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Volumn 7, Issue , 2016, Pages 27-38

New developments in the management of moderate-to-severe hemophilia B

Author keywords

Bleeding disorders; Factor IX; Hemophilia B; Plasma half life; Therapy

Indexed keywords

BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 9;

EID: 84979725668     PISSN: None     EISSN: 11792736     Source Type: Journal    
DOI: 10.2147/JBM.S81520     Document Type: Review
Times cited : (27)

References (96)
  • 2
    • 0036218048 scopus 로고    scopus 로고
    • Haemophilia A and haemophilia B: Molecular insights
    • Bowen DJ. Haemophilia A and haemophilia B: molecular insights. Mol Pathol. 2002; 55(2): 127-144.
    • (2002) Mol Pathol , vol.55 , Issue.2 , pp. 127-144
    • Bowen, D.J.1
  • 4
    • 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
  • 5
    • 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, Lusher JM, Rothschild C, Ingerslev J. 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    Lusher, J.M.4    Rothschild, C.5    Ingerslev, J.6
  • 6
    • 83555173200 scopus 로고    scopus 로고
    • Exploration of the pathogenesis of haemophilic joint arthropathy: Understanding implications for optimal clinical management
    • Acharya SS. Exploration of the pathogenesis of haemophilic joint arthropathy: understanding implications for optimal clinical management. Br J Haematol. 2012; 156(1): 13-23.
    • (2012) Br J Haematol , vol.156 , Issue.1 , pp. 13-23
    • Acharya, S.S.1
  • 7
    • 79952657396 scopus 로고    scopus 로고
    • Update on treatment regimens: Prophylaxis versus on-demand therapy
    • Manco-Johnson MJ. Update on treatment regimens: prophylaxis versus on-demand therapy. Semin Hematol. 2003; 40(3 Suppl 3): 3-9.
    • (2003) Semin Hematol , vol.40 , Issue.3 , pp. 3-9
    • Manco-Johnson, M.J.1
  • 8
    • 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
  • 9
    • 0027123107 scopus 로고
    • Molecular and cellular biology of blood coagulation
    • Furie B, Furie BC. Molecular and cellular biology of blood coagulation. N Engl J Med. 1992; 326(12): 800-806.
    • (1992) N Engl J Med , vol.326 , Issue.12 , pp. 800-806
    • Furie, B.1    Furie, B.C.2
  • 10
    • 0031860158 scopus 로고    scopus 로고
    • Post-translational modifications required for coagulation factor secretion and function
    • Kaufman RJ. Post-translational modifications required for coagulation factor secretion and function. Thromb Haemost. 1998; 79(6): 1068-1079.
    • (1998) Thromb Haemost , vol.79 , Issue.6 , pp. 1068-1079
    • Kaufman, R.J.1
  • 11
    • 0032697618 scopus 로고    scopus 로고
    • Biochemistry and physiology of blood coagulation
    • Mann KG. Biochemistry and physiology of blood coagulation. Thromb Haemost. 1999; 82(2): 165-174.
    • (1999) Thromb Haemost , vol.82 , Issue.2 , pp. 165-174
    • Mann, K.G.1
  • 12
    • 0029144243 scopus 로고
    • Factors IXa and Xa play distinct roles in tissue factor-dependent initiation of coagulation
    • Hoffman M, Monroe DM, Oliver JA, Roberts HR. Factors IXa and Xa play distinct roles in tissue factor-dependent initiation of coagulation. Blood. 1995; 86(5): 1794-1801.
    • (1995) Blood , vol.86 , Issue.5 , pp. 1794-1801
    • Hoffman, M.1    Monroe, D.M.2    Oliver, J.A.3    Roberts, H.R.4
  • 14
    • 0035189391 scopus 로고    scopus 로고
    • Pharmacokinetics of coagulation factors: Clinical relevance for patients with haemophilia
    • Björkman S, Berntorp E. Pharmacokinetics of coagulation factors: clinical relevance for patients with haemophilia. Clin Pharmacokinet. 2001; 40(11): 815-832.
    • (2001) Clin Pharmacokinet , vol.40 , Issue.11 , pp. 815-832
    • Björkman, S.1    Berntorp, E.2
  • 15
    • 0028365342 scopus 로고
    • Pharmacokinetics of factor IX in patients with haemophilia B. Methodological aspects and physiological interpretation
    • Björkman S, Carlsson M, Berntorp E. Pharmacokinetics of factor IX in patients with haemophilia B. Methodological aspects and physiological interpretation. Eur J Clin Pharmacol. 1994; 46(4): 325-332.
    • (1994) Eur J Clin Pharmacol , vol.46 , Issue.4 , pp. 325-332
    • Björkman, S.1    Carlsson, M.2    Berntorp, E.3
  • 17
    • 84355163092 scopus 로고    scopus 로고
    • Factor IX replacement to cover total knee replacement surgery in haemophilia B: A single-centre experience, 2000-2010
    • Uprichard J, Adamidou D, Goddard NJ, Mann HA, Yee TT. Factor IX replacement to cover total knee replacement surgery in haemophilia B: a single-centre experience, 2000-2010. Haemophilia. 2012; 18(1): 46-49.
    • (2012) Haemophilia , vol.18 , Issue.1 , pp. 46-49
    • Uprichard, J.1    Adamidou, D.2    Goddard, N.J.3    Mann, H.A.4    Yee, T.T.5
  • 18
    • 0023270387 scopus 로고
    • In vivo evidence of intravascular binding sites for coagulation factor IX
    • Stern DM, Knitter G, Kisiel W, Nawroth PP. In vivo evidence of intravascular binding sites for coagulation factor IX. Br J Haematol. 1987; 66(2): 227-232.
    • (1987) Br J Haematol , vol.66 , Issue.2 , pp. 227-232
    • Stern, D.M.1    Knitter, G.2    Kisiel, W.3    Nawroth, P.P.4
  • 19
    • 84889632409 scopus 로고    scopus 로고
    • Evidence of clinically significant extravascular stores of factor IX
    • Feng D, Stafford KA, Broze GJ, Stafford DW. Evidence of clinically significant extravascular stores of factor IX. J Thromb Haemost. 2013; 11(12): 2176-2178.
    • (2013) J Thromb Haemost , vol.11 , Issue.12 , pp. 2176-2178
    • Feng, D.1    Stafford, K.A.2    Broze, G.J.3    Stafford, D.W.4
  • 21
    • 0036660414 scopus 로고    scopus 로고
    • Circulating and binding characteristics of wild-type factor IX and certain Gla domain mutants in vivo
    • Gui T, Lin HF, Jin DY, et al. Circulating and binding characteristics of wild-type factor IX and certain Gla domain mutants in vivo. Blood. 2002; 100(1): 153-158.
    • (2002) Blood , vol.100 , Issue.1 , pp. 153-158
    • Gui, T.1    Lin, H.F.2    Jin, D.Y.3
  • 22
    • 0019785751 scopus 로고
    • A simplified procedure for purification of human prothrombin, factor IX and factor X
    • Bajaj SP, Rapaport SI, Prodanos C. A simplified procedure for purification of human prothrombin, factor IX and factor X. Prep Biochem. 1981; 11(4): 397-412.
    • (1981) Prep Biochem , vol.11 , Issue.4 , pp. 397-412
    • Bajaj, S.P.1    Rapaport, S.I.2    Prodanos, C.3
  • 23
    • 70449453157 scopus 로고    scopus 로고
    • Abnormal hemostasis in a knock-in mouse carrying a variant of factor IX with impaired binding to collagen type IV
    • Gui T, Reheman A, Ni H, et al. Abnormal hemostasis in a knock-in mouse carrying a variant of factor IX with impaired binding to collagen type IV. J Thromb Haemost. 2009; 7(11): 1843-1851.
    • (2009) J Thromb Haemost , vol.7 , Issue.11 , pp. 1843-1851
    • Gui, T.1    Reheman, A.2    Ni, H.3
  • 24
    • 0031977163 scopus 로고    scopus 로고
    • Clinical evaluation of recombinant factor IX
    • White G, Shapiro A, Ragni M, et al. Clinical evaluation of recombinant factor IX. Semin Hematol. 1998; 35(2 Suppl 2): 33-38.
    • (1998) Semin Hematol , vol.35 , Issue.2 , pp. 33-38
    • White, G.1    Shapiro, A.2    Ragni, M.3
  • 25
    • 0029043733 scopus 로고
    • Variability of in vivo recovery of factor IX after infusion of monoclonal antibody purified factor IX concentrates in patients with hemophilia B. The Mononine Study Group
    • White GC 2nd, Shapiro AD, Kurczynski EM, Kim HC, Bergman GE. Variability of in vivo recovery of factor IX after infusion of monoclonal antibody purified factor IX concentrates in patients with hemophilia B. The Mononine Study Group. Thromb Haemost. 1995; 73(5): 779-784.
    • (1995) Thromb Haemost , vol.73 , Issue.5 , pp. 779-784
    • White, G.C.1    Shapiro, A.D.2    Kurczynski, E.M.3    Kim, H.C.4    Bergman, G.E.5
  • 26
    • 0036484156 scopus 로고    scopus 로고
    • Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B
    • Ewenstein BM, Joist JH, Shapiro AD, et al. Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B. Transfusion. 2002; 42(2): 190-197.
    • (2002) Transfusion , vol.42 , Issue.2 , pp. 190-197
    • Ewenstein, B.M.1    Joist, J.H.2    Shapiro, A.D.3
  • 27
    • 0036123458 scopus 로고    scopus 로고
    • Recombinant factor IX recovery and inhibitor safety: A Canadian post-licensure surveillance study
    • Poon MC, Lillicrap D, Hensman C, Card R, Scully MF. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thromb Haemost. 2002; 87(3): 431-435.
    • (2002) Thromb Haemost , vol.87 , Issue.3 , pp. 431-435
    • Poon, M.C.1    Lillicrap, D.2    Hensman, C.3    Card, R.4    Scully, M.F.5
  • 28
    • 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
  • 29
    • 0035165638 scopus 로고    scopus 로고
    • Posttranslational modifications of recombinant myotube-synthesized human factor IX
    • Arruda VR, Hagstrom JN, Deitch J, et al. Posttranslational modifications of recombinant myotube-synthesized human factor IX. Blood. 2001; 97(1): 130-138.
    • (2001) Blood , vol.97 , Issue.1 , pp. 130-138
    • Arruda, V.R.1    Hagstrom, J.N.2    Deitch, J.3
  • 30
    • 45749147034 scopus 로고    scopus 로고
    • Analysis of the N-glycans of recombinant human factor IX purified from transgenic pig milk
    • Gil GC, Velander WH, Van Cott KE. Analysis of the N-glycans of recombinant human factor IX purified from transgenic pig milk. Glycobiology. 2008; 18(7): 526-539.
    • (2008) Glycobiology , vol.18 , Issue.7 , pp. 526-539
    • Gil, G.C.1    Velander, W.H.2    Van Cott, K.E.3
  • 32
    • 0019391468 scopus 로고
    • Prothrombin complex concentrates: Clinical use
    • Menache D. Prothrombin complex concentrates: clinical use. Ann N Y Acad Sci. 1981; 370: 747-756.
    • (1981) Ann N Y Acad Sci , vol.370 , pp. 747-756
    • Menache, D.1
  • 33
    • 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
  • 34
    • 0027255828 scopus 로고
    • Clinical evaluation of viral safety of coagulation factor VIII and IX concentrates
    • Mannucci PM. Clinical evaluation of viral safety of coagulation factor VIII and IX concentrates. Vox Sang. 1993; 64(4): 197-203.
    • (1993) Vox Sang , vol.64 , Issue.4 , pp. 197-203
    • Mannucci, P.M.1
  • 35
    • 34547436844 scopus 로고    scopus 로고
    • Coagulation factor concentrates: Past, present, and future
    • Key NS, Negrier C. Coagulation factor concentrates: past, present, and future. Lancet. 2007; 370(9585): 439-448.
    • (2007) Lancet , vol.370 , Issue.9585 , pp. 439-448
    • Key, N.S.1    Negrier, C.2
  • 36
    • 0029151126 scopus 로고
    • High-purity factor IX concentrates for treatment of hemophilia B: Relative purity and thrombogenic potential
    • Limentani SA, Gowell KP, Deitcher SR. High-purity factor IX concentrates for treatment of hemophilia B: relative purity and thrombogenic potential. Acta Haematol. 1995; 94(Suppl 1): 12-17.
    • (1995) Acta Haematol , vol.94 , pp. 12-17
    • Limentani, S.A.1    Gowell, K.P.2    Deitcher, S.R.3
  • 37
    • 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
  • 38
    • 0026635406 scopus 로고
    • Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
    • Nilsson IM, Berntorp E, Löfqvist 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    Löfqvist, T.3    Pettersson, H.4
  • 39
    • 78650316037 scopus 로고    scopus 로고
    • Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States
    • Witmer C, Presley R, Kulkarni R, Soucie JM, Manno CS, Raffini L. Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States. Br J Haematol. 2011; 152(2): 211-216.
    • (2011) Br J Haematol , vol.152 , Issue.2 , pp. 211-216
    • Witmer, C.1    Presley, R.2    Kulkarni, R.3    Soucie, J.M.4    Manno, C.S.5    Raffini, L.6
  • 40
    • 84942191461 scopus 로고    scopus 로고
    • Health-related quality of life assessment in haemophilia patients on prophylaxis therapy: A systematic review of results from prospective clinical trials
    • Oladapo AO, Epstein JD, Williams E, Ito D, Gringeri A, Valentino LA. Health-related quality of life assessment in haemophilia patients on prophylaxis therapy: a systematic review of results from prospective clinical trials. Haemophilia. 2015; 21: e344-e358.
    • (2015) Haemophilia , vol.21 , pp. e344-e358
    • Oladapo, A.O.1    Epstein, J.D.2    Williams, E.3    Ito, D.4    Gringeri, A.5    Valentino, L.A.6
  • 41
    • 84857019132 scopus 로고    scopus 로고
    • Prophylaxis in children with hemophilia: Evidence-based achievements, old and new challenges
    • Coppola A, Tagliaferri A, Di Capua M, Franchini M. Prophylaxis in children with hemophilia: evidence-based achievements, old and new challenges. Semin Thromb Hemost. 2012; 38(1): 79-94.
    • (2012) Semin Thromb Hemost , vol.38 , Issue.1 , pp. 79-94
    • Coppola, A.1    Tagliaferri, A.2    Di Capua, M.3    Franchini, M.4
  • 42
    • 70449562279 scopus 로고    scopus 로고
    • Joint outcomes in patients with haemophilia: The importance of adherence to preventive regimens
    • Berntorp E. Joint outcomes in patients with haemophilia: the importance of adherence to preventive regimens. Haemophilia. 2009; 15(6): 1219-1227.
    • (2009) Haemophilia , vol.15 , Issue.6 , pp. 1219-1227
    • Berntorp, E.1
  • 43
    • 27844499655 scopus 로고    scopus 로고
    • Management of hemophilia with minimal factor replacement in developing countries: Role of ancillary therapy
    • Chandy M. Management of hemophilia with minimal factor replacement in developing countries: role of ancillary therapy. Semin Thromb Hemost. 2005; 31(5): 501-506.
    • (2005) Semin Thromb Hemost , vol.31 , Issue.5 , pp. 501-506
    • Chandy, M.1
  • 44
    • 0026501455 scopus 로고
    • Purified factor IX using monoclonal immunoaffinity technique: Clinical trials in hemophilia B and comparison to prothrombin complex concentrates
    • Kim HC, McMillan CW, White GC, Bergman GE, Horton MW, Saidi P. Purified factor IX using monoclonal immunoaffinity technique: clinical trials in hemophilia B and comparison to prothrombin complex concentrates. Blood. 1992; 79(3): 568-575.
    • (1992) Blood , vol.79 , Issue.3 , pp. 568-575
    • Kim, H.C.1    McMillan, C.W.2    White, G.C.3    Bergman, G.E.4    Horton, M.W.5    Saidi, P.6
  • 45
    • 0027161439 scopus 로고
    • Reduced coagulation activation following infusion of a highly purified factor IX concentrate compared to a prothrombin complex concentrate
    • Hampton KK, Preston FE, Lowe GD, Walker ID, Sampson B. Reduced coagulation activation following infusion of a highly purified factor IX concentrate compared to a prothrombin complex concentrate. Br J Haematol. 1993; 84(2): 279-284.
    • (1993) Br J Haematol , vol.84 , Issue.2 , pp. 279-284
    • Hampton, K.K.1    Preston, F.E.2    Lowe, G.D.3    Walker, I.D.4    Sampson, B.5
  • 46
    • 0032756734 scopus 로고    scopus 로고
    • The epidemiology of virus transmission by plasma derivatives: Clinical studies verifying the lack of transmission of hepatitis B and C viruses and HIV type 1
    • Tabor E. The epidemiology of virus transmission by plasma derivatives: clinical studies verifying the lack of transmission of hepatitis B and C viruses and HIV type 1. Transfusion. 1999; 39(11-12): 1160-1168.
    • (1999) Transfusion , vol.39 , Issue.11-12 , pp. 1160-1168
    • Tabor, E.1
  • 47
    • 0035108777 scopus 로고    scopus 로고
    • Bovine spongiform encephalopathy and variant Creutzfeldt-Jakob disease: Background, evolution, and current concerns
    • Brown P, Will RG, Bradley R, Asher DM, Detwiler L. Bovine spongiform encephalopathy and variant Creutzfeldt-Jakob disease: background, evolution, and current concerns. Emerg Infect Dis. 2001; 7(1): 6-16.
    • (2001) Emerg Infect Dis , vol.7 , Issue.1 , pp. 6-16
    • Brown, P.1    Will, R.G.2    Bradley, R.3    Asher, D.M.4    Detwiler, L.5
  • 48
    • 0023898771 scopus 로고
    • Human parvovirus infection in haemophiliacs first infused with treated clotting factor concentrates
    • Bartolomei Corsi O, Azzi A, Morfini M, Fanci R, Rossi Ferrini P. Human parvovirus infection in haemophiliacs first infused with treated clotting factor concentrates. J Med Virol. 1988; 25(2): 165-170.
    • (1988) J Med Virol , vol.25 , Issue.2 , pp. 165-170
    • Bartolomei Corsi, O.1    Azzi, A.2    Morfini, M.3    Fanci, R.4    Rossi Ferrini, P.5
  • 49
    • 84979723887 scopus 로고    scopus 로고
    • The safety of plasma-derived versus recombinant concentrates
    • Mannucci PM. The safety of plasma-derived versus recombinant concentrates. Occasional Papers. 2004; (5): 1-4.
    • (2004) Occasional Papers , Issue.5 , pp. 1-4
    • Mannucci, P.M.1
  • 50
    • 79959499738 scopus 로고    scopus 로고
    • A clinical study assessing the pharmacokinetics, efficacy and safety of AlphaNine®, a high-purity factor IX concentrate, in patients with severe haemophilia B
    • Lissitchkov T, Matysiak M, Zavilska K, et al. A clinical study assessing the pharmacokinetics, efficacy and safety of AlphaNine®, a high-purity factor IX concentrate, in patients with severe haemophilia B. Haemophilia. 2011; 17(4): 590-596.
    • (2011) Haemophilia , vol.17 , Issue.4 , pp. 590-596
    • Lissitchkov, T.1    Matysiak, M.2    Zavilska, K.3
  • 51
    • 0029161174 scopus 로고
    • Safety and recovery of Mononine in multiple-dose, high-dose regimens
    • discussion 57-58
    • White GC 2nd. Safety and recovery of Mononine in multiple-dose, high-dose regimens. Acta Haematol. 1995; 94(Suppl 1): 53-57; discussion 57-58.
    • (1995) Acta Haematol , vol.94 , pp. 53-57
    • White, G.C.1
  • 52
    • 2642704209 scopus 로고    scopus 로고
    • Efficacy and safety of continuous infusion of Mononine during five surgical procedures in three hemophilic patients
    • Ménart C, Petit PY, Attali O, Massignon D, Dechavanne M, Négrier C. Efficacy and safety of continuous infusion of Mononine during five surgical procedures in three hemophilic patients. Am J Hematol. 1998; 58(2): 110-116.
    • (1998) Am J Hematol , vol.58 , Issue.2 , pp. 110-116
    • Ménart, C.1    Petit, P.Y.2    Attali, O.3    Massignon, D.4    Dechavanne, M.5    Négrier, C.6
  • 53
    • 0037366699 scopus 로고    scopus 로고
    • Continuous intravenous infusion of a plasma-derived factor IX concentrate (Mononine) in haemophilia B
    • Hoots WK, Leissinger C, Stabler S, et al. Continuous intravenous infusion of a plasma-derived factor IX concentrate (Mononine) in haemophilia B. Haemophilia. 2003; 9(2): 164-172.
    • (2003) Haemophilia , vol.9 , Issue.2 , pp. 164-172
    • Hoots, W.K.1    Leissinger, C.2    Stabler, S.3
  • 54
    • 84916625055 scopus 로고    scopus 로고
    • Pharmacokinetic and pharmacodynamic properties of plasma-derived vs recombinant factor IX in patients with hemophilia B: A prospective crossover study
    • Alamelu J, Bevan D, Sorensen B, Rangarajan S. Pharmacokinetic and pharmacodynamic properties of plasma-derived vs recombinant factor IX in patients with hemophilia B: a prospective crossover study. J Thromb Haemost. 2014; 12(12): 2044-2048.
    • (2014) J Thromb Haemost , vol.12 , Issue.12 , pp. 2044-2048
    • Alamelu, J.1    Bevan, D.2    Sorensen, B.3    Rangarajan, S.4
  • 55
    • 79955141563 scopus 로고    scopus 로고
    • An ultrapure plasma-derived monoclonal antibody-purified factor IX concentrate (Nonafact(R)), results of phase III and IV clinical studies
    • Mauser-Bunschoten EP, Kleine Budde I, Lopaciuk S, et al. An ultrapure plasma-derived monoclonal antibody-purified factor IX concentrate (Nonafact(R)), results of phase III and IV clinical studies. Haemophilia. 2011; 17(3): 439-445.
    • (2011) Haemophilia , vol.17 , Issue.3 , pp. 439-445
    • Mauser-Bunschoten, E.P.1    Kleine Budde, I.2    Lopaciuk, S.3
  • 56
    • 84864280808 scopus 로고    scopus 로고
    • Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: Implications for dosing in prophylaxis
    • Björkman S, Ahlén V. Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis. Eur J Clin Pharmacol. 2012; 68(6): 969-977.
    • (2012) Eur J Clin Pharmacol , vol.68 , Issue.6 , pp. 969-977
    • Björkman, S.1    Ahlén, V.2
  • 58
    • 0019957533 scopus 로고
    • Molecular cloning of the gene for human anti-haemophilic factor IX
    • Choo KH, Gould KG, Rees DJ, Brownlee GG. Molecular cloning of the gene for human anti-haemophilic factor IX. Nature. 1982; 299(5879): 178-180.
    • (1982) Nature , vol.299 , Issue.5879 , pp. 178-180
    • Choo, K.H.1    Gould, K.G.2    Rees, D.J.3    Brownlee, G.G.4
  • 59
    • 0023037823 scopus 로고
    • Expression, purification, and characterization of recombinant gamma-carboxylated factor IX synthesized in Chinese hamster ovary cells
    • Kaufman RJ, Wasley LC, Furie BC, Furie B, Shoemaker CB. Expression, purification, and characterization of recombinant gamma-carboxylated factor IX synthesized in Chinese hamster ovary cells. J Biol Chem. 1986; 261(21): 9622-9628.
    • (1986) J Biol Chem , vol.261 , Issue.21 , pp. 9622-9628
    • Kaufman, R.J.1    Wasley, L.C.2    Furie, B.C.3    Furie, B.4    Shoemaker, C.B.5
  • 60
    • 0035895058 scopus 로고    scopus 로고
    • Human recombinant factor IX: Safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates
    • Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood. 2001; 98(13): 3600-3606.
    • (2001) Blood , vol.98 , Issue.13 , pp. 3600-3606
    • Roth, D.A.1    Kessler, C.M.2    Pasi, K.J.3
  • 61
    • 19944429262 scopus 로고    scopus 로고
    • The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B
    • Shapiro AD, Di Paola J, Cohen A, et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood. 2005; 105(2): 518-525.
    • (2005) Blood , vol.105 , Issue.2 , pp. 518-525
    • Shapiro, A.D.1    Di Paola, J.2    Cohen, A.3
  • 62
    • 84937023941 scopus 로고    scopus 로고
    • EQOFIX: A combined economic and quality-of-life study of hemophilia B treatments in France
    • Polack B, Calvez T, Chambost H, et al. EQOFIX: a combined economic and quality-of-life study of hemophilia B treatments in France. Transfusion. 2015; 55(7): 1787-1797.
    • (2015) Transfusion , vol.55 , Issue.7 , pp. 1787-1797
    • Polack, B.1    Calvez, T.2    Chambost, H.3
  • 63
    • 82755197856 scopus 로고    scopus 로고
    • Strategies for extended serum half-life of protein therapeutics
    • Kontermann RE. Strategies for extended serum half-life of protein therapeutics. Curr Opin Biotechnol. 2011; 22(6): 868-876.
    • (2011) Curr Opin Biotechnol , vol.22 , Issue.6 , pp. 868-876
    • Kontermann, R.E.1
  • 64
    • 84862908041 scopus 로고    scopus 로고
    • Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients
    • Shapiro AD, Ragni MV, Valentino LA, et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood. 2012; 119(3): 666-672.
    • (2012) Blood , vol.119 , Issue.3 , pp. 666-672
    • Shapiro, A.D.1    Ragni, M.V.2    Valentino, L.A.3
  • 65
    • 84889769562 scopus 로고    scopus 로고
    • Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B
    • Powell JS, Pasi KJ, Ragni MV, et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013; 369(24): 2313-2323.
    • (2013) N Engl J Med , vol.369 , Issue.24 , pp. 2313-2323
    • Powell, J.S.1    Pasi, K.J.2    Ragni, M.V.3
  • 66
    • 84915749692 scopus 로고    scopus 로고
    • Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates
    • Powell J, Shapiro A, Ragni M, et al. Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates. Br J Haematol. 2015; 168(1): 113-123.
    • (2015) Br J Haematol , vol.168 , Issue.1 , pp. 113-123
    • Powell, J.1    Shapiro, A.2    Ragni, M.3
  • 68
    • 70449461639 scopus 로고    scopus 로고
    • Genetic fusion to albumin improves the pharmacokinetic properties of factor IX
    • Metzner HJ, Weimer T, Kronthaler U, Lang W, Schulte S. Genetic fusion to albumin improves the pharmacokinetic properties of factor IX. Thromb Haemost. 2009; 102(4): 634-644.
    • (2009) Thromb Haemost , vol.102 , Issue.4 , pp. 634-644
    • Metzner, H.J.1    Weimer, T.2    Kronthaler, U.3    Lang, W.4    Schulte, S.5
  • 69
    • 84865209564 scopus 로고    scopus 로고
    • Improved kinetics of rIX-FP, a recombinant fusion protein linking factor IX with albumin, in cynomolgus monkeys and hemophilia B dogs
    • Nolte MW, Nichols TC, Mueller-Cohrs J, et al. Improved kinetics of rIX-FP, a recombinant fusion protein linking factor IX with albumin, in cynomolgus monkeys and hemophilia B dogs. J Thromb Haemost. 2012; 10(8): 1591-1599.
    • (2012) J Thromb Haemost , vol.10 , Issue.8 , pp. 1591-1599
    • Nolte, M.W.1    Nichols, T.C.2    Mueller-Cohrs, J.3
  • 70
    • 84875522099 scopus 로고    scopus 로고
    • PROLONG-9FP clinical development program-phase I results of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP)
    • Santagostino E. PROLONG-9FP clinical development program-phase I results of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). Thromb Res. 2013; 131(Suppl 2): S7-S10.
    • (2013) Thromb Res , vol.131 , pp. S7-S10
    • Santagostino, E.1
  • 71
    • 84946493489 scopus 로고    scopus 로고
    • Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients
    • Martinowitz U, Lissitchkov T, Lubetsky A, et al. Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients. Haemophilia. 2015; 21(6): 784-790.
    • (2015) Haemophilia , vol.21 , Issue.6 , pp. 784-790
    • Martinowitz, U.1    Lissitchkov, T.2    Lubetsky, A.3
  • 72
    • 84979723011 scopus 로고    scopus 로고
    • Efficacy, pharmacokinetics (PK) and safety results of a phase 3 clinical study of recombinant fusion protein linking coagulation factor IX with albumin (RIX-FP) in previously treated children with hemophilia B
    • Paper presented at, Toronto, Canada
    • Kenet G, Chambost H, Male C, et al. Efficacy, pharmacokinetics (PK) and safety results of a phase 3 clinical study of recombinant fusion protein linking coagulation factor IX with albumin (RIX-FP) in previously treated children with hemophilia B. Paper presented at: International Society of Thrombosis and Haemostasis Meeting; 2015, Toronto, Canada.
    • (2015) International Society of Thrombosis and Haemostasis Meeting
    • Kenet, G.1    Chambost, H.2    Male, C.3
  • 73
    • 84979724265 scopus 로고    scopus 로고
    • Efficacy and safety results of a phase 3 pivotal clinical study of recombinant fusion protein linking coagulation factor IX with albumin (RIX-FP) in previously treated patients with hemophilia B
    • Paper presented at, Toronto, Canada
    • Santagostino E, Martinowitz U, Lissitchkov T, et al. Efficacy and safety results of a phase 3 pivotal clinical study of recombinant fusion protein linking coagulation factor IX with albumin (RIX-FP) in previously treated patients with hemophilia B. Paper presented at: International Society of Thrombosis and Haemostasis Meeting, 2015, Toronto, Canada.
    • (2015) International Society of Thrombosis and Haemostasis Meeting
    • Santagostino, E.1    Martinowitz, U.2    Lissitchkov, T.3
  • 74
    • 84979724053 scopus 로고    scopus 로고
    • Efficacy and safety of recombinant fusion protein linking coagulation factor IX with albumin (RIX-FP) in previously treated patients with hemophilia B undergoing a surgical procedure
    • Paper presented at, June 20-25, Toronto, Canada
    • Negrier C, Lepatan LM, Lubetsky A, et al. Efficacy and safety of recombinant fusion protein linking coagulation factor IX with albumin (RIX-FP) in previously treated patients with hemophilia B undergoing a surgical procedure. Paper presented at: International Society of Thrombosis and Haemostasis Meeting, June 20-25, 2015, Toronto, Canada.
    • (2015) International Society of Thrombosis and Haemostasis Meeting
    • Negrier, C.1    Lepatan, L.M.2    Lubetsky, A.3
  • 75
    • 0344420226 scopus 로고    scopus 로고
    • Pharmacokinetic and biodistribution properties of poly(ethylene glycol)-protein conjugates
    • Caliceti P, Veronese FM. Pharmacokinetic and biodistribution properties of poly(ethylene glycol)-protein conjugates. Adv Drug Deliv Rev. 2003; 55(10): 1261-1277.
    • (2003) Adv Drug Deliv Rev , vol.55 , Issue.10 , pp. 1261-1277
    • Caliceti, P.1    Veronese, F.M.2
  • 76
    • 80052167452 scopus 로고    scopus 로고
    • Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide
    • Østergaard H, Bjelke JR, Hansen L, et al. Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide. Blood. 2011; 118(8): 2333-2341.
    • (2011) Blood , vol.118 , Issue.8 , pp. 2333-2341
    • Østergaard, H.1    Bjelke, J.R.2    Hansen, L.3
  • 77
    • 80052669248 scopus 로고    scopus 로고
    • Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: A first human dose trial in patients with hemophilia B
    • Negrier C, Knobe K, Tiede A, Giangrande P, Møss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011; 118(10): 2695-2701.
    • (2011) Blood , vol.118 , Issue.10 , pp. 2695-2701
    • Negrier, C.1    Knobe, K.2    Tiede, A.3    Giangrande, P.4    Møss, J.5
  • 78
    • 84919491430 scopus 로고    scopus 로고
    • Recombinant long-acting glycoPEGylated factor IX in hemophilia B: A multinational randomized phase 3 trial
    • Collins PW, Young G, Knobe K, et al. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood. 2014; 124(26): 3880-3886.
    • (2014) Blood , vol.124 , Issue.26 , pp. 3880-3886
    • Collins, P.W.1    Young, G.2    Knobe, K.3
  • 79
    • 84880837069 scopus 로고    scopus 로고
    • Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo
    • Kao CY, Yang SJ, Tao MH, Jeng YM, Yu IS, Lin SW. Incorporation of the factor IX Padua mutation into FIX-Triple improves clotting activity in vitro and in vivo. Thromb Haemost. 2013; 110(2): 244-256.
    • (2013) Thromb Haemost , vol.110 , Issue.2 , pp. 244-256
    • Kao, C.Y.1    Yang, S.J.2    Tao, M.H.3    Jeng, Y.M.4    Yu, I.S.5    Lin, S.W.6
  • 80
    • 84883764333 scopus 로고    scopus 로고
    • Glycoengineered factor IX variants with improved pharmacokinetics and subcutaneous efficacy
    • Brooks AR, Sim D, Gritzan U, et al. Glycoengineered factor IX variants with improved pharmacokinetics and subcutaneous efficacy. J Thromb Haemost. 2013; 11(9): 1699-1706.
    • (2013) J Thromb Haemost , vol.11 , Issue.9 , pp. 1699-1706
    • Brooks, A.R.1    Sim, D.2    Gritzan, U.3
  • 81
    • 84930178037 scopus 로고    scopus 로고
    • Selective disruption of heparin and antithrombin-mediated regulation of human factor IX
    • Westmark PR, Tanratana P, Sheehan JP. Selective disruption of heparin and antithrombin-mediated regulation of human factor IX. J Thromb Haemost. 2015; 13(6): 1053-1063.
    • (2015) J Thromb Haemost , vol.13 , Issue.6 , pp. 1053-1063
    • Westmark, P.R.1    Tanratana, P.2    Sheehan, J.P.3
  • 82
    • 84923101508 scopus 로고    scopus 로고
    • Genetic determinants of immunogenicity to factor IX during the treatment of haemophilia B
    • Saini S, Hamasaki-Katagiri N, Pandey GS, et al. Genetic determinants of immunogenicity to factor IX during the treatment of haemophilia B. Haemophilia. 2015; 21(2): 210-218.
    • (2015) Haemophilia , vol.21 , Issue.2 , pp. 210-218
    • Saini, S.1    Hamasaki-Katagiri, N.2    Pandey, G.S.3
  • 83
    • 84890857750 scopus 로고    scopus 로고
    • Prevalent inhibitors in haemophilia B subjects enrolled in the Universal Data Collection database
    • Puetz J, Soucie JM, Kempton CL, Monahan PE, Hemophilia Treatment Center Network I. Prevalent inhibitors in haemophilia B subjects enrolled in the Universal Data Collection database. Haemophilia. 2014; 20(1): 25-31.
    • (2014) Haemophilia , vol.20 , Issue.1 , pp. 25-31
    • Puetz, J.1    Soucie, J.M.2    Kempton, C.L.3    Monahan, P.E.4
  • 84
    • 0030062131 scopus 로고    scopus 로고
    • Prevalence of FIX Inhibitors among patients with hemophilia B: Results of large scale North America survey
    • Katz J. Prevalence of FIX Inhibitors among patients with hemophilia B: results of large scale North America survey. Haemophilia. 1996; 2: 28-31.
    • (1996) Haemophilia , vol.2 , pp. 28-31
    • Katz, J.1
  • 85
    • 34447128837 scopus 로고    scopus 로고
    • Inhibitor development in haemophilia B: An orphan disease in need of attention
    • DiMichele D. Inhibitor development in haemophilia B: an orphan disease in need of attention. Br J Haematol. 2007; 138(3): 305-315.
    • (2007) Br J Haematol , vol.138 , Issue.3 , pp. 305-315
    • DiMichele, D.1
  • 86
  • 87
    • 0026548917 scopus 로고
    • Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs
    • Ehrenforth S, Kreuz W, Scharrer I, et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet. 1992; 339(8793): 594-598.
    • (1992) Lancet , vol.339 , Issue.8793 , pp. 594-598
    • Ehrenforth, S.1    Kreuz, W.2    Scharrer, I.3
  • 88
    • 79955139784 scopus 로고    scopus 로고
    • A retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia B
    • Recht M, Pollmann H, Tagliaferri A, Musso R, Janco R, Neuman WR. A retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia B. Haemophilia. 2011; 17(3): 494-499.
    • (2011) Haemophilia , vol.17 , Issue.3 , pp. 494-499
    • Recht, M.1    Pollmann, H.2    Tagliaferri, A.3    Musso, R.4    Janco, R.5    Neuman, W.R.6
  • 89
    • 65449139202 scopus 로고    scopus 로고
    • Health status and quality of life of elderly persons with severe hemophilia born before the advent of modern replacement therapy
    • Siboni SM, Mannucci PM, Gringeri A, et al. Health status and quality of life of elderly persons with severe hemophilia born before the advent of modern replacement therapy. J Thromb Haemost. 2009; 7(5): 780-786.
    • (2009) J Thromb Haemost , vol.7 , Issue.5 , pp. 780-786
    • Siboni, S.M.1    Mannucci, P.M.2    Gringeri, A.3
  • 90
    • 33845938292 scopus 로고    scopus 로고
    • PEGylated proteins: Evaluation of their safety in the absence of definitive metabolism studies
    • Webster R, Didier E, Harris P, et al. PEGylated proteins: evaluation of their safety in the absence of definitive metabolism studies. Drug Metab Dispos. 2007; 35(1): 9-16.
    • (2007) Drug Metab Dispos , vol.35 , Issue.1 , pp. 9-16
    • Webster, R.1    Didier, E.2    Harris, P.3
  • 91
    • 84898821672 scopus 로고    scopus 로고
    • Biodistribution of the recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in rats
    • Herzog E, Harris S, Henson C, et al. Biodistribution of the recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in rats. Thromb Res. 2014; 133(5): 900-907.
    • (2014) Thromb Res , vol.133 , Issue.5 , pp. 900-907
    • Herzog, E.1    Harris, S.2    Henson, C.3
  • 92
    • 80052339245 scopus 로고    scopus 로고
    • Haemophilia B: Impact on patients and economic burden of disease
    • Gater A, Thomson TA, Strandberg-Larsen M. Haemophilia B: impact on patients and economic burden of disease. Thromb Haemost. 2011; 106(3): 398-404.
    • (2011) Thromb Haemost , vol.106 , Issue.3 , pp. 398-404
    • Gater, A.1    Thomson, T.A.2    Strandberg-Larsen, M.3
  • 93
    • 84862499521 scopus 로고    scopus 로고
    • Hemostatic effect of a monoclonal antibody mAb 2021 blocking the interaction between FXa and TFPI in a rabbit hemophilia model
    • Hilden I, Lauritzen B, Sørensen BB, et al. Hemostatic effect of a monoclonal antibody mAb 2021 blocking the interaction between FXa and TFPI in a rabbit hemophilia model. Blood. 2012; 119(24): 5871-5878.
    • (2012) Blood , vol.119 , Issue.24 , pp. 5871-5878
    • Hilden, I.1    Lauritzen, B.2    Sørensen, B.B.3
  • 94
    • 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. 2015; 13(5): 743-754.
    • (2015) J Thromb Haemost , vol.13 , Issue.5 , pp. 743-754
    • Chowdary, P.1    Lethagen, S.2    Friedrich, U.3
  • 95
    • 84937764228 scopus 로고    scopus 로고
    • An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
    • Sehgal A, Barros S, Ivanciu L, et al. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med. 2015; 21(5): 492-497.
    • (2015) Nat Med , vol.21 , Issue.5 , pp. 492-497
    • Sehgal, A.1    Barros, S.2    Ivanciu, L.3
  • 96
    • 84978795568 scopus 로고    scopus 로고
    • A subcutaneously administered investigational RNAi therapeutic (ALN-AT3) targeting antithrombin for treatment of hemophilia: Interim weekly and monthly dosing results in patients with hemophilia A or B
    • Pasi KJ, Georgiev P, Mant T, et al. A subcutaneously administered investigational RNAi therapeutic (ALN-AT3) targeting antithrombin for treatment of hemophilia: interim weekly and monthly dosing results in patients with hemophilia A or B. Blood. 2015; 126: 551.
    • (2015) Blood , vol.126 , pp. 551
    • Pasi, K.J.1    Georgiev, P.2    Mant, T.3


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