메뉴 건너뛰기




Volumn 6, Issue , 2015, Pages 131-141

Profile of efraloctocog alfa and its potential in the treatment of hemophilia A

Author keywords

Bioengineered products; Efraloctocog alfa; Factor VIII; Fc fusion; Hemophilia A

Indexed keywords

ALN AT3; BLOOD CLOTTING FACTOR 8; CONCIZUMAB; FC RECEPTOR; IMMUNOGLOBULIN G; N8 GP; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG;

EID: 84939120787     PISSN: None     EISSN: 11792736     Source Type: Journal    
DOI: None     Document Type: Article
Times cited : (13)

References (69)
  • 2
    • 0035822038 scopus 로고    scopus 로고
    • The hemophilias – from royal genes to gene therapy
    • Mannucci PM, Tuddenham EG. The hemophilias – from royal genes to gene therapy. N Engl J Med. 2001;344(23):1773–1779.
    • (2001) N Engl J Med , vol.344 , Issue.23 , pp. 1773-1779
    • Mannucci, P.M.1    Tuddenham, E.G.2
  • 3
    • 0035077234 scopus 로고    scopus 로고
    • Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis
    • White GC 2nd, Rosendaal F, Aledort LM, et al. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 2001;85(3):560.
    • (2001) Thromb Haemost , vol.85 , Issue.3 , pp. 560
    • White, G.C.1    Rosendaal, F.2    Aledort, L.M.3
  • 4
    • 0000553473 scopus 로고
    • High-potency antihaemophilic factor concentrate prepared from cryoglobulin precipitate
    • Pool JG, Gershgold EJ, Pappenhagen AR. High-potency antihaemophilic factor concentrate prepared from cryoglobulin precipitate. Nature. 1964;203:312.
    • (1964) Nature , vol.203 , pp. 312
    • Pool, J.G.1    Gershgold, E.J.2    Pappenhagen, A.R.3
  • 5
    • 33750049639 scopus 로고    scopus 로고
    • The tragic history of AIDS in the hemophilia population, 1982–1984
    • Evatt BL. The tragic history of AIDS in the hemophilia population, 1982–1984. J Thromb Haemost. 2006;4(11):2295–2301.
    • (2006) J Thromb Haemost , vol.4 , Issue.11 , pp. 2295-2301
    • Evatt, B.L.1
  • 6
    • 43949134531 scopus 로고    scopus 로고
    • Recombinant clotting factors
    • Pipe SW. Recombinant clotting factors. Thromb Haemost. 2008; 99(5):840–850.
    • (2008) Thromb Haemost , vol.99 , Issue.5 , pp. 840-850
    • Pipe, S.W.1
  • 7
    • 0026635406 scopus 로고
    • Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B
    • Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232(1):25–32.
    • (1992) J Intern Med , vol.232 , Issue.1 , pp. 25-32
    • Nilsson, I.M.1    Berntorp, E.2    Lofqvist, T.3    Pettersson, H.4
  • 8
    • 33746684369 scopus 로고    scopus 로고
    • Neonatal Fc receptor for IgG regulates mucosal immune responses to luminal bacteria
    • Yoshida M, Kobayashi K, Kuo TT, et al. Neonatal Fc receptor for IgG regulates mucosal immune responses to luminal bacteria. J Clin Invest. 2006;116(8):2142–2151.
    • (2006) J Clin Invest , vol.116 , Issue.8 , pp. 2142-2151
    • Yoshida, M.1    Kobayashi, K.2    Kuo, T.T.3
  • 9
    • 0029664769 scopus 로고    scopus 로고
    • The impact of prophylactic treatment on children with severe haemophilia
    • Liesner RJ, Khair K, Hann IM. The impact of prophylactic treatment on children with severe haemophilia. Br J Haematol. 1996;92(4): 973–978.
    • (1996) Br J Haematol , vol.92 , Issue.4 , pp. 973-978
    • Liesner, R.J.1    Khair, K.2    Hann, I.M.3
  • 10
    • 0035106558 scopus 로고    scopus 로고
    • Long-term outcome of individualized prophylactic treatment of children with severe haemophilia
    • van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol. 2001;112(3):561–565.
    • (2001) Br J Haematol , vol.112 , Issue.3 , pp. 561-565
    • Van Den Berg, H.M.1    Fischer, K.2    Mauser-Bunschoten, E.P.3
  • 11
    • 34547757915 scopus 로고    scopus 로고
    • Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia
    • Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535–544.
    • (2007) N Engl J Med , vol.357 , Issue.6 , pp. 535-544
    • Manco-Johnson, M.J.1    Abshire, T.C.2    Shapiro, A.D.3
  • 12
    • 0036529818 scopus 로고    scopus 로고
    • The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia
    • Fischer K, van der Bom JG, Mauser-Bunschoten EP, et al. The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia. Blood. 2002;99(7):2337–2341.
    • (2002) Blood , vol.99 , Issue.7 , pp. 2337-2341
    • Fischer, K.1    Van Der Bom, J.G.2    Mauser-Bunschoten, E.P.3
  • 13
    • 0034003628 scopus 로고    scopus 로고
    • Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. The French Study Group
    • Molho P, Rolland N, Lebrun T, et al. Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. The French Study Group. secretariat.haemophiles@cch.ap-hop-paris.fr. Haemophilia. 2000;6(1):23–32.
    • (2000) Haemophilia , vol.6 , Issue.1 , pp. 23-32
    • Molho, P.1    Rolland, N.2    Lebrun, T.3
  • 14
    • 84885642596 scopus 로고    scopus 로고
    • Present and future challenges in the treatment of haemophilia: A clinician’s perspective
    • Mannucci PM, Franchini M. Present and future challenges in the treatment of haemophilia: a clinician’s perspective. Blood Transfus. 2013;11(Suppl 4):S77–S81.
    • (2013) Blood Transfus , vol.11 , Issue.4 , pp. S77-S81
    • Mannucci, P.M.1    Franchini, M.2
  • 15
    • 33645998423 scopus 로고    scopus 로고
    • Mortality and causes of death in patients with hemophilia, 1992–2001: A prospective cohort study
    • Plug I, Van Der Bom JG, Peters M, et al. Mortality and causes of death in patients with hemophilia, 1992–2001: a prospective cohort study. J Thromb Haemost. 2006;4(3):510–516.
    • (2006) J Thromb Haemost , vol.4 , Issue.3 , pp. 510-516
    • Plug, I.1    Van Der Bom, J.G.2    Peters, M.3
  • 16
    • 84879462669 scopus 로고    scopus 로고
    • Novel coagulation factor concentrates: Issues relating to their clinical implementation and pharmacokinetic assessment for optimal prophylaxis in haemophilia patients
    • Ljung R, Auerswald G, Benson G, et al. Novel coagulation factor concentrates: issues relating to their clinical implementation and pharmacokinetic assessment for optimal prophylaxis in haemophilia patients. Haemophilia. 2013;19(4):481–486.
    • (2013) Haemophilia , vol.19 , Issue.4 , pp. 481-486
    • Ljung, R.1    Auerswald, G.2    Benson, G.3
  • 17
    • 34447136593 scopus 로고    scopus 로고
    • Quebec, Canada: World Federation of Hemophilia. Available from:, Accessed October 2, 2015
    • World Federation of Hemophilia. Guidelines for the Management of Hemophilia. Quebec, Canada: World Federation of Hemophilia. Available from: http://www1.wfh.org/publication/files/pdf-1472.pdf. Accessed October 2, 2015.
    • Guidelines for the Management of Hemophilia
  • 18
    • 84862872608 scopus 로고    scopus 로고
    • Personalized prophylaxis
    • Collins PW. Personalized prophylaxis. Haemophilia. 2012;18(Suppl 4):131–135.
    • (2012) Haemophilia , vol.18 , Issue.4 , pp. 131-135
    • Collins, P.W.1
  • 19
    • 84899562558 scopus 로고    scopus 로고
    • Changing paradigm of prophylaxis with longer acting factor concentrates
    • Carcao M. Changing paradigm of prophylaxis with longer acting factor concentrates. Haemophilia. 2014;20(Suppl 4):99–105.
    • (2014) Haemophilia , vol.20 , Issue.4 , pp. 99-105
    • Carcao, M.1
  • 21
    • 0021677942 scopus 로고
    • Structure of human factor VIII
    • Vehar G, Keyt B, Eaton D, et al. Structure of human factor VIII. Nature. 1984;312:337–342.
    • (1984) Nature , vol.312 , pp. 337-342
    • Vehar, G.1    Keyt, B.2    Eaton, D.3
  • 22
    • 69449088981 scopus 로고    scopus 로고
    • Models for prediction of factor VIII half-life in severe haemophiliacs: Distinct approaches for blood group O and non-O patients
    • Fischer K, Pendu R, van Schooten CJ, et al. Models for prediction of factor VIII half-life in severe haemophiliacs: distinct approaches for blood group O and non-O patients. PLoS One. 2009;4(8):e6745.
    • (2009) Plos One , vol.4 , Issue.8
    • Fischer, K.1    Pendu, R.2    Van Schooten, C.J.3
  • 23
    • 0028810479 scopus 로고
    • Inter-individual variation in half-life of infused recombinant factor VIII is related to pre-infusion von Willebrand factor antigen levels
    • Fijnvandraat K, Peters M, ten Cate JW. Inter-individual variation in half-life of infused recombinant factor VIII is related to pre-infusion von Willebrand factor antigen levels. Br J Haematol. 1995;91(2): 474–476.
    • (1995) Br J Haematol , vol.91 , Issue.2 , pp. 474-476
    • Fijnvandraat, K.1    Peters, M.2    Ten Cate, J.W.3
  • 24
    • 0034913232 scopus 로고    scopus 로고
    • Barriers to compliance with prophylaxis therapy in haemophilia
    • Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia. 2001;7(4): 392–396.
    • (2001) Haemophilia , vol.7 , Issue.4 , pp. 392-396
    • Hacker, M.R.1    Geraghty, S.2    Manco-Johnson, M.3
  • 25
    • 51249087295 scopus 로고    scopus 로고
    • A survey of adherence to haemophilia therapy in six European countries: Results and recommendations
    • De Moerloose P, Urbancik W, Van Den Berg HM, Richards M. A survey of adherence to haemophilia therapy in six European countries: results and recommendations. Haemophilia. 2008;14(5):931–938.
    • (2008) Haemophilia , vol.14 , Issue.5 , pp. 931-938
    • De Moerloose, P.1    Urbancik, W.2    Van Den Berg, H.M.3    Richards, M.4
  • 26
    • 33750595983 scopus 로고    scopus 로고
    • Catheter-related deep venous thrombosis and other catheter complications in children with cancer
    • Journeycake JM, Buchanan GR. Catheter-related deep venous thrombosis and other catheter complications in children with cancer. J Clin Oncol. 2006;24(28):4575–4580.
    • (2006) J Clin Oncol , vol.24 , Issue.28 , pp. 4575-4580
    • Journeycake, J.M.1    Buchanan, G.R.2
  • 27
    • 84856866914 scopus 로고    scopus 로고
    • International Immune Tolerance Study. 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(6):1335–1344.
    • (2012) Blood , vol.119 , Issue.6 , pp. 1335-1344
    • Hay, C.R.1    Dimichele, D.M.2
  • 28
    • 30344434999 scopus 로고    scopus 로고
    • Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A
    • Goudemand J, Rothschild C, Demiguel V, et al. Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood. 2006; 107(1):46–51.
    • (2006) Blood , vol.107 , Issue.1 , pp. 46-51
    • Goudemand, J.1    Rothschild, C.2    Demiguel, V.3
  • 29
    • 34249711370 scopus 로고    scopus 로고
    • Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: The CANAL cohort study
    • Gouw SC, van der Bom JG, Auerswald G, Ettinghausen CE, Tedgard U, van den Berg HM. Recombinant versus plasma-derived factor VIII products and the development of inhibitors in previously untreated patients with severe hemophilia A: the CANAL cohort study. Blood. 2007;109(11):4693–4697.
    • (2007) Blood , vol.109 , Issue.11 , pp. 4693-4697
    • Gouw, S.C.1    Van Der Bom, J.G.2    Auerswald, G.3    Ettinghausen, C.E.4    Tedgard, U.5    Van Den Berg, H.M.6
  • 30
    • 77954506432 scopus 로고    scopus 로고
    • Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: A systematic review
    • Iorio A, Halimeh S, Holzhauer S, et al. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost. 2010;8(6):1256–1265.
    • (2010) J Thromb Haemost , vol.8 , Issue.6 , pp. 1256-1265
    • Iorio, A.1    Halimeh, S.2    Holzhauer, S.3
  • 31
    • 84855912746 scopus 로고    scopus 로고
    • Cumulative inhibitor incidence in previously untreated patients with severe hemophilia A treated with plasma-derived versus recombinant factor VIII concentrates: A critical systematic review
    • Franchini M, Tagliaferri A, Mengoli C, Cruciani M. Cumulative inhibitor incidence in previously untreated patients with severe hemophilia A treated with plasma-derived versus recombinant factor VIII concentrates: a critical systematic review. Crit Rev Oncol Hematol. 2012;81(1): 82–93.
    • (2012) Crit Rev Oncol Hematol , vol.81 , Issue.1 , pp. 82-93
    • Franchini, M.1    Tagliaferri, A.2    Mengoli, C.3    Cruciani, M.4
  • 32
    • 84914142516 scopus 로고    scopus 로고
    • Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000–2011
    • Collins PW, Palmer BP, Chalmers EA, et al. Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000–2011. Blood. 2014;124(23): 3389–3397.
    • (2014) Blood , vol.124 , Issue.23 , pp. 3389-3397
    • Collins, P.W.1    Palmer, B.P.2    Chalmers, E.A.3
  • 33
    • 84914140043 scopus 로고    scopus 로고
    • Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A
    • Calvez T, Chambost H, Claeyssens-Donadel S, et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A. Blood. 2014;124(23):3398–3408.
    • (2014) Blood , vol.124 , Issue.23 , pp. 3398-3408
    • Calvez, T.1    Chambost, H.2    Claeyssens-Donadel, S.3
  • 34
    • 84872450786 scopus 로고    scopus 로고
    • Factor VIII products and inhibitor development in severe hemophilia A
    • Gouw SC, van der Bom JG, Ljung R, et al. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013; 368(3):231–239.
    • (2013) N Engl J Med , vol.368 , Issue.3 , pp. 231-239
    • Gouw, S.C.1    Van Der Bom, J.G.2    Ljung, R.3
  • 35
    • 84873043409 scopus 로고    scopus 로고
    • Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein
    • Peters RT, Toby G, Lu Q, et al. Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein. J Thromb Haemost. 2013;11(1):132–141.
    • (2013) J Thromb Haemost , vol.11 , Issue.1 , pp. 132-141
    • Peters, R.T.1    Toby, G.2    Lu, Q.3
  • 36
    • 84859192874 scopus 로고    scopus 로고
    • Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs
    • Dumont JA, Liu T, Low SC, et al. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood. 2012;119(13):3024–3030.
    • (2012) Blood , vol.119 , Issue.13 , pp. 3024-3030
    • Dumont, J.A.1    Liu, T.2    Low, S.C.3
  • 37
    • 84859197329 scopus 로고    scopus 로고
    • Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
    • Powell JS, Josephson NC, Quon D, et al. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012;119(13):3031–3037.
    • (2012) Blood , vol.119 , Issue.13 , pp. 3031-3037
    • Powell, J.S.1    Josephson, N.C.2    Quon, D.3
  • 38
    • 84893123337 scopus 로고    scopus 로고
    • Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
    • Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–325.
    • (2014) Blood , vol.123 , Issue.3 , pp. 317-325
    • Mahlangu, J.1    Powell, J.S.2    Ragni, M.V.3
  • 40
    • 34548229364 scopus 로고    scopus 로고
    • FcRn: The neonatal Fc receptor comes of age
    • Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 2007;7(9):715–725.
    • (2007) Nat Rev Immunol , vol.7 , Issue.9 , pp. 715-725
    • Roopenian, D.C.1    Akilesh, S.2
  • 41
    • 0020541752 scopus 로고
    • Intracellular distribution and degradation of immunoglobulin G and immunoglobulin G fragments injected into HeLa cells
    • McGarry T, Hough R, Rogers S, Rechsteiner M. Intracellular distribution and degradation of immunoglobulin G and immunoglobulin G fragments injected into HeLa cells. J Cell Biol. 1983;96(2):338–346.
    • (1983) J Cell Biol , vol.96 , Issue.2 , pp. 338-346
    • McGarry, T.1    Hough, R.2    Rogers, S.3    Rechsteiner, M.4
  • 42
    • 77950391547 scopus 로고    scopus 로고
    • Prolonged activity of factor IX as a monomeric Fc fusion protein
    • Peters RT, Low SC, Kamphaus GD, et al. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood. 2010;115(10):2057–2064.
    • (2010) Blood , vol.115 , Issue.10 , pp. 2057-2064
    • Peters, R.T.1    Low, S.C.2    Kamphaus, G.D.3
  • 43
    • 84860912187 scopus 로고    scopus 로고
    • Production platforms for biotherapeutic glycoproteins. Occurrence, impact, and challenges of non-human sialylation
    • Ghaderi D, Zhang M, Hurtado-Ziola N, Varki A. Production platforms for biotherapeutic glycoproteins. Occurrence, impact, and challenges of non-human sialylation. Biotechnol Genet Eng Rev. 2012;28:147–175.
    • (2012) Biotechnol Genet Eng Rev , vol.28 , pp. 147-175
    • Ghaderi, D.1    Zhang, M.2    Hurtado-Ziola, N.3    Varki, A.4
  • 44
    • 84911430479 scopus 로고    scopus 로고
    • Design of the INHIBIT trial: Preventing inhibitors by avoiding ‘danger’, prolonging half-life and promoting tolerance
    • Ragni MV, Malec LM. Design of the INHIBIT trial: preventing inhibitors by avoiding ‘danger’, prolonging half-life and promoting tolerance. Expert Rev Hematol. 2014;7(6):747–755.
    • (2014) Expert Rev Hematol , vol.7 , Issue.6 , pp. 747-755
    • Ragni, M.V.1    Malec, L.M.2
  • 45
    • 84922973668 scopus 로고    scopus 로고
    • The incidence of factor VIII inhibitors in severe haemophilia A following a major switch from full-length to B-domain-deleted factor VIII: A prospective cohort comparison
    • Hay CR, Palmer BP, Chalmers EA, et al. The incidence of factor VIII inhibitors in severe haemophilia A following a major switch from full-length to B-domain-deleted factor VIII: a prospective cohort comparison. Haemophilia. 2015;21(2):219–226.
    • (2015) Haemophilia , vol.21 , Issue.2 , pp. 219-226
    • Hay, C.R.1    Palmer, B.P.2    Chalmers, E.A.3
  • 46
    • 3543091053 scopus 로고    scopus 로고
    • Efficacy and inhibitor development in previously treated patients with haemophilia A switched to a B domain-deleted recombinant factor VIII
    • Gringeri A, Tagliaferri A, Tagariello G, et al. Efficacy and inhibitor development in previously treated patients with haemophilia A switched to a B domain-deleted recombinant factor VIII. Br J Haematol. 2004;126(3):398–404.
    • (2004) Br J Haematol , vol.126 , Issue.3 , pp. 398-404
    • Gringeri, A.1    Tagliaferri, A.2    Tagariello, G.3
  • 47
    • 54049088585 scopus 로고    scopus 로고
    • Activation of natural regulatory T cells by IgG Fc-derived peptide “Tregitopes
    • De Groot AS, Moise L, McMurry JA, et al. Activation of natural regulatory T cells by IgG Fc-derived peptide “Tregitopes”. Blood. 2008; 112(8):3303–3311.
    • (2008) Blood , vol.112 , Issue.8 , pp. 3303-3311
    • De Groot, A.S.1    Moise, L.2    McMurry, J.A.3
  • 48
    • 20444476217 scopus 로고    scopus 로고
    • Induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins
    • Lei TC, Scott DW. Induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins. Blood. 2005;105(12):4865–4870.
    • (2005) Blood , vol.105 , Issue.12 , pp. 4865-4870
    • Lei, T.C.1    Scott, D.W.2
  • 49
    • 84867166712 scopus 로고    scopus 로고
    • Association between physical activity and risk of bleeding in children with hemophilia
    • Broderick CR, Herbert RD, Latimer J, et al. Association between physical activity and risk of bleeding in children with hemophilia. JAMA. 2012;308(14):1452–1459.
    • (2012) JAMA , vol.308 , Issue.14 , pp. 1452-1459
    • Broderick, C.R.1    Herbert, R.D.2    Latimer, J.3
  • 52
    • 84862867193 scopus 로고    scopus 로고
    • WFH: Closing the global gap – achieving optimal care
    • Skinner MW. WFH: closing the global gap – achieving optimal care. Haemophilia. 2012;18(Suppl 4):1–12.
    • (2012) Haemophilia , vol.18 , Issue.4 , pp. 1-12
    • Skinner, M.W.1
  • 53
    • 84876182114 scopus 로고    scopus 로고
    • Enhancing the pharmacokinetic properties of recombinant factor VIII: First-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A
    • Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013;11(4):670–678.
    • (2013) J Thromb Haemost , vol.11 , Issue.4 , pp. 670-678
    • Tiede, A.1    Brand, B.2    Fischer, R.3
  • 54
    • 84873625664 scopus 로고    scopus 로고
    • BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation
    • Turecek PL, Bossard MJ, Graninger M, et al. BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation. Hamostaseologie. 2012;32(Suppl 1): S29–S38.
    • (2012) Hamostaseologie , vol.32 , Issue.1 , pp. S29-S38
    • Turecek, P.L.1    Bossard, M.J.2    Graninger, M.3
  • 55
    • 84898028710 scopus 로고    scopus 로고
    • Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A
    • Coyle TE, Reding MT, Lin JC, Michaels LA, Shah A, Powell J. Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. J Thromb Haemost. 2014;12(4):488–496.
    • (2014) J Thromb Haemost , vol.12 , Issue.4 , pp. 488-496
    • Coyle, T.E.1    Reding, M.T.2    Lin, J.C.3    Michaels, L.A.4    Shah, A.5    Powell, J.6
  • 56
    • 84883770241 scopus 로고    scopus 로고
    • Preclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIII
    • Zollner SB, Raquet E, Muller-Cohrs J, et al. Preclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIII. Thromb Res. 2013;132(2):280–287.
    • (2013) Thromb Res , vol.132 , Issue.2 , pp. 280-287
    • Zollner, S.B.1    Raquet, E.2    Muller-Cohrs, J.3
  • 57
    • 84902547350 scopus 로고    scopus 로고
    • Non-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant single-chain factor VIII
    • Zollner S, Raquet E, Claar P, et al. Non-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant single-chain factor VIII. Thromb Res. 2014;134(1):125–131.
    • (2014) Thromb Res , vol.134 , Issue.1 , pp. 125-131
    • Zollner, S.1    Raquet, E.2    Claar, P.3
  • 58
    • 84870302675 scopus 로고    scopus 로고
    • A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model
    • Kitazawa T, Igawa T, Sampei Z, et al. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 2012;18(10):1570–1574.
    • (2012) Nat Med , vol.18 , Issue.10 , pp. 1570-1574
    • Kitazawa, T.1    Igawa, T.2    Sampei, Z.3
  • 59
    • 84893537691 scopus 로고    scopus 로고
    • Anti-factor IXa/X bispecific antibody (ACE910): Hemostatic potency against ongoing bleeds in a hemophilia A model and the possibility of routine supplementation
    • Muto A, Yoshihashi K, Takeda M, et al. Anti-factor IXa/X bispecific antibody (ACE910): hemostatic potency against ongoing bleeds in a hemophilia A model and the possibility of routine supplementation. J Thromb Haemost. 2014;12(2):206–213.
    • (2014) J Thromb Haemost , vol.12 , Issue.2 , pp. 206-213
    • Muto, A.1    Yoshihashi, K.2    Takeda, M.3
  • 61
    • 84928764693 scopus 로고    scopus 로고
    • Safety and pharmacokinetics of anti-TFPI antibody (Concizumab) in healthy volunteers and patients with hemophilia: A randomized first human dose trial
    • Chowdary P, Lethagen S, Friedrich U, et al. Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. J Thromb Haemost. Epub January 31, 2015.
    • (2015) J Thromb Haemost. Epub January , pp. 31
    • Chowdary, P.1    Lethagen, S.2    Friedrich, U.3
  • 63
    • 84855161388 scopus 로고    scopus 로고
    • Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    • Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357–2365.
    • (2011) N Engl J Med , vol.365 , Issue.25 , pp. 2357-2365
    • Nathwani, A.C.1    Tuddenham, E.G.2    Rangarajan, S.3
  • 64
    • 84911383748 scopus 로고    scopus 로고
    • Long-term safety and efficacy of factor IX gene therapy in hemophilia B
    • Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371(21):1994–2004.
    • (2014) N Engl J Med , vol.371 , Issue.21 , pp. 1994-2004
    • Nathwani, A.C.1    Reiss, U.M.2    Tuddenham, E.G.3
  • 65
    • 84880427173 scopus 로고    scopus 로고
    • Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype
    • Siner JI, Iacobelli NP, Sabatino DE, et al. Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype. Blood. 2013;121(21):4396–4403.
    • (2013) Blood , vol.121 , Issue.21 , pp. 4396-4403
    • Siner, J.I.1    Iacobelli, N.P.2    Sabatino, D.E.3
  • 66
    • 0037988905 scopus 로고    scopus 로고
    • Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
    • Mingozzi F, Liu YL, Dobrzynski E, et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest. 2003;111(9):1347–1356.
    • (2003) J Clin Invest , vol.111 , Issue.9 , pp. 1347-1356
    • Mingozzi, F.1    Liu, Y.L.2    Dobrzynski, E.3
  • 67
    • 84924997217 scopus 로고    scopus 로고
    • AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
    • Crudele JM, Finn JD, Siner JI, et al. AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood. 2015;125(10):1553–1561.
    • (2015) Blood , vol.125 , Issue.10 , pp. 1553-1561
    • Crudele, J.M.1    Finn, J.D.2    Siner, J.I.3
  • 68
    • 78650647673 scopus 로고    scopus 로고
    • Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy
    • Finn JD, Ozelo MC, Sabatino DE, et al. Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy. Blood. 2010;116(26):5842–5848.
    • (2010) Blood , vol.116 , Issue.26 , pp. 5842-5848
    • Finn, J.D.1    Ozelo, M.C.2    Sabatino, D.E.3
  • 69
    • 53449090226 scopus 로고    scopus 로고
    • Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity
    • Shi Q, Fahs SA, Wilcox DA, et al. Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity. Blood. 2008;112(7):2713–2721.
    • (2008) Blood , vol.112 , Issue.7 , pp. 2713-2721
    • Shi, Q.1    Fahs, S.A.2    Wilcox, D.A.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.