The humoral response to human factor VIII in hemophilia A mice

Journal of Thrombosis and Haemostasis

Volumn 5, Issue 3, 2007, Pages 512-519

  Healey, J F ^a   Parker, E T ^a   Barrow, R T ^a   Langley, T J ^a   Church, W R ^b   Lollar, P ^a,c  

^a CHILDREN'S HEALTHCARE OF ATLANTA   (United States)

^b Green Mountain Antibodies   (United States)

^c Emory Children's Center   (United States)

Author keywords

Factor VIII; Hemophilia A; Immunogenicity; Inhibitor

Indexed keywords

ANTIGEN; BLOOD CLOTTING FACTOR 8; FC RECEPTOR; IMMUNOGLOBULIN G1; IMMUNOGLOBULIN G2A; COAGULATING AGENT; HYBRID PROTEIN; IMMUNOGLOBULIN CLASS; MONOCLONAL ANTIBODY;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY SPECIFICITY; ARTICLE; ENZYME LINKED IMMUNOSORBENT ASSAY; HEMOPHILIA A; HUMORAL IMMUNITY; HYBRIDOMA CELL CULTURE; IMMUNOGENICITY; MOUSE; NONHUMAN; POLYCLONAL ACTIVATION; PRIORITY JOURNAL; ANIMAL; ANTIBODY COMBINING SITE; ANTIBODY PRODUCTION; BINDING COMPETITION; BIOSYNTHESIS; BLOOD; C57BL MOUSE; CELL LINE; COMPARATIVE STUDY; CYTOLOGY; DISEASE MODEL; DRUG ADMINISTRATION; EPITOPE MAPPING; GENETIC TRANSFECTION; GENETICS; HAMSTER; HUMAN; HYBRIDOMA; IMMUNOLOGY; METABOLISM; METHODOLOGY; REPRODUCIBILITY; SPLEEN; SWINE;

ANIMALS; ANTIBODIES, MONOCLONAL; ANTIBODY FORMATION; ANTIBODY SPECIFICITY; BINDING SITES, ANTIBODY; BINDING, COMPETITIVE; CELL LINE; COAGULANTS; CRICETINAE; DISEASE MODELS, ANIMAL; DRUG ADMINISTRATION SCHEDULE; ENZYME-LINKED IMMUNOSORBENT ASSAY; EPITOPE MAPPING; FACTOR VIII; HEMOPHILIA A; HUMANS; HYBRIDOMAS; IMMUNOGLOBULIN ISOTYPES; MICE; MICE, INBRED C57BL; RECOMBINANT FUSION PROTEINS; REPRODUCIBILITY OF RESULTS; SPLEEN; SWINE; TRANSFECTION;

EID: 34247591462     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/j.1538-7836.2007.02373.x     Document Type: Article

References (35)

1. Lusher JM, Arkin S, Abildgaard CF, Schwartz RS and the Kogenate Previously Untreated Patient Study Group. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and the development of inhibitors. N Engl J Med 1993; 328: 453-9.
2. Ling M, Duncan EM, Rodgers SE, Street AM, Lloyd JV. Low detection rate of antibodies to non-functional epitopes on factor VIII in patients with hemophilia A and negative for inhibitors by Bethesda assay. J Thromb Haemost 2003; 1: 2548-53.
3. Hoyer LW, Gawryl MS, de la Fuente B. Immunochemical characterization of factor VIII inhibitors. In: Hoyer LW, ed. Factor VIII Inhibitors. New York: Alan R. Liss, 1984: 73-85.
4. Lollar P. Pathogenic antibodies to coagulation factors. I. Factor VIII and factor IX. J Thromb Haemost 2004; 2: 1082-95.
5. Prescott R, Nakai H, Saenko EL, Scharrer I, Nilsson IM, Humphries J, Hurst D, Bray G, Scandella D. The inhibitory antibody response is more complex in hemophilia A patients than in most nonhemophiliacs with fVIII autoantibodies. Blood 1997; 89: 3663-71.
6. Healey JF, Lubin IM, Nakai H, Saenko EL, Hoyer LW, Scandella D, Lollar P. Residues 484-508 contain a major determinant of the inhibitory epitope in the A2 domain of human factor VIII. J Biol Chem 1995; 270: 14505-9.
7. Lollar P, Parker ET, Curtis JE, Helgerson SL, Hoyer LW, Scott ME, Scandella D. Inhibition of human factor VIIIa by anti-A2 subunit antibodies. J Clin Invest 1994; 93: 2497-504.
8. Fay PJ, Scandella D. Human inhibitor antibodies specific for the factor VIII A2 domain disrupt the interaction between the subunit and factor IXa. J Biol Chem 1999; 274: 29826-30.
9. Arai M, Scandella D, Hoyer LW. Molecular basis of factor-VIII inhibition by human antibodies - antibodies that bind to the factor-VIII light chain prevent the interaction of factor-VIII with phospholipid. J Clin Invest 1989; 83: 1978-84.
10. Jacquemin MG, Desqueper BG, Benhida A, Vander Elst L, Hoylaerts MF, Bakkus M, Thielemans K, Arnout J, Peerlinck K, Gilles JG, Vermylen J, Saint-Remy JM. Mechanism and kinetics of factor VIII inactivation: Study with an IgG4 monoclonal antibody derived from a hemophilia A patient with inhibitor. Blood 1998; 92: 496-506.
11. Scandella D, Gilbert GE, Shima M, Nakai H, Eagleson C, Felch M, Prescott R, Rajalakshimi KJ, Hoyer JW, Saenko E. Some human inhibitor antibodies recognize a common epitope corresponding to C2 domain amino acids 2248-2312 which overlap a phospholipid binding site. Blood 1995; 86: 1811-9.
12. Nogami K, Shima M, Hosokawa K, Nagata M, Koide T, Saenko EL, Tanaka I, Shibata M, Yoshioka A. Factor VIII C2 domain contains the thrombin-binding site responsible for thrombin-catalyzed cleavage at Arg1689. J Biol Chem 2000; 275 (33): 25774-80.
13. Nogami K, Shima M, Hosokawa K, Suzuki T, Koide T, Saenko EL, Scandella D, Shibata M, Kamisue S, Tanaka I, Yoshioka A. Role of factor VIII C2 domain in factor VIII binding to factor Xa. J Biol Chem 1999; 274: 31000-7.
14. Saenko EL, Shima M, Gilbert GE, Scandella D. Slowed release of thrombin-cleaved factor VIII from von Willebrand factor by a monoclonal and human antibody is a novel mechanism for factor VIII inhibition. J Biol Chem 1996; 271: 27424-31.
15. Shima M, Scandella D, Yoshioka A, Nakai H, Tanaka I, Kamisue S, Terada S, Fukui H. A factor VIII neutralizing monoclonal antibody and a human inhibitor alloantibody recognizing epitopes in the C2 domain inhibit factor VIII binding to von Willebrand factor and to phosphatidylserine. Thromb Haemost 1993; 69: 240-6.
16. Doering CB, Healey JF, Parker ET, Barrow RT, Lollar P. High-level expression of recombinant porcine coagulation factor VIII. J Biol Chem 2002; 277: 38345-9.
17. Barrow RT, Healey JF, Gailani D, Scandella D, Lollar P. Reduction of the antigenicity of factor VIII toward complex inhibitory plasmas using multiply-substituted hybrid human/porcine factor VIII molecules. Blood 2000; 95: 557-61.
18. Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 1995; 10: 119-21.
19. Parker ET, Healey JF, Barrow RT, Craddock HN, Lollar P. Reduction of the inhibitory antibody response to human factor VIII in hemophilia A mice by mutagenesis of the A2 domain B cell epitope. Blood 2004; 104: 704-10.
20. Kohler G, Milstein C. Derivation of specific antibody-producing tissue culture and tumor lines by cell fusion. Eur J Immunol 1976; 6: 511-9.
21. Kasper CK, Aledort LM, Counts RB, Edson JR, Fratantoni J, Green D, Hampton JW, Hilgartner MW, Lazerson J, Levine PH, McMillan CW, Pool JG, Shapiro SS, Shulman NR, van Eys J. A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 1975; 34: 869-72.
22. Parker ET, Craddock HN, Barrow RT, Lollar P. Comparative immunogenicity of recombinant B domain-deleted porcine factor VIII and Hyate:C in hemophilia A mice pre-sensitized to human factor VIII. J Thromb Haemost 2004; 2: 605-11.
23. Doering CB, Parker ET, Healey JF, Craddock HN, Barrow RT, Lollar P. Expression and characterization of recombinant murine factor VIII. Thromb Haemost 2002; 88: 450-8.
24. Marascuilo LA, McSweeney M. Chi-square tests of homogeneity of proportions for qualitative variables in multiple independent samples In: Non-Parametric and Distribution-Free Methods for the Social Sciences. Monterey: Brooks/Cole, 1977: 127-63.
25. Qian J, Borovak M, Bi L, Kazazian HH Jr, Hoyer LW. Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A. Thromb Haemost 1999; 81: 240-4.
26. Reipert BM, Ahmad RU, Turecek PL, Schwarz HP. Characterization of antibodies induced by human factor VIII in a murine knockout model of hemophilia A. Thromb Haemost 2000; 84: 826-32.
27. Scandella D, Timmons L, Mattingly M, Trabold N, Hoyer LW. A soluble recombinant factor VIII fragment containing the A2 domain binds to some human anti-factor VIII antibodies that are not detected by immunoblotting. Thromb Haemostas 1992; 67: 665-71.
28. Janeway Jr CA. Approaching the asymptote? Evolution and revolution in immunology. Cold Spring Harb Symp Quant Biol 1989; 54 (Pt 1): 1-13.
29. Matzinger P. Tolerance, danger, and the extended family. Annu Rev Immunol 1994; 12: 991-1045.
30. Connelly S, Gardner JM, Lyons RM, McClelland A, Kaleko M. Sustained expression of therapeutic levels of human factor VIII in mice. Blood 1996; 87: 4671-7.
31. Parker ET, Lollar P. A quantitative measure of the efficacy of factor VIII in hemophilia A mice. Thromb Haemost 2003; 89: 480-5.
32. Nimmerjahn F, Ravetch JV. Divergent immunoglobulin G subclass activity through selective Fc receptor binding. Science 2005; 310: 1510-2.
33. Markine-Goriaynoff D, Coutelier JP. Increased efficacy of the immunoglobulin G2a subclass in antibody-mediated protection against lactate dehydrogenase-elevating virus-induced polioencephalomyelitis revealed with switch mutants. J Virol 2002; 76: 432-5.
34. Fossati-Jimack L, Ioan-Facsinay A, Reininger L, Chicheportiche Y, Watanabe N, Saito T, Hofhuis FM, Gessner JE, Schiller C, Schmidt RE, Honjo T, Verbeek JS, Izui S. Markedly different pathogenicity of four immunoglobulin G isotype-switch variants of an antierythrocyte autoantibody is based on their capacity to interact in vivo with the low-affinity Fcgamma receptor III. J Exp Med 2000; 191: 1293-302.
35. Jun JE, Goodnow CC. Scaffolding of antigen receptors for immunogenic versus tolerogenic signaling. Nat Immunol 2003; 4: 1057-64.