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Volumn 13, Issue 1, 2015, Pages 72-81

Expanding the ortholog approach for hemophilia treatment complicated by factor VIII inhibitors

Author keywords

Antibody specificity; Cross reactions; Factor VIII; Hemophilia A, acquired; Hemophilia A, congenital

Indexed keywords

ANTIGEN; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; BLOOD CLOTTING FACTOR 8 INHIBITOR; EPITOPE; IMMUNOGLOBULIN; PROCOAGULANT; RECOMBINANT BLOOD CLOTTING FACTOR 8; AUTOANTIBODY; BIOLOGICAL MARKER; COAGULATING AGENT; F8 PROTEIN, HUMAN; RECOMBINANT PROTEIN;

EID: 84920971123     PISSN: 15387933     EISSN: 15387836     Source Type: Journal    
DOI: 10.1111/jth.12755     Document Type: Article
Times cited : (11)

References (43)
  • 2
    • 33847411624 scopus 로고    scopus 로고
    • Acquired hemophilia A in the United Kingdom: a 2-year national surveillance study by the United Kingdom Haemophilia Centre Doctors' Organisation
    • Collins PW, Hirsch S, Baglin TP, Dolan G, Hanley J, Makris M, Keeling DM, Liesner R, Brown SA, Hay CR; UK Haemophilia Centre Doctors' Organisation. Acquired hemophilia A in the United Kingdom: a 2-year national surveillance study by the United Kingdom Haemophilia Centre Doctors' Organisation. Blood 2007; 109: 1870-7.
    • (2007) Blood , vol.109 , pp. 1870-1877
    • Collins, P.W.1    Hirsch, S.2    Baglin, T.P.3    Dolan, G.4    Hanley, J.5    Makris, M.6    Keeling, D.M.7    Liesner, R.8    Brown, S.A.9    Hay, C.R.10
  • 3
    • 0027473752 scopus 로고
    • Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group
    • Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group. N Engl J Med 1993; 328: 453-9.
    • (1993) N Engl J Med , vol.328 , pp. 453-459
    • Lusher, J.M.1    Arkin, S.2    Abildgaard, C.F.3    Schwartz, R.S.4
  • 4
    • 0028266130 scopus 로고
    • A multicenter study of recombinant factor VIII (recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. The Recombinate Study Group
    • Bray GL, Gomperts ED, Courter S, Gruppo R, Gordon EM, Manco-Johnson M, Shapiro A, Scheibel E, White G III, Lee M. A multicenter study of recombinant factor VIII (recombinate): safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. The Recombinate Study Group. Blood 1994; 83: 2428-35.
    • (1994) Blood , vol.83 , pp. 2428-2435
    • Bray, G.L.1    Gomperts, E.D.2    Courter, S.3    Gruppo, R.4    Gordon, E.M.5    Manco-Johnson, M.6    Shapiro, A.7    Scheibel, E.8    White, G.9    Lee, M.10
  • 5
    • 0037276262 scopus 로고    scopus 로고
    • The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A
    • Lusher JM, Lee CA, Kessler CM, Bedrosian CL. The safety and efficacy of B-domain deleted recombinant factor VIII concentrate in patients with severe haemophilia A. Haemophilia 2003; 9: 38-49.
    • (2003) Haemophilia , vol.9 , pp. 38-49
    • Lusher, J.M.1    Lee, C.A.2    Kessler, C.M.3    Bedrosian, C.L.4
  • 7
    • 0030926974 scopus 로고    scopus 로고
    • The inhibitor antibody response is more complex in hemophilia A patients than in most nonhemophiliacs with factor VIII autoantibodies. Recombinate and Kogenate Study Groups
    • Prescott R, Nakai H, Saenko EL, Scharrer I, Nilsson IM, Humphries JE, Hurst D, Bray G, Scandella D. The inhibitor antibody response is more complex in hemophilia A patients than in most nonhemophiliacs with factor VIII autoantibodies. Recombinate and Kogenate Study Groups. Blood 1997; 89: 3663-71.
    • (1997) Blood , vol.89 , pp. 3663-3671
    • Prescott, R.1    Nakai, H.2    Saenko, E.L.3    Scharrer, I.4    Nilsson, I.M.5    Humphries, J.E.6    Hurst, D.7    Bray, G.8    Scandella, D.9
  • 8
    • 0029030435 scopus 로고
    • Residues 484-508 contain a major determinant of the inhibitory epitope in the A2 domain of human factor VIII
    • 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.
    • (1995) J Biol Chem , vol.270 , pp. 14505-14509
    • Healey, J.F.1    Lubin, I.M.2    Nakai, H.3    Saenko, E.L.4    Hoyer, L.W.5    Scandella, D.6    Lollar, P.7
  • 9
    • 0034651022 scopus 로고    scopus 로고
    • Reduction of the antigenicity of factor VIII toward complex inhibitory antibody plasmas using multiply-substituted hybrid human/porcine factor VIII molecules
    • Barrow RT, Healey JF, Gailani D, Scandella D, Lollar P. Reduction of the antigenicity of factor VIII toward complex inhibitory antibody plasmas using multiply-substituted hybrid human/porcine factor VIII molecules. Blood 2000; 95: 564-8.
    • (2000) Blood , vol.95 , pp. 564-568
    • Barrow, R.T.1    Healey, J.F.2    Gailani, D.3    Scandella, D.4    Lollar, P.5
  • 10
    • 39649102400 scopus 로고    scopus 로고
    • Antihuman factor VIII C2 domain antibodies in hemophilia A mice recognize a functionally complex continuous spectrum of epitopes dominated by inhibitors of factor VIII activation
    • Meeks SL, Healey JF, Parker ET, Barrow RT, Lollar P. Antihuman factor VIII C2 domain antibodies in hemophilia A mice recognize a functionally complex continuous spectrum of epitopes dominated by inhibitors of factor VIII activation. Blood 2007; 110: 4234-42.
    • (2007) Blood , vol.110 , pp. 4234-4242
    • Meeks, S.L.1    Healey, J.F.2    Parker, E.T.3    Barrow, R.T.4    Lollar, P.5
  • 11
    • 84878218869 scopus 로고    scopus 로고
    • The diversity of the immune response to the A2 domain of human factor VIII
    • Markovitz RC, Healey JF, Parker ET, Meeks SL, Lollar P. The diversity of the immune response to the A2 domain of human factor VIII. Blood 2013; 121: 2785-95.
    • (2013) Blood , vol.121 , pp. 2785-2795
    • Markovitz, R.C.1    Healey, J.F.2    Parker, E.T.3    Meeks, S.L.4    Lollar, P.5
  • 13
    • 84891332314 scopus 로고    scopus 로고
    • Structure of the factor VIII C2 domain in a ternary complex with 2 inhibitor antibodies reveals classical and nonclassical epitopes
    • Walter JD, Werther RA, Brison CM, Cragerud RK, Healey JF, Meeks SL, Lollar P, Spiegel PC Jr. Structure of the factor VIII C2 domain in a ternary complex with 2 inhibitor antibodies reveals classical and nonclassical epitopes. Blood 2013; 122: 4270-8.
    • (2013) Blood , vol.122 , pp. 4270-4278
    • Walter, J.D.1    Werther, R.A.2    Brison, C.M.3    Cragerud, R.K.4    Healey, J.F.5    Meeks, S.L.6    Lollar, P.7    Spiegel Jr, P.C.8
  • 14
    • 84875984071 scopus 로고    scopus 로고
    • Characterization and solution structure of the factor VIII C2 domain in a ternary complex with classical and non-classical inhibitor antibodies
    • Walter JD, Werther RA, Polozova MS, Pohlman J, Healey JF, Meeks SL, Lollar P, Spiegel PC Jr. Characterization and solution structure of the factor VIII C2 domain in a ternary complex with classical and non-classical inhibitor antibodies. J Biol Chem 2013; 288: 9905-14.
    • (2013) J Biol Chem , vol.288 , pp. 9905-9914
    • Walter, J.D.1    Werther, R.A.2    Polozova, M.S.3    Pohlman, J.4    Healey, J.F.5    Meeks, S.L.6    Lollar, P.7    Spiegel Jr, P.C.8
  • 15
    • 84889646833 scopus 로고    scopus 로고
    • Epitope mapping of inhibitory antibodies targeting the C2 domain of coagulation factor VIII by hydrogen-deuterium exchange mass spectrometry
    • Sevy AM, Healey JF, Deng W, Spiegel PC, Meeks SL, Li R. Epitope mapping of inhibitory antibodies targeting the C2 domain of coagulation factor VIII by hydrogen-deuterium exchange mass spectrometry. J Thromb Haemost 2013; 11: 2128-36.
    • (2013) J Thromb Haemost , vol.11 , pp. 2128-2136
    • Sevy, A.M.1    Healey, J.F.2    Deng, W.3    Spiegel, P.C.4    Meeks, S.L.5    Li, R.6
  • 16
    • 84856866914 scopus 로고    scopus 로고
    • The principal results of the International Immune Tolerance Study: a randomized dose comparison
    • Hay CR, DiMichele DM, International Immune Tolerance Study. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood 2012; 119: 1335-44.
    • (2012) Blood , vol.119 , pp. 1335-1344
    • Hay, C.R.1    DiMichele, D.M.2
  • 18
    • 1342282981 scopus 로고    scopus 로고
    • Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion
    • Doering CB, Healey JF, Parker ET, Barrow RT, Lollar P. Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion. J Biol Chem 2004; 279: 6546-52.
    • (2004) J Biol Chem , vol.279 , pp. 6546-6552
    • Doering, C.B.1    Healey, J.F.2    Parker, E.T.3    Barrow, R.T.4    Lollar, P.5
  • 19
    • 33646585786 scopus 로고    scopus 로고
    • High-level expression of porcine factor VIII from genetically modified bone marrow-derived stem cells
    • Gangadharan B, Parker ET, Ide LM, Spencer HT, Doering CB. High-level expression of porcine factor VIII from genetically modified bone marrow-derived stem cells. Blood 2006; 107: 3859-64.
    • (2006) Blood , vol.107 , pp. 3859-3864
    • Gangadharan, B.1    Parker, E.T.2    Ide, L.M.3    Spencer, H.T.4    Doering, C.B.5
  • 20
    • 35349015845 scopus 로고    scopus 로고
    • Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens
    • Ide LM, Gangadharan B, Chiang KY, Doering CB, Spencer HT. Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood 2007; 110: 2855-63.
    • (2007) Blood , vol.110 , pp. 2855-2863
    • Ide, L.M.1    Gangadharan, B.2    Chiang, K.Y.3    Doering, C.B.4    Spencer, H.T.5
  • 21
    • 34249737517 scopus 로고    scopus 로고
    • Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity
    • Doering CB, Gangadharan B, Dukart HZ, Spencer HT. Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity. Mol Ther 2007; 15: 1093-9.
    • (2007) Mol Ther , vol.15 , pp. 1093-1099
    • Doering, C.B.1    Gangadharan, B.2    Dukart, H.Z.3    Spencer, H.T.4
  • 26
    • 0029909770 scopus 로고    scopus 로고
    • The cDNA and derived amino acid sequence of porcine factor VIII
    • Healey JF, Lubin IM, Lollar P. The cDNA and derived amino acid sequence of porcine factor VIII. Blood 1996; 88: 4209-14.
    • (1996) Blood , vol.88 , pp. 4209-4214
    • Healey, J.F.1    Lubin, I.M.2    Lollar, P.3
  • 28
    • 51649102381 scopus 로고    scopus 로고
    • Nonclassical anti-C2 domain antibodies are present in patients with factor VIII inhibitors
    • Meeks SL, Healey JF, Parker ET, Barrow RT, Lollar P. Nonclassical anti-C2 domain antibodies are present in patients with factor VIII inhibitors. Blood 2008; 112: 1151-3.
    • (2008) Blood , vol.112 , pp. 1151-1153
    • Meeks, S.L.1    Healey, J.F.2    Parker, E.T.3    Barrow, R.T.4    Lollar, P.5
  • 29
    • 33748757657 scopus 로고    scopus 로고
    • Neutralization of antifactor VIII inhibitors by recombinant porcine factor VIII
    • Barrow RT, Lollar P. Neutralization of antifactor VIII inhibitors by recombinant porcine factor VIII. J Thromb Haemost 2006; 4: 2223-9.
    • (2006) J Thromb Haemost , vol.4 , pp. 2223-2229
    • Barrow, R.T.1    Lollar, P.2
  • 32
    • 33751017726 scopus 로고    scopus 로고
    • The epidemiology of factor VIII inhibitors
    • discussion 8-9
    • Hay CR. The epidemiology of factor VIII inhibitors. Haemophilia 2006; 6: 23-8; discussion 8-9.
    • (2006) Haemophilia , vol.6 , pp. 23-28
    • Hay, C.R.1
  • 33
    • 0035158301 scopus 로고    scopus 로고
    • Human immune response to recombinant human proteins
    • Porter S. Human immune response to recombinant human proteins. J Pharm Sci 2001; 90: 1-11.
    • (2001) J Pharm Sci , vol.90 , pp. 1-11
    • Porter, S.1
  • 34
    • 63049117124 scopus 로고    scopus 로고
    • Non-classical anti-factor VIII C2 domain antibodies are pathogenic in a murine in vivo bleeding model
    • Meeks SL, Healey JF, Parker ET, Barrow RT, Lollar P. Non-classical anti-factor VIII C2 domain antibodies are pathogenic in a murine in vivo bleeding model. J Thromb Haemost 2009; 7: 658-64.
    • (2009) J Thromb Haemost , vol.7 , pp. 658-664
    • Meeks, S.L.1    Healey, J.F.2    Parker, E.T.3    Barrow, R.T.4    Lollar, P.5
  • 35
    • 0011936231 scopus 로고
    • The purification of antihaemophilic globulin from animal plasma
    • Bidwell E. The purification of antihaemophilic globulin from animal plasma. Br J Haematol 1955; 1: 386-9.
    • (1955) Br J Haematol , vol.1 , pp. 386-389
    • Bidwell, E.1
  • 38
    • 0021368359 scopus 로고
    • Clinical experience with polyelectrolyte-fractionated porcine factor VIII concentrate in the treatment of hemophiliacs with antibodies to factor VIII
    • Kernoff PB, Thomas ND, Lilley PA, Matthews KB, Goldman E, Tuddenham EG. Clinical experience with polyelectrolyte-fractionated porcine factor VIII concentrate in the treatment of hemophiliacs with antibodies to factor VIII. Blood 1984; 63: 31-41.
    • (1984) Blood , vol.63 , pp. 31-41
    • Kernoff, P.B.1    Thomas, N.D.2    Lilley, P.A.3    Matthews, K.B.4    Goldman, E.5    Tuddenham, E.G.6
  • 39
    • 0033694552 scopus 로고    scopus 로고
    • Mapping factor VIII inhibitor epitopes using hybrid human/porcine factor VIII molecules
    • Lollar P. Mapping factor VIII inhibitor epitopes using hybrid human/porcine factor VIII molecules. Haematologica 2000; 85: 26-8.
    • (2000) Haematologica , vol.85 , pp. 26-28
    • Lollar, P.1
  • 40
    • 0032533220 scopus 로고    scopus 로고
    • Residues Glu2181-Val2243 contain a major determinant of the inhibitory epitope in the C2 domain of human factor VIII
    • Healey JF, Barrow RT, Tamim HM, Lubin IM, Shima M, Scandella D, Lollar P. Residues Glu2181-Val2243 contain a major determinant of the inhibitory epitope in the C2 domain of human factor VIII. Blood 1998; 92: 3701-9.
    • (1998) Blood , vol.92 , pp. 3701-3709
    • Healey, J.F.1    Barrow, R.T.2    Tamim, H.M.3    Lubin, I.M.4    Shima, M.5    Scandella, D.6    Lollar, P.7
  • 41
    • 0030858423 scopus 로고    scopus 로고
    • Analysis of factor VIII inhibitors using hybrid human/porcine factor VIII
    • Lollar P. Analysis of factor VIII inhibitors using hybrid human/porcine factor VIII. Thromb Haemost 1997; 78: 647-51.
    • (1997) Thromb Haemost , vol.78 , pp. 647-651
    • Lollar, P.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.