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Volumn 2016, Issue 1, 2016, Pages 650-656

New therapies for hemophilia

Author keywords

[No Author keywords available]

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT FACTOR VIII FC FUSION PROTEIN; UNCLASSIFIED DRUG; VON WILLEBRAND FACTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; F8 PROTEIN, HUMAN;

EID: 85020240344     PISSN: 15204391     EISSN: 15204383     Source Type: Journal    
DOI: 10.1182/asheducation-2016.1.650     Document Type: Article
Times cited : (26)

References (43)
  • 1
    • 84930268161 scopus 로고    scopus 로고
    • The current status of prophylactic replacement therapy in children and adults with haemophilia
    • Ljung R, Gretenkort Andersson N. The current status of prophylactic replacement therapy in children and adults with haemophilia. Br J Haematol. 2015; 169(6): 777-786.
    • (2015) Br J Haematol , vol.169 , Issue.6 , pp. 777-786
    • Ljung, R.1    Gretenkort Andersson, N.2
  • 2
    • 56649088061 scopus 로고    scopus 로고
    • Secondary prophylaxis in adolescent and adult haemophiliacs
    • Tagliaferri A, Di Perna C, Rivolta GF. Secondary prophylaxis in adolescent and adult haemophiliacs. Blood Transfus. 2008; 6(Suppl. 2): s17-s20.
    • (2008) Blood Transfus , vol.6 , pp. s17-s20
    • Tagliaferri, A.1    Di Perna, C.2    Rivolta, G.F.3
  • 3
    • 84975796441 scopus 로고    scopus 로고
    • Men with severe hemophilia in the United States: Birth cohort analysis of a large national database
    • Mazepa MA, Monahan PE, Baker JR, Riske BK, Soucie JM; US Hemophilia Treatment Center Network. Men with severe hemophilia in the United States: birth cohort analysis of a large national database. Blood. 2016; 127(24): 3073-3081.
    • (2016) Blood , vol.127 , Issue.24 , pp. 3073-3081
    • Mazepa, M.A.1    Monahan, P.E.2    Baker, J.R.3    Riske, B.K.4    Soucie, J.M.5
  • 4
    • 84926139661 scopus 로고    scopus 로고
    • Optimal treatment strategies for hemophilia: Achievements and limitations of current prophylactic regimens
    • Oldenburg J. Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens. Blood. 2015; 125(13): 2038-2044.
    • (2015) Blood , vol.125 , Issue.13 , pp. 2038-2044
    • Oldenburg, J.1
  • 5
    • 59849093369 scopus 로고    scopus 로고
    • Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A
    • Collins PW, Blanchette VS, Fischer K, et al.; rAHF-PFM Study Group. Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A. J Thromb Haemost. 2009; 7(3): 413-420.
    • (2009) J Thromb Haemost , vol.7 , Issue.3 , pp. 413-420
    • Collins, P.W.1    Blanchette, V.S.2    Fischer, K.3
  • 6
    • 84946710809 scopus 로고    scopus 로고
    • Targeting factor replacement therapy in severe hemophilia: Which level is important?
    • Fischer K, Berntorp E. Targeting factor replacement therapy in severe hemophilia: which level is important? Semin Thromb Hemost. 2015; 41(8): 860-863.
    • (2015) Semin Thromb Hemost. , vol.41 , Issue.8 , pp. 860-863
    • Fischer, K.1    Berntorp, E.2
  • 7
    • 84900839290 scopus 로고    scopus 로고
    • Factor VIII therapy for hemophilia A: Current and future issues
    • Aledort L, Ljung R, Mann K, Pipe S. Factor VIII therapy for hemophilia A: current and future issues. Expert Rev Hematol. 2014; 7(3): 373-385.
    • (2014) Expert Rev Hematol. , vol.7 , Issue.3 , pp. 373-385
    • Aledort, L.1    Ljung, R.2    Mann, K.3    Pipe, S.4
  • 8
    • 84890321123 scopus 로고    scopus 로고
    • The first recombinant FVIII produced in human cells - An update on its clinical development programme
    • Valentino LA, Negrier C, Kohla G, et al. The first recombinant FVIII produced in human cells - an update on its clinical development programme. Haemophilia. 2014; 20(Suppl. 1): 1-9.
    • (2014) Haemophilia , vol.20 , pp. 1-9
    • Valentino, L.A.1    Negrier, C.2    Kohla, G.3
  • 9
    • 84954488034 scopus 로고    scopus 로고
    • Changing paradigm of hemophilia management: Extended half-life factor concentrates and gene therapy
    • Kumar R, Dunn A, Carcao M. Changing paradigm of hemophilia management: extended half-life factor concentrates and gene therapy. Semin Thromb Hemost. 2016; 42(1): 18-29.
    • (2016) Semin Thromb Hemost. , vol.42 , Issue.1 , pp. 18-29
    • Kumar, R.1    Dunn, A.2    Carcao, M.3
  • 10
    • 84976870484 scopus 로고    scopus 로고
    • Prophylaxis for hemophilia in the era of extended half-life factor VIII/factor IX products
    • Berntorp E, Andersson NG. Prophylaxis for hemophilia in the era of extended half-life factor VIII/factor IX products. Semin Thromb Hemost. 2016; 42(5): 518-525.
    • (2016) Semin Thromb Hemost. , vol.42 , Issue.5 , pp. 518-525
    • Berntorp, E.1    Andersson, N.G.2
  • 11
    • 84958580623 scopus 로고    scopus 로고
    • The past and future of haemophilia: Diagnosis, treatments, and its complications
    • (10040)
    • Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016; 388(10040): 187-197.
    • (2016) Lancet , vol.388 , pp. 187-197
    • Peyvandi, F.1    Garagiola, I.2    Young, G.3
  • 12
    • 84892526766 scopus 로고    scopus 로고
    • Molecular approaches for improved clotting factors for hemophilia
    • Kaufman RJ, Powell JS. Molecular approaches for improved clotting factors for hemophilia. Blood. 2013; 122(22): 3568-3574.
    • (2013) Blood , vol.122 , Issue.22 , pp. 3568-3574
    • Kaufman, R.J.1    Powell, J.S.2
  • 13
    • 84957536370 scopus 로고    scopus 로고
    • Factor VIII/factor IX prophylaxis for severe hemophilia
    • Carcao M, Srivastava A. Factor VIII/factor IX prophylaxis for severe hemophilia. Semin Hematol. 2016; 53(1): 3-9.
    • (2016) Semin Hematol. , vol.53 , Issue.1 , pp. 3-9
    • Carcao, M.1    Srivastava, A.2
  • 14
    • 84959046658 scopus 로고    scopus 로고
    • Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A
    • Nolan B, Mahlangu J, Perry D, et al. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016; 22(1): 72-80.
    • (2016) Haemophilia , vol.22 , Issue.1 , pp. 72-80
    • Nolan, B.1    Mahlangu, J.2    Perry, D.3
  • 16
    • 84857951468 scopus 로고    scopus 로고
    • Clinical severity of haemophilia A: Does the classification of the 1950s still stand?
    • Den Uijl IE, Mauser Bunschoten EP, Roosendaal G, et al. Clinical severity of haemophilia A: does the classification of the 1950s still stand? Haemophilia. 2011; 17(6): 849-853.
    • (2011) Haemophilia , vol.17 , Issue.6 , pp. 849-853
    • Den Uijl, I.E.1    Mauser Bunschoten, E.P.2    Roosendaal, G.3
  • 17
    • 84946731812 scopus 로고    scopus 로고
    • N8-GP - A new long-acting factor VIII for the treatment of patients with haemophilia A
    • Ehrenforth S. N8-GP - a new long-acting factor VIII for the treatment of patients with haemophilia A. Haemophilia. 2014; 20(suppl 3): 188.
    • (2014) Haemophilia , vol.20 , pp. 188
    • Ehrenforth, S.1
  • 18
    • 84960333582 scopus 로고    scopus 로고
    • Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4)
    • Young G, Collins PW, Colberg T, et al. Nonacog beta pegol (N9-GP) in haemophilia B: a multinational phase III safety and efficacy extension trial (paradigm™4). Thromb Res. 2016; 141: 69-76.
    • (2016) Thromb Res. , vol.141 , pp. 69-76
    • Young, G.1    Collins, P.W.2    Colberg, T.3
  • 19
    • 84963542247 scopus 로고    scopus 로고
    • Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: Results of a phase 3 trial
    • Santagostino E, Martinowitz U, Lissitchkov T, et al.; PROLONG-9FP Investigators Study Group. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016; 127(14): 1761-1769.
    • (2016) Blood , vol.127 , Issue.14 , pp. 1761-1769
    • Santagostino, E.1    Martinowitz, U.2    Lissitchkov, T.3
  • 20
    • 84983509569 scopus 로고    scopus 로고
    • Analysis of bleeding rates in pediatric subjects with target joints and severe hemophilia A receiving prophylaxis with recombinant factor VIII Fc fusion protein (rFVIIIFC) in kids A-long
    • Nolan B, Young G, Rangarajan S, et al. Analysis of bleeding rates in pediatric subjects with target joints and severe hemophilia A receiving prophylaxis with recombinant factor VIII Fc fusion protein (rFVIIIFC) in Kids A-Long. J Thromb Haemost. 2015; 13(S2): 347.
    • (2015) J Thromb Haemost , vol.13 , Issue.S2 , pp. 347
    • Nolan, B.1    Young, G.2    Rangarajan, S.3
  • 21
    • 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
  • 22
    • 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.; A-LONG Investigators. 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
  • 23
    • 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.; B-LONG Investigators. 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
  • 24
    • 84930179981 scopus 로고    scopus 로고
    • Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A
    • Young G, Mahlangu J, Kulkarni R, et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015; 13(6): 967-977.
    • (2015) J Thromb Haemost , vol.13 , Issue.6 , pp. 967-977
    • Young, G.1    Mahlangu, J.2    Kulkarni, R.3
  • 25
    • 84942546645 scopus 로고    scopus 로고
    • Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A
    • Konkle BA, Stasyshyn O, Chowdary P, et al. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015; 126(9): 1078-1085.
    • (2015) Blood , vol.126 , Issue.9 , pp. 1078-1085
    • Konkle, B.A.1    Stasyshyn, O.2    Chowdary, P.3
  • 26
    • 84857572936 scopus 로고    scopus 로고
    • A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management
    • Valentino LA, Mamonov V, Hellmann A, et al.; Prophylaxis Study Group. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. J Thromb Haemost. 2012; 10(3): 359-367.
    • (2012) J Thromb Haemost , vol.10 , Issue.3 , pp. 359-367
    • Valentino, L.A.1    Mamonov, V.2    Hellmann, A.3
  • 27
    • 79951472629 scopus 로고    scopus 로고
    • Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia
    • Collins PW, Fischer K, Morfini M, Blanchette VS, Bjorkman S. Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia. 2011; 17(1): 2-10.
    • (2011) Haemophilia , vol.17 , Issue.1 , pp. 2-10
    • Collins, P.W.1    Fischer, K.2    Morfini, M.3    Blanchette, V.S.4    Bjorkman, S.5
  • 28
    • 77954851703 scopus 로고    scopus 로고
    • Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A
    • Björkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia. 2010; 16(4): 597-605.
    • (2010) Haemophilia , vol.16 , Issue.4 , pp. 597-605
    • Björkman, S.1
  • 29
    • 0019227125 scopus 로고
    • Haptens bound to self IgG induce immunologic tolerance, while when coupled to syngeneic spleen cells they induce immune suppression
    • Borel Y. Haptens bound to self IgG induce immunologic tolerance, while when coupled to syngeneic spleen cells they induce immune suppression. Immunol Rev. 1980; 50: 71-104.
    • (1980) Immunol Rev. , vol.50 , pp. 71-104
    • Borel, Y.1
  • 30
    • 0029995883 scopus 로고    scopus 로고
    • Epitope-specific tolerance induction with an engineered immunoglobulin
    • Zambidis ET, Scott DW. Epitope-specific tolerance induction with an engineered immunoglobulin. Proc Natl Acad Sci USA. 1996; 93(10): 5019-5024.
    • (1996) Proc Natl Acad Sci USA , vol.93 , Issue.10 , pp. 5019-5024
    • Zambidis, E.T.1    Scott, D.W.2
  • 31
    • 38349191376 scopus 로고    scopus 로고
    • Expansion of CD4+CD25+ regulatory T cells by intravenous immunoglobulin: A critical factor in controlling experimental autoimmune encephalomyelitis
    • Ephrem A, Chamat S, Miquel C, et al. Expansion of CD4+CD25+ regulatory T cells by intravenous immunoglobulin: a critical factor in controlling experimental autoimmune encephalomyelitis. Blood. 2008; 111(2): 715-722.
    • (2008) Blood , vol.111 , Issue.2 , pp. 715-722
    • Ephrem, A.1    Chamat, S.2    Miquel, C.3
  • 32
    • 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
  • 33
    • 84887986948 scopus 로고    scopus 로고
    • Do tregitopes have the potential to impact the current treatment landscape of autoimmune diseases?
    • De Groot AS. Do Tregitopes have the potential to impact the current treatment landscape of autoimmune diseases? Expert Rev Clin Immunol. 2013; 9(12): 1155-1157.
    • (2013) Expert Rev Clin Immunol. , vol.9 , Issue.12 , pp. 1155-1157
    • De Groot, A.S.1
  • 35
    • 84905090765 scopus 로고    scopus 로고
    • Fc glycan-modulated immunoglobulin G effector functions
    • Quast I, Lünemann JD. Fc glycan-modulated immunoglobulin G effector functions. J Clin Immunol. 2014; 34(Suppl. 1): S51-S55.
    • (2014) J Clin Immunol. , vol.34 , pp. S51-S55
    • Quast, I.1    Lünemann, J.D.2
  • 36
    • 84923107017 scopus 로고    scopus 로고
    • Regulation of immune responses to protein therapeutics by transplacental induction of T cell tolerance
    • Gupta N, Culina S, Meslier Y, et al. Regulation of immune responses to protein therapeutics by transplacental induction of T cell tolerance. Sci Transl Med. 2015; 7(275): 275ra21.
    • (2015) Sci Transl Med. , vol.7 , Issue.275 , pp. 275ra21
    • Gupta, N.1    Culina, S.2    Meslier, Y.3
  • 37
    • 84961230835 scopus 로고    scopus 로고
    • Reduction of factor VIII inhibitor titers during immune tolerance induction with recombinant factor VIII-Fc fusion protein
    • Groomes CL, Gianferante DM, Crouch GD, Parekh DS, Scott DW, Lieuw K. Reduction of factor VIII inhibitor titers during immune tolerance induction with recombinant factor VIII-Fc fusion protein. Pediatr Blood Cancer. 2016; 63(5): 922-924.
    • (2016) Pediatr Blood Cancer , vol.63 , Issue.5 , pp. 922-924
    • Groomes, C.L.1    Gianferante, D.M.2    Crouch, G.D.3    Parekh, D.S.4    Scott, D.W.5    Lieuw, K.6
  • 38
    • 85020243994 scopus 로고    scopus 로고
    • Immune tolerance induction using rFVIIIFc (Eloctate) [abstract]
    • 3531
    • Malec LM, Ragni MV, Journeycake JM, Alabek M. Immune tolerance induction using rFVIIIFc (Eloctate) [abstract]. Blood. 2015; 126(23). Abstract 3531.
    • (2015) Blood , vol.126 , Issue.23
    • Malec, L.M.1    Ragni, M.V.2    Journeycake, J.M.3    Alabek, M.4
  • 39
    • 84971222569 scopus 로고    scopus 로고
    • A randomized trial of factor VIII and neutralizing antibodies in hemophilia A
    • Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016; 374(21): 2054-2064.
    • (2016) N Engl J Med. , vol.374 , Issue.21 , pp. 2054-2064
    • Peyvandi, F.1    Mannucci, P.M.2    Garagiola, I.3
  • 40
    • 85010953465 scopus 로고    scopus 로고
    • Emerging genetic and pharmacologic therapies for controlling hemostasis: Beyond recombinant clotting factors
    • Monahan PE Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors. Hematology Am Soc Hematol Educ Program. 2015; 2015: 33-40.
    • (2015) Hematology Am Soc Hematol Educ Program , vol.2015 , pp. 33-40
    • Monahan, P.E.1
  • 41
    • 84966669624 scopus 로고    scopus 로고
    • The future of hemophilia treatment: Longer-acting factor concentrates versus gene therapy
    • Giangrande P. The future of hemophilia treatment: longer-acting factor concentrates versus gene therapy. Semin Thromb Hemost. 2016; 42(5): 513-517.
    • (2016) Semin Thromb Hemost. , vol.42 , Issue.5 , pp. 513-517
    • Giangrande, P.1
  • 42
    • 84977137707 scopus 로고    scopus 로고
    • Current and future prospects for hemophilia gene therapy
    • Ward P, Walsh CE. Current and future prospects for hemophilia gene therapy. Expert Rev Hematol. 2016; 9(7): 649-659.
    • (2016) Expert Rev Hematol. , vol.9 , Issue.7 , pp. 649-659
    • Ward, P.1    Walsh, C.E.2
  • 43
    • 85020249454 scopus 로고    scopus 로고
    • Impact Education, LLC, National Hemophilia Foundation, Postgraduate Institute for Medicine Accessed 20 September, 2016
    • Impact Education, LLC, National Hemophilia Foundation, Postgraduate Institute for Medicine. Factor VIII and IX therapeutic agents recently approved or in late phase clinical development. Available at: www.managedcarehemo.com. Accessed 20 September, 2016.
    • Factor VIII and IX Therapeutic Agents Recently Approved or in Late Phase Clinical Development


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