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Volumn 369, Issue 24, 2013, Pages 2313-2323

Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B

(28)  Powell, Jerry S a   Pasi, K John b   Ragni, Margaret V c   Ozelo, Margareth C d   Valentino, Leonard A e   Mahlangu, Johnny N f   Josephson, Neil C h   Perry, David i   Manco Johnson, Marilyn J j   Apte, Shashikant k   Baker, Ross I l   Chan, Godfrey C m   Novitzky, Nicolas g   Wong, Raymond S n   Krassova, Snejana o   Allen, Geoffrey o   Jiang, Haiyan o   Innes, Alison o   Li, Shuanglian o   Cristiano, Lynda M o   more..


Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 9; FC RECOMBINANT FACTOR IX FUSION PROTEIN; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG;

EID: 84889769562     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1305074     Document Type: Article
Times cited : (297)

References (40)
  • 2
    • 0035822038 scopus 로고    scopus 로고
    • The hemophilias - From royal genes to gene therapy
    • Erratum, N Engl J Med 2001;345:384
    • Mannucci PM, Tuddenham EG. The hemophilias - from royal genes to gene therapy. N Engl J Med 2001;344:1773-9. [Erratum, N Engl J Med 2001;345:384.]
    • (2001) N Engl J Med , vol.344 , pp. 1773-1779
    • Mannucci, P.M.1    Tuddenham, E.G.2
  • 3
    • 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;232:25-32.
    • (1992) J Intern Med , vol.232 , pp. 25-32
    • Nilsson, I.M.1    Berntorp, E.2    Löfqvist, T.3    Pettersson, H.4
  • 4
    • 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;357:535-44.
    • (2007) N Engl J Med , vol.357 , pp. 535-544
    • Manco-Johnson, M.J.1    Abshire, T.C.2    Shapiro, A.D.3
  • 5
    • 0028004222 scopus 로고
    • A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs
    • Aledort LM, Haschmeyer RH, Pettersson H. A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. J Intern Med 1994;236:391-9.
    • (1994) J Intern Med , vol.236 , pp. 391-399
    • Aledort, L.M.1    Haschmeyer, R.H.2    Pettersson, H.3
  • 6
    • 0038441408 scopus 로고    scopus 로고
    • The overall effectiveness of prophylaxis in severe haemophilia
    • Panicker J, Warrier I, Thomas R, Lusher JM. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia 2003;9:272-8.
    • (2003) Haemophilia , vol.9 , pp. 272-278
    • Panicker, J.1    Warrier, I.2    Thomas, R.3    Lusher, J.M.4
  • 9
    • 85031074883 scopus 로고    scopus 로고
    • MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding)
    • National Hemophilia Foundation. MASAC recommendation concerning prophylaxis (regular administration of clotting factor concentrate to prevent bleeding). Medical and Scientific Advisory Council document #179 (http://www.hemophilia.org/NHFWeb/Resource/StaticPages/menu0/menu5/menu57/ masac179.pdf).
    • Medical and Scientific Advisory Council Document #179
  • 10
    • 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. Hemophilia 2001;7:392-6.
    • (2001) Hemophilia , vol.7 , pp. 392-396
    • Hacker, M.R.1    Geraghty, S.2    Manco-Johnson, M.3
  • 13
    • 84868204378 scopus 로고    scopus 로고
    • Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: Repeat pharmacokinetic evaluation and sialylation analysis
    • Martinowitz U, Shapiro A, Quon DV, et al. Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysis. Haemophilia 2012;18:881-7.
    • (2012) Haemophilia , vol.18 , pp. 881-887
    • Martinowitz, U.1    Shapiro, A.2    Quon, D.V.3
  • 14
    • 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;118:2695-701.
    • (2011) Blood , vol.118 , pp. 2695-2701
    • Negrier, C.1    Knobe, K.2    Tiede, A.3    Giangrande, P.4    Møss, J.5
  • 15
    • 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;115:2057-64.
    • (2010) Blood , vol.115 , pp. 2057-2064
    • Peters, R.T.1    Low, S.C.2    Kamphaus, G.D.3
  • 16
    • 84866594053 scopus 로고    scopus 로고
    • Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients
    • Santagostino E, Negrier C, Klamroth R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood 2012;120:2405-11.
    • (2012) Blood , vol.120 , pp. 2405-2411
    • Santagostino, E.1    Negrier, C.2    Klamroth, R.3
  • 17
    • 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. Blood2012;119:666-72.
    • (2012) Blood , vol.119 , pp. 666-672
    • Shapiro, A.D.1    Ragni, M.V.2    Valentino, L.A.3
  • 18
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006;12:342-7.
    • (2006) Nat Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 19
    • 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;365:2357-65.
    • (2011) N Engl J Med , vol.365 , pp. 2357-2365
    • Nathwani, A.C.1    Tuddenham, E.G.2    Rangarajan, S.3
  • 20
    • 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;7:715-25.
    • (2007) Nat Rev Immunol , vol.7 , pp. 715-725
    • Roopenian, D.C.1    Akilesh, S.2
  • 21
    • 84889806818 scopus 로고    scopus 로고
    • Fc-fusion proteins and FcRn: Structural insights for longer lasting and more effective therapeutics
    • October 24 (Epub ahead of print).
    • Rath T, Baker K, Dumont JA, et al. Fc-fusion proteins and FcRn: structural insights for longer lasting and more effective therapeutics. Crit Rev Biotechnol 2013 October 24 (Epub ahead of print).
    • (2013) Crit Rev Biotechnol
    • Rath, T.1    Baker, K.2    Dumont, J.A.3
  • 22
    • 60549104490 scopus 로고    scopus 로고
    • Committee for Medicinal Products for Human Use (CHMP) London: European Medicines Agency
    • Committee for Medicinal Products for Human Use (CHMP). Guideline on immunogenicity assessment of biotechnology-derived therapeutic proteins. London: European Medicines Agency, 2007 (http://www.ema.europa.eu/docs/en-GB/document- library/Scientific-guideline/2009/09/WC500003946.pdf).
    • (2007) Guideline on Immunogenicity Assessment of Biotechnology-derived Therapeutic Proteins
  • 24
    • 0001072895 scopus 로고
    • The use of confidence or fiducial limits illustrated in the case of the binomial
    • Clopper C, Pearson ES. The use of confidence or fiducial limits illustrated in the case of the binomial. Biometrika 1934;26:404-13.
    • (1934) Biometrika , vol.26 , pp. 404-413
    • Clopper, C.1    Pearson, E.S.2
  • 26
    • 0038779279 scopus 로고    scopus 로고
    • Prophylaxis in factor IX deficiency product and patient variation
    • Kisker CT, Eisberg A, Schwartz B. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia 2003;9:279-84.
    • (2003) Haemophilia , vol.9 , pp. 279-284
    • Kisker, C.T.1    Eisberg, A.2    Schwartz, B.3
  • 27
    • 34248532042 scopus 로고    scopus 로고
    • Reformulated BeneFix: Efficacy and safety in previously treated patients with moderately severe to severe haemophilia B
    • Erratum, Haemophilia 2007;13:450
    • Lambert T, Recht M, Valentino LA, et al. Reformulated BeneFix: efficacy and safety in previously treated patients with moderately severe to severe haemophilia B. Haemophilia 2007;13:233-43. [Erratum, Haemophilia 2007;13:450.]
    • (2007) Haemophilia , vol.13 , pp. 233-243
    • Lambert, T.1    Recht, M.2    Valentino, L.A.3
  • 28
    • 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;42:190-7.
    • (2002) Transfusion , vol.42 , pp. 190-197
    • Ewenstein, B.M.1    Joist, J.H.2    Shapiro, A.D.3
  • 30
    • 34248659669 scopus 로고    scopus 로고
    • Pharmacokinetic study of recombinant human factor IX in previously treated patients with hemophilia B in Taiwan
    • Chang HH, Yang YL, Hung MH, Tsay W, Shen MC. Pharmacokinetic study of recombinant human factor IX in previously treated patients with hemophilia B in Taiwan. J Formos Med Assoc 2007;106:281-7.
    • (2007) J Formos Med Assoc , vol.106 , pp. 281-287
    • Chang, H.H.1    Yang, Y.L.2    Hung, M.H.3    Tsay, W.4    Shen, M.C.5
  • 31
    • 84883055784 scopus 로고    scopus 로고
    • Head-to-head comparison of the pharmacokinetic profiles of a high-purity factor IX concentrate (AlphaNine) and a recombinant factor IX (BeneFIX) in patients with severe haemophilia B
    • Lissitchkov T, Matysiak M, Zavilska K, et al. Head-to-head comparison of the pharmacokinetic profiles of a high-purity factor IX concentrate (AlphaNine) and a recombinant factor IX (BeneFIX) in patients with severe haemophilia B. Haemophilia 2013;19:674-8.
    • (2013) Haemophilia , vol.19 , pp. 674-678
    • Lissitchkov, T.1    Matysiak, M.2    Zavilska, K.3
  • 32
    • 0036588771 scopus 로고    scopus 로고
    • Inhibitors: Resolving diagnostic and therapeutic dilemmas
    • Dimichele D. Inhibitors: resolving diagnostic and therapeutic dilemmas. Haemophilia 2002;8:280-7.
    • (2002) Haemophilia , vol.8 , pp. 280-287
    • Dimichele, D.1
  • 33
    • 69949092766 scopus 로고    scopus 로고
    • Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006)
    • Chitlur M, Warrier I, Rajpurkar M, Lusher JM. Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006). Haemophilia 2009;15:1027-31.
    • (2009) Haemophilia , vol.15 , pp. 1027-1031
    • Chitlur, M.1    Warrier, I.2    Rajpurkar, M.3    Lusher, J.M.4
  • 34
    • 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;17:494-9.
    • (2011) Haemophilia , vol.17 , pp. 494-499
    • Recht, M.1    Pollmann, H.2    Tagliaferri, A.3    Musso, R.4    Janco, R.5    Neuman, W.R.6
  • 35
    • 84863332257 scopus 로고    scopus 로고
    • Treatment of inhibitors in hemophilia B
    • Lee CA, Berntorp E, Hoots WK, eds. 2nd ed. Hoboken, NJ: Wiley-Blackwell
    • Tandra A, Shapiro AD. Treatment of inhibitors in hemophilia B. In: Lee CA, Berntorp E, Hoots WK, eds. Textbook of hemophilia. 2nd ed. Hoboken, NJ: Wiley-Blackwell, 2010:97-103.
    • (2010) Textbook of Hemophilia , pp. 97-103
    • Tandra, A.1    Shapiro, A.D.2
  • 36
    • 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;138:305-15.
    • (2007) Br J Haematol , vol.138 , pp. 305-315
    • DiMichele, D.1
  • 37
    • 84871012263 scopus 로고    scopus 로고
    • Antibody formation and specificity in Bethesda-negative brother pairs with haemophilia A
    • Klintman J, Hillarp A, Donfield S, Berntorp E, Astermark J. Antibody formation and specificity in Bethesda-negative brother pairs with haemophilia A. Haemophilia 2013;19:106-12.
    • (2013) Haemophilia , vol.19 , pp. 106-112
    • Klintman, J.1    Hillarp, A.2    Donfield, S.3    Berntorp, E.4    Astermark, J.5
  • 38
    • 84873583409 scopus 로고    scopus 로고
    • Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients
    • Whelan SF, Hofbauer CJ, Horling FM, et al. Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients. Blood 2013;121:1039-48.
    • (2013) Blood , vol.121 , pp. 1039-1048
    • Whelan, S.F.1    Hofbauer, C.J.2    Horling, F.M.3
  • 40


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