Models for assessing immunogenicity and efficacy of new therapeutics for the treatment of haemophilia

Haemophilia

Volumn 18, Issue SUPPL.4, 2012, Pages 43-47

  Saint Remy, Jean Marie ^a   Reipert, Birgit M ^b   Monroe, Dougald M ^c  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b BAXTER BIOSCIENCE   (Austria)

^c UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

Antigen presentation; Factor VIII; Factor VIII bypassing activity; Factor VIII inhibitors; Haemophilia A; Immune tolerance induction

Indexed keywords

BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 CONCENTRATE; BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 CONCENTRATE; MAJOR HISTOCOMPATIBILITY ANTIGEN CLASS 2;

ANTIGEN PRESENTATION; ARTICLE; BLEEDING; DRUG EFFICACY; HEMOPHILIA; HEMOPHILIA; CONCENTRATE; HEMOSTASIS; IMMUNE RESPONSE; IMMUNOGENICITY; IMMUNOLOGICAL TOLERANCE; INNATE IMMUNITY; NONHUMAN; PRIORITY JOURNAL; RISK ASSESSMENT;

ANIMALS; ANTIGENS, CD; BLOOD COAGULATION FACTOR INHIBITORS; COAGULANTS; DISEASE MODELS, ANIMAL; DOGS; FACTOR VIII; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; MICE;

EID: 84862863776     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2012.02828.x     Document Type: Article

References (26)

1. Key NS. Inhibitors in congenital coagulation disorders. Br J Haematol 2004; 127: 379-91.
2. Zhang A-H, Skupsky J, Scott DW. Effect of B-cell depletion using anti-CD20 therapy on inhibitory antibody formation to human FVIII in hemophilia A mice. Blood 2011; 117: 2223-6.
3. Billmann-Born S, Lipinski S, Böck J, Till A, Rosenstiel P, Schreiber S. The complex interplay of NOD-like receptors and the autophagy machinery in the pathophysiology of Crohn disease. Eur J Cell Biol 2011; 90: 593-602.
4. Pautard B, D'Oiron R, Li Thiao Te V et al. Successful immune tolerance induction by FVIII in hemophilia A patients with inhibitor may occur without deletion of FVIII-specific T cells. J Thromb Haemost 2011; 9: 1163-70.
5. Andersson IE, Batsalova T, Dzhambazov B et al. Oxazole-modified glycopeptides that target arthritis-associated class II MHC Aq and DR4 proteins. Org Biomol Chem 2010; 8: 2931-40.
6. van Helden PM, Unterthurner S, Hermann C et al. Maintenance and break of immune tolerance against human factor VIII in a new transgenic hemophilic mouse model. Blood 2011; 118: 3698-707.
7. Lenk C, Unterthurner S, Antoine G et al. Maintenance and break of immune tolerance in a new mouse model that is immunologically tolerant to native human factor VIIa. J Thromb Haemost 2011; 9: 595.
8. Reipert B, Arruda V, Lillicrap D. Animal models of inhibitors. Haemophilia 2010; 16: 47-53.
9. Reipert BM, Steinitz KN, van Helden PM et al. Opportunities and limitations of mouse models humanized for HLA class II antigens. J Thromb Haemost 2009; 7: 92-7.
10. von Boehmer H. Selection of the T-cell repertoire: receptor-controlled checkpoints in T-cell development. Adv Immunol 2004; 84: 201-38.
11. Rajewsky K. Clonal selection and learning in the antibody system. Nature 1996; 381: 751-8.
12. Vinuesa CG, Tangye SG, Moser B, Mackay CR. Follicular B helper T cells in antibody responses and autoimmunity. Nat Rev Immunol 2005; 5: 853-65.
13. Sinigaglia F, Hammer J. Defining rules for the peptide-MHC class II interaction. Curr Opin Immunol 1994; 6: 52-6.
14. Suri A, Lovitch SB, Unanue ER. The wide diversity and complexity of peptides bound to class II MHC molecules. Curr Opin Immunol 2006; 18: 70-7.
15. Banchereau J, Steinman RM. Dendritic cells and the control of immunity. Nature 1998; 392: 245-52.
16. van Haren SD, Herczenik E, ten Brinke A, Mertens K, Voorberg J, Meijer AB. HLA-DR-presented peptide repertoires derived from human monocyte-derived dendritic cells pulsed with blood coagulation factor VIII. Mol Cell Proteomics 2011; 10: M110.
17. Giles AR, Tinlin S, Greenwood R. A canine model of hemophilic (factor VIII:C deficiency) bleeding. Blood 1982; 60: 727-30.
18. Brinkhous KM, Sandberg H, Garris JB et al. Purified human factor VIII procoagulant protein: comparative hemostatic response after infusions into hemophilic and von Willebrand disease dogs. Proc Natl Acad Sci USA 1985; 82: 8752-6.
19. Kung SH, Hagstrom JN, Cass D et al. Human factor IX corrects the bleeding diathesis of mice with hemophilia B. Blood 1998; 91: 784-90.
20. Buyue Y, Whinna HC, Sheehan JP. The heparin-binding exosite of factor IXa is a critical regulator of plasma thrombin generation and venous thrombosis. Blood 2008; 112: 3234-41.
21. Møller F, Tranholm M. A ferric chloride induced arterial injury model used as haemostatic effect model. Haemophilia 2010; 16: e216-22.
22. Baumgartner B, Jaki T, Wolfsegger MJ et al. Optimization, refinement and reduction of murine in vivo experiments to assess therapeutic approaches for haemophilia A. Lab Anim 2010; 44: 211-7.
23. Margaritis P, Roy E, Aljamali MN et al. Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood 2009; 113: 3682-9.
24. McDonald A, Hoffman M, Hedner U, Roberts HR, Monroe DM. Restoring hemostatic thrombin generation at the time of cutaneous wounding does not normalize healing in hemophilia B. J Thromb Haemost 2007; 5: 1577-83.
25. Lafeber FPJG, Miossec P, Valentino LA. Physiopathology of haemophilic arthropathy. Haemophilia 2008; 14: 3-9.
26. Sun J, Hakobyan N, Valentino LA, Feldman BL, Samulski RJ, Monahan PE. Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX. Blood 2008; 112: 4532-41.