Thrombosis and Haemostasis, Table of Contents Thromb Haemost 1997; 78(01): 641-646DOI: 10.1055/s-0038-1657604 Factor VIII and factor IX inhibitors Schattauer GmbH Stuttgart Factor VIII Inhibitors Jean Guy G Gilles Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium , Marc G Jacquemin Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium , Jean-Marie R Saint-Remy Center for Molecular and Vascular Biology, University of Leuven, Leuven, Belgium › Author Affiliations Recommend Article Abstract PDF Download Buy Article PDF (1172 kb) References References 1 Rizza CR, Biggs R. The treatment of patients who have factor VIII antibodies. Br J Haematol 1973; 24: 65 2 Kasper CK, Aledort LM, Aronson D, Counts R, Edson JR, van Eys J, Fratantoni J, Green D, Hampton J, Hilgartner M, Levine P, Lazerson J, McMillan C, Penner J, Shapiro S, Shulman NR. Proceedings: a more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 1975; 34: 612 3 Verbruggen B, Novakova I, Wessels H, Boezman J, Van den Berg M, Mauser-Bunschoten E. The Nijmegen modification of the Bethesda assay for FVIII: C inhibitors: improved specificity and reliability. Thromb Haemost 1995; 73: 247-251 4 Svendsen L, Brogli M, Lindeberg G, Stocker K. Differentiation of thrombin-and factor Xa-related amidolytic activity in plasma by means of synthetic thrombin inhibitor. Thromb Res 1984; 34: 457-462 5 Gilles JG, Arnout J, Vermylen J, Saint-Remy JMR. Anti-factor VIII antibodies of hemophiliac patients are frequently directed towards nonfunctional determinants and do not exhibit isotypic restriction. Blood 1993; 82: 2452-2461 6 Scandella D, Mattingly M, de Graaf S, Fulcher CA. Localization of epitopes for human factor VIII inhibitor antibodies by immunoblotting and antibody neutralization. Blood 1989; 74: 1618-1626 7 Briët E, Rosendaal FR, Kreuz W, Rasi V, Peerlinck K, Vermylen J, Ljung R, Rocino A. et al High titer inhibitors in severe haemophilia A. A meta-analysis based on eight long-term follow-up studies concerning inhibitors associated with crude or intermediate purity factor VIII products. Thromb Haemost 1994; 72: 162-164 8 Gilles JG, Saint-Remy JMR. Healthy subjects produce both antifactor VIII and specific anti-idiotypic antibodies. J Clin Invest 1994; 94: 1496-1505 9 Bray GL, Gomperts ED, Courter S, Gruppo R, Gordon E, Manco-Johanson M, Shapiro A, Scheibel E. et al. A multi-center study of recombinant factor VIII (Recombinate): safety, efficacy and inhibitor risk in previously untreated patients with hemophilia A. Blood 1994; 83: 2428-2435 10 Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Kogenate previously untreated patient study group. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. N Engl J Med 1993; 328: 453-459 11 Peerlinck K, Rosendaal F, Vermylen J. Incidence of inhibitor development in a group of young hemophilia A patients treated exclusively with lyophilized cryoprecipitate. Blood 1993; 81: 3332-3335 12 Hoyer LW. Immunochemical properties of factor VIII and factor IX inhibitors. Blood Coag Fibrinolysis 1991; 2: 11S-15S 13 Tuddenham EGD, Schwaab R, Seehafer J, Millar DS, Gitschier J, Higuchi M, Bidichandani S, Conor JM. et al. Haemophilia A: database of nucleotide substitutions, deletions, insertions and rearrangements of the factor VIII gene, second edition. Nucl Acids Res 1994; 22: 3511-3533 14 Antonarakis SE, Rossiter JP, Young M, Horst J, de Moerloose P, Sommer SS, Ketterling RP, Kazazian HH. et al. Factor VIII gene inversions in severe hemophilia A: results of an international consortium study. Blood 1995; 86: 2206-2212 15 Hay CRM, Oilier W, Pepper L, Cumming A, Keeney S, Goodeve AC, Peake JR. HLA class II profile: a determinant of factor VIII inhibitor development in severe haemophilia A. Blood 1996; 88 (Suppl. 10) 329a 16 Peerlinck K, Arnout J, Gilles JG, Saint-Remy JMR, Vermylen J. A higher than expected incidence of factor VIII inhibitors in multitransfused haemophilia A patients treated with an intermediate purity pasteurized factor VIII concentrate. Thromb Haemost 1993; 69: 115-118 17 Peerlinck K, Arnout J, Di Giambattista M, Gilles JG, Saint-Remy JMR, Vermylen J. Factor VIII inhibitors in previously treated haemophilia A patients with double virus-inactivated plasma derived factor VIII concentrate. Thromb Haemost 1997; 77: 80-86 18 Gilles JG, Saint-Remy JMR. Strategy for pre-clinical evaluafactor VIII concentrates. Blood Coagulation and Fibrinolysis 1995; 06 (Suppl. 02) S58-S61 19 Fulcher C, De Graaf Mahoney S, Zimmerman T. Factor VIII inhibitor IgG subclass and FVIII polypeptide specificity determined by immunoblotting. Blood 1987; 69: 1475-1480 20 Fulcher C, de Graaf Mahoney S, Roberts J, Kasper C, Zimmerman T. Localisation of human factor VIII inhibitor epitopes to two polypeptide fragments. Proc Natl Acad Sci 1985; 82: 7728-7732 21 Scandella DD, Graaf Mahoney S, Mattingly M, Roeder D, Timmons L, Fulcher C. Epitope mapping of human factor VIII inhibitor antibodies by deletion analysis of FVIII fragments expressed in Escherichia Coli. Proc Natl Acad Sci USA 1988; 85: 6152-6157 22 Gawryl M, Hoyer L. Inactivation of factor VIII coagulant activity by two different types of human antibodies. Blood 1982; 60: 1103-1108 23 Hultin M, Nemerson Y. Activation of factor X by factor IXa and VIII; a specific assay for factor IXa in the presence of thrombin-activated factor VIII. Blood 1978; 52: 928-934 24 Van Dieijen G, Tans G, Rosing J, Hemker H. The role of phospholipid and factor Villa in the activation of bovine factor X. J Biol Chem 1981; 256: 3433-3436 25 Hamer RJ, Koedam JA, Beeser-Visser NH, Bertina RM, Van Mourik JA, Sixma JJ. Factor VIII binds to von Willebrand factor via its Mr 80,000 light chain. Eur J Biochem 1987; 166: 37-43 26 Saenko EL, Shima M, Rajalakshmi KJ, Scandella D. A role for the C2 domain of factor VIII in binding to von Willebrand factor. J Biol Chem 1994; 269: 11601-11605 27 Saenko EL, Scandella D. A mechanism for inhibition of factor VIII binding to phospholipid by von Willebrand factor. J Biol Chem 1995; 270: 13826-13833 28 Foster PA, Fulcher CA, Houghten RA, Zimmerman TS. Synthetic factor VIII peptides with amino acid sequences contained within the C2 domain of factor VIII inhibit factor VIII binding to phosphatidylserine. Blood 1990; 75: 1999-2004 29 Foster PA, Fulcher CA, Houghten RA, Zimmerman TS. An immunogenic region within residues Vall670-Glul684 of the factor VIII light chain induces antibodies which inhibit binding of factor VIII to von Willebrand factor. J Biol Chem 1988; 263: 5230-5234 30 Leyte A, Verbeet MP, Brodniewicz-Proba T, Van Mourik JA, Mertens K. The interaction between human blood coagulation factor VIII and von Willebrand factor. Characterization of a high-affinity binding site on factor VIII. Biochem J 1989; 257: 679-683 31 Shima M, Yoshioka A, Nakai H, Tanaka I, Sawamoto Y, Kamisue S, Terada S, Fukui H. Epitope localization of monoclonal antibodies against factor VIII light chain which inhibit complex formation by factor VIII with von Willebrand factor. Int J Hematol 1991; 54: 515-522 32 Saenko E, Shima M, Gilbert G, Scandella D. Slowed release of thrombin-cleaved factor VIII from von Willebrand factor by a monoclonal and a human antibody is a novel mechanism for factor VIII inhibition. J. Biol Chem 1996; 271: 27424-27431 33 Nilsson I, Berntorp E, Zettervall O, Dahlbäck B. Noncoagulation inhibitory factor VIII antibodies after induction of tolerance to factor VIII in hemophilia A patients. Blood 1990; 75: 378-383 34 Kesler C. An introduction to factor VIII inhibitors: the detection and quantitation. Am J Med 1991; 91 (Suppl. 05) 15-19 35 Barnhart M, Chen S, Lusher J. DDAVP-does the drug have a direct effect on the vessel wall ? Thromb Res. 1983; 31: 239 36 Kesteven P, Holland L, Lawarie A, Savidge G. Inhibitor to factor VIII in mild haemophilia. Thromb Haemost 1984; 52 (01) 50-52 37 Lusher J, Blatt P, Penner J, Aledort L, Levine P, White A, Warrier I, Whitehurst L. Autoplex versus Proplex: a controlled doubleblind study of effectiveness in acute hemarthroses in hemophiliacs with inhibitors to FVIII. Blood 1983; 62: 1135-1139 38 Sjamsoedin L, Heijnen L, Mauser-Bunschoten EP. The effect of activated prothrombin complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII: a double-blind clinical trial. N Engl J Med 1981; 305: 717-721 39 Hedner U, Glazer S, Falch J. Recombinant activated factor VII in the treatment of bleeding episodes in patients with inherited and acquired bleeding disorders. Trans Med Rev 1993; 7: 78-83 40 Hay CRM, Lozier JN, Lee CA. et al. Safety profile of porcine factor VIII and its use as hospital and home-therapy for patients with haemophilia A and inhibitors: the results of an international survey. Thromb Haemost 1996; 75: 25-29 41 Brackmann H, Etzel F, Hoffmann P, Egli H. The successful treatment of acquired inhibitors against factor VIII. Thromb Haemost 1977; 38: 269 42 Brackmann H. Sixteen years experience with the immunotolerance induction in hemophilia patients at the Bonn Hemophilia Center. XX Int Cong World Fed Hemophilia, Athens 1992 (abstract 12). 43 Van Leeuwen E, Mauser-Bunschoten E, Van Dijken P, Kok A, Sjamsoendin-Visser E, Sixma J. Disappearance of factor VIII: C antibodies in patients with haemophilia A upon frequent administration of FVIII in intermediate or low dose. Br J Haematol 1986; 64: 291 44 Mauser-Bunschoten R, Nilsson I, Kasper C. Immune tolerance, a 1990 approach. In: Hemophilia and von Willebrand’s disease in the 1990s. Lusher JM, Kessler CM. eds Elsevier Science; New York: 1991. pp. 265 45 Mariani G, Ghirardini A, Belloco R. Immune tolerance in hemophilia-principal results from the international registry. Thromb Haemost 1994; 72: 155-158 46 Nilsson I, Sundqvist S, Freiburghaus C. Extracorporeal protein A-sepharose and specific affinity chromatography for removal of antibodies. Prog Clin Biol Res 1984; 150: 225-229 47 Nilsson I, Berntorp E, Zettervall O. Induction of immune tolerance in patients with hemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII. N Engl J Med 1988; 318: 947 48 Sultan Y, Kazatchikine M, Maisonneuve P, Nydegger U. Anti-idiotypic suppression of auto-antibodies to factor VIII (antihemophilic factor) by high dose intravenous gammaglobulin. Lancet 1984; 2: 765-768 49 Rossi F, Sultan Y, Kazatchkine M. Anti-idiotypes against autoantibodies and alloantibodies to VIII: C (anti-haemophilic factor) are present in therapeutic polyspecific normal immunoglobulins. Clin Exp Immunol 1988; 74: 311 50 Desqueper B, Gilles JG, Jacquemin M, DI Giambattista M, Laub R, Saint-Remy JMR. Cohn’s fractions as a source of anti-factor VIII and corresponding anti-idiotypic antibodies for possible therapeutic purposes of haemophilia A patients with inhibitors. Thromb Haemost 1995; 73 Abstract 426: 1011 51 Gilles JGG, Desqueper B, Lenk H, Vermylen J, Saint-Remy JMR. Neutralising anti-idiotypic antibodies to factor VIII inhibitors following desensitization in patients with hemophilia A. J Clin Invest 1996; 97: 1382-1388 52 Lollar P, Parker ET, Curtis JE, Helgerson SL, Hoyer LW, Scott ME, Scandella D. Inhibition of human factor Villa by anti-A2 subunit antibodies. J Clin Invest 1994; 93: 2497-2504
