Analysis of Factor VIII Inhibitors Using Hybrid Human/Porcine Factor VIII

• References

• 1 Lusher JM, Arkin S, Abildgaard CF, Schwartz RSKogenate. 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: 453-459
• 2 Kasper CK, Aledort LM, Counts RB, Edson JR, Frantatoni J, Gree D, Hampton JW, Hilgartner MW, Lazerson J, Levine PH, McMillan CW, Pool JG, Shapiro SS, Shulman NR, van Eyes J. A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 1975; 34: 869-872
• 3 Vehar GA, Keyt B, Eaton D, Rodriguez H, O’Brien DP, Rotblat F, Oppermann H, Keck R, Wood WI, Harkins RN, Tuddenham EGD, Lawn RM, Capon DJ. Structure of human factor VIII. Nature 1984; 312: 337-342
• 4 Fulcher CA, Mahoney SD, Roberts JR, Kasper CK, Zimmerman TS. Localization of human factor FVIII inhibitor epitopes to two polypeptide fragments. Proc. Natl. Acad. Sci. USA 1985; 82: 7728-7732
• 5 Scandella D, Mahoney SD, Mattingly M, Roeder D, Timmons L, Fulcher CA. Epitope mapping of human factor VIII inhibitor antibodies by deletion analysis of factor VIII fragments expressed in Escherichia coli. Proc. Natl. Acad. Sci. USA 1988; 85: 6152-6156
• 6 Scandella D, Mattingly M, de Graaf S, Fulcher CA. Localization of epitopes for human factor VIII inhibitor antibodies by immuno-blotting and antibody neutralization. Blood 1989; 74: 1618-1626
• 7 Scandella D, Mattingly MPrescott R. A recombinant factor VIII A2 domain polypeptide quantitatively neutralizes human inhibitor antibodies which bind to A2. Blood 1993; 82: 1767-1775
• 8 Nakai H, Lollar P, Healey JF, Scandella D. Properties of affinity purified anti-factor VIII antibodies from patients with factor VIII inhibitors. Blood 1994; 84: 224a
• 9 Zhong D, Scandella D. Epitope of a hemophilia A inhibitor antibody overlaps the factor VIII binding site for factor IX. Blood 1996; 88: 324a (Abstr.)
• 10 Gilles JGG, Amout J, Vermylen J, Saint-Remy J-MR. Anti-factor VIII antibodies of hemophiliac patients are frequently directed towards nonfunctional determinants and do not exhibit isotypic restriction. Blood 1993; 82: 2452-2461
• 11 Lollar P, Parker ET, Curti JE, Helgerson SL, Hoyer LW, Scott ME, and Scandella D. Inhibition of human factor Villa by anti-A2 subunit antibodies. J Clin Invest 1994; 93: 2497-2504
• 12 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: 1978-1984
• 13 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 binding to von Willebrand factor and to phosphatidylserine. Thromb Haemostas 1993; 69: 240-246
• 14 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: 665-671
• 15 Lubin IM, Healey JF, Scandella D, Runge MS, Lollar P. Elimination of a major inhibitor epitope in factor VIII. J Biol Chem 1994; 269: 8639-8641
• 16 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: 14505-14509
• 17 Koshihara K, Qian J, Lollar P, Hoyer LW. Immunoblot crossreactivity of factor Vin inhibitors with porcine factor VIII. Blood 1995; 86: 2183-2190
• 18 Ware J, MacDonald MJ, Lo M, de Graaf S, Fulcher CA. Epitope mapping of human factor VIE inhibitor antibodies by site-directed mutagenesis of a factor VIII polypeptide. Blood Coagul Fibrinolysis 1992; 3: 703-716
• 19 Scandella D, Gilbert GE, Shima M, Nakai H, Eagleson C, Felch M, Prescott R, Rajalakshmi KJ, Hoyer LW, 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: 1811-1819
• 20 Shima M, Scandella D, Yoshioka A, Hanai 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: 240-246
• 21 Healey J F, Scandella D, Lollar P. Expression of a recombinant hybrid human/porcine factor VIE molecule with reduced reactivity toward anti-C2 domain inhibitory antibodies. Blood 1996; 88: 658a
• 22 Shulman NR, Hirschman RJ. Acquired hemophilia. Trans Assoc Am Physicians 1969; 82: 388-397
• 23 Cunningham BC, Jhurani P, Ng P, Wells JA. Receptor and antibody epitopes in human growth hormone identified by homolog-scanning mutagenesis. Science 1989; 243: 1330-1336
• 24 Toole JJ, Pittman DD, Orr EC, Murtha P, Wasley LC, Kaufman RJ. A large region (approximately equal to 95 kDa) of human factor VIE is dispensible for in vitro procoagulant activity. Proc Natl Acad Sci USA 1986; 83: 5939-5942
• 25 Healey JF, Lubin IM, Lollar P. The cDNA and derived amino acid sequence of porcine factor VIII. Blood 1996; 88: 4209-4214
• 26 Elder B, Lakich D, Gitschier J. Sequence of the murine factor VIII cDNA. Genomics 1993; 16: 374-379
• 27 Horton RM, Ho SN, Pullen JK, Hunt HD, Cai Z, Pease LR. Gene splicing by overlap extension. Methods Enzymol 1993; 217: 270-279
• 28 Laver WG, Air GM, Webster RG, Smith-Gill SJ. Epitopes on protein antigens: misconceptions and realities. Cell 1990; 61: 553-556
• 29 Jin L, Fendly BM, Wells JA. High resolution functional analysis of antibody-antigen interactions. J Mol Biol 1992; 226: 851-865
• 30 Ziatseva I, Zaitsev V, Card G, Moshkov K, Bax B, Ralph A, Lindley P. The X-ray structure of human serum ceruloplasmin at 3.1 Angstroms: nature of the copper centres. JBIC 1996; 1: 15-23
• 31 Pemberton S, Lindley P, Zaitsev V, Card G, Tuddenham EGD, Kemball-Cook G. A molecular model for the triplicated A domains of human factor VIII based on the crystal structure of human caeruloplasmin. Blood. in press
• 32 Brettler DM. Inhibitors in congenital haemophilia. Bailliere’s Clin Haematol 1996; 9: 319-329
• 33 Kessler CM. Drug utilization and economics of patient care in acquired hemophilia. In Kessler CM. (ed.) Acquired Hemophilia. Princeton: Excerpta Medica; 1995: 131-147
• 34 Hedner U, Glazer S, Falch J. Recombinant activated factor VII in the treatment of bleeding episodes in patients with inherited and acquired bleeding disorders. Transfusion Med Rev 1993; 7: 78-83
• 35 Hay C, Lozier JN. Porcine factor VIII therapy in patients with factor VIII inhibitors. In Aledort LM, Hoyer LW, Lusher JM, Reisner HM, White GCII. (eds.) Inhibitors to Coagulation Factors. New York: Plenum Press; 1995: 143-151
• 36 Kemoff PBA. Porcine factor VIII: preparation and use in treatment of inhibitor patients. In Hoyer LW. (ed) Factor VIII Inhibitors. New York: Alan R. Liss; 1984: 207-224
• 37 Lubin IM, Healey JF, Scandella D, Lollar P. Analysis of the human factor VIIIA2 inhibitor epitope by alanine scanning mutagenesis. Submitted for publication.
• 38 Tinlin S, Webster S, Giles AR. The development of homologous (canine/anti-canine) antibodies in dogs with hemophilia A (factor Vm) deficiency: a ten-year longitudinal study. Thromb Haemost 1993; 69: 21-24
• 39 Jarvis MA, Levin LG, Harrison JA, DePianto DJ, Suzuki CM, Ziaja CL, Brown JE, Jolly KW, Reisner HM, Abildgaard CF, Powell JS. Induction of human factor VIII inhibitors in rats by immunization with human recombinant factor VIII: a small animal model for humans with high responder inhibitor phenotype. Thromb Haemost 1996; 75: 318-325