tripartite Guideline: Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals, S6. 1997. Available at: www.fda.gov/downloads/Downloads/Drugs/ GuidanceComplicaneRegulatoryInformation/Guidances/UCM074957.pdf Accessed June 17, 2010
25. Motto D G., Chauhan A K., Zhu G et al. Shigatoxin triggers thrombotic thrombocytopenic purpura in genetically susceptible ADAMTS13-deficient mice. J Clin Invest 2005 115 (10) 2752-2761
26. Denis C, Methia N, Frenette P S. et al. A mouse model of severe von Willebrand disease: defects in hemostasis and thrombosis. Proc Natl Acad Sci U S A 1998 95 (16) 9524-9529