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Volumn 42, Issue 1, 2016, Pages 18-29

Changing Paradigm of Hemophilia Management: Extended Half-Life Factor Concentrates and Gene Therapy

Author keywords

gene therapy; hemophilia; prophylaxis

Indexed keywords

ALBUMIN; ANTITHROMBIN; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR CONCENTRATE; HYBRID PROTEIN; IMMUNOGLOBULIN FC FRAGMENT; IMMUNOGLOBULIN G1; MACROGOL; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY ACE910; PARVOVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; TISSUE FACTOR PATHWAY INHIBITOR; UNCLASSIFIED DRUG; BLOOD CLOTTING FACTOR;

EID: 84954488034     PISSN: 00946176     EISSN: 10989064     Source Type: Journal    
DOI: 10.1055/s-0035-1568877     Document Type: Article
Times cited : (21)

References (113)
  • 2
    • 73949126440 scopus 로고    scopus 로고
    • A study of variations in the reported haemophilia A prevalence around the world
    • Stonebraker J. S., Bolton-Maggs P. H., Soucie J. M., Walker I., Brooker M. A study of variations in the reported haemophilia A prevalence around the world. Haemophilia: 2010; 16 1 20 32
    • (2010) Haemophilia , vol.16 , Issue.1 , pp. 20-32
    • Stonebraker, J.S.1    Bolton-Maggs, P.H.2    Soucie, J.M.3    Walker, I.4    Brooker, M.5
  • 3
    • 84887528087 scopus 로고    scopus 로고
    • Inherited abnormalities of coagulation: hemophilia, von Willebrand disease, and beyond
    • Kumar R., Carcao M. Inherited abnormalities of coagulation: hemophilia, von Willebrand disease, and beyond. Pediatr Clin North Am: 2013; 60 6 1419 1441
    • (2013) Pediatr Clin North Am , vol.60 , Issue.6 , pp. 1419-1441
    • Kumar, R.1    Carcao, M.2
  • 4
    • 84859596442 scopus 로고    scopus 로고
    • Modern haemophilia care
    • Berntorp E., Shapiro A. D. Modern haemophilia care. Lancet: 2012; 379 9824 1447 1456
    • (2012) Lancet , vol.379 , Issue.9824 , pp. 1447-1456
    • Berntorp, E.1    Shapiro, A.D.2
  • 5
    • 84874028913 scopus 로고    scopus 로고
    • History of prophylaxis
    • Berntorp E. History of prophylaxis. Haemophilia: 2013; 19 2 163 165
    • (2013) Haemophilia , vol.19 , Issue.2 , pp. 163-165
    • Berntorp, E.1
  • 6
    • 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
  • 7
    • 0013836217 scopus 로고
    • Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B
    • Ahlberg A. Haemophilia in Sweden. VII. Incidence, treatment and prophylaxis of arthropathy and other musculo-skeletal manifestations of haemophilia A and B. Acta Orthop Scand Suppl: 1965; 77 3 132
    • (1965) Acta Orthop Scand Suppl , vol.77 , pp. 3-132
    • Ahlberg, A.1
  • 8
    • 0006699504 scopus 로고
    • Haemophilia in Sweden. III. Symptomatology, with special reference to differences between haemophilia A and B
    • Ramgren O. Haemophilia in Sweden. III. Symptomatology, with special reference to differences between haemophilia A and B. Acta Med Scand: 1962; 171 237 242
    • (1962) Acta Med Scand , vol.171 , pp. 237-242
    • Ramgren, O.1
  • 9
    • 34547757915 scopus 로고    scopus 로고
    • Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia
    • Manco-Johnson M. J., Abshire T. C., Shapiro A. D., 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
  • 11
    • 84922590933 scopus 로고    scopus 로고
    • The history of haemophilia - a short review
    • 01
    • Schramm W. The history of haemophilia - a short review. Thromb Res: 2014; 134 01 S4 S9
    • (2014) Thromb Res , vol.134 , pp. S4-S9
    • Schramm, W.1
  • 12
    • 0030186421 scopus 로고    scopus 로고
    • An account of an hemorrhagic disposition existing in certain families
    • Otto J. C. An account of an hemorrhagic disposition existing in certain families. Clin Orthop Relat Res: 1996; 328 4 6
    • (1996) Clin Orthop Relat Res , Issue.328 , pp. 4-6
    • Otto, J.C.1
  • 13
    • 0035822038 scopus 로고    scopus 로고
    • The hemophilias - from royal genes to gene therapy
    • Mannucci P. M., Tuddenham E. G. 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
  • 14
    • 0033109018 scopus 로고    scopus 로고
    • The history of haemophilia in the royal families of Europe
    • Stevens R. F. The history of haemophilia in the royal families of Europe. Br J Haematol: 1999; 105 1 25 32
    • (1999) Br J Haematol , vol.105 , Issue.1 , pp. 25-32
    • Stevens, R.F.1
  • 16
    • 0000334774 scopus 로고
    • Hemophilia. II. Some properties of a substance obtained from normal human plasma effective in accelerating the coagulation of hemophilic blood
    • Patek A. J., Taylor F. H. Hemophilia. II. Some properties of a substance obtained from normal human plasma effective in accelerating the coagulation of hemophilic blood. J Clin Invest: 1937; 16 1 113 124
    • (1937) J Clin Invest , vol.16 , Issue.1 , pp. 113-124
    • Patek, A.J.1    Taylor, F.H.2
  • 17
    • 84873764787 scopus 로고
    • Christmas disease: a condition previously mistaken for haemophilia
    • Biggs R., Douglas A. S., MacFarlane R. G., Dacie J. V., Pitney W. R., Merskey. Christmas disease: a condition previously mistaken for haemophilia. Br Med J: 1952; 2 4799 1378 1382
    • (1952) Br Med J , vol.2 , Issue.4799 , pp. 1378-1382
    • Biggs, R.1    Douglas, A.S.2    MacFarlane, R.G.3    Dacie, J.V.4    Pitney, W.R.5    Merskey6
  • 18
    • 0014103739 scopus 로고
    • Thirty years of haemophilia treatment in Oxford
    • Biggs R. Thirty years of haemophilia treatment in Oxford. Br J Haematol: 1967; 13 4 452 463
    • (1967) Br J Haematol , vol.13 , Issue.4 , pp. 452-463
    • Biggs, R.1
  • 19
    • 0000553473 scopus 로고
    • High-potency antihaemophilic factor concentrate prepared from cryoglobulin precipitate
    • Pool J. G., Gershgold E. J., Pappenhagen A. R. 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
  • 21
    • 0032554268 scopus 로고    scopus 로고
    • New-variant Creutzfeldt-Jakob disease and treatment of haemophilia
    • Baxter T., Black D., Birks D. New-variant Creutzfeldt-Jakob disease and treatment of haemophilia. Lancet: 1998; 351 9102 600 601
    • (1998) Lancet , vol.351 , Issue.9102 , pp. 600-601
    • Baxter, T.1    Black, D.2    Birks, D.3
  • 22
    • 0024555758 scopus 로고
    • Use of recombinant antihemophilic factor in the treatment of two patients with classic hemophilia
    • White G. C. II, McMillan C. W., Kingdon H. S., Shoemaker C. B. Use of recombinant antihemophilic factor in the treatment of two patients with classic hemophilia. N Engl J Med: 1989; 320 3 166 170
    • (1989) N Engl J Med , vol.320 , Issue.3 , pp. 166-170
    • White, G.C.I.I.1    McMillan, C.W.2    Kingdon, H.S.3    Shoemaker, C.B.4
  • 24
    • 0026635406 scopus 로고
    • Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
    • Nilsson I. M., 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
  • 25
    • 0033028402 scopus 로고    scopus 로고
    • Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized
    • Astermark J., Petrini P., Tengborn L., Schulman S., Ljung R., Berntorp E. Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized. Br J Haematol: 1999; 105 4 1109 1113
    • (1999) Br J Haematol , vol.105 , Issue.4 , pp. 1109-1113
    • Astermark, J.1    Petrini, P.2    Tengborn, L.3    Schulman, S.4    Ljung, R.5    Berntorp, E.6
  • 26
    • 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 J. G., Mauser-Bunschoten E. P., 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
  • 27
    • 79953306825 scopus 로고    scopus 로고
    • A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)
    • Gringeri A., Lundin B., von Mackensen S., Mantovani L., Mannucci P. M.; ESPRIT Study Group. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost: 2011; 9 4 700 710
    • (2011) J Thromb Haemost , vol.9 , Issue.4 , pp. 700-710
    • Gringeri, A.1    Lundin, B.2    Von Mackensen, S.3    Mantovani, L.4    Mannucci, P.M.5
  • 28
    • 0031876988 scopus 로고    scopus 로고
    • When should prophylactic treatment in patients with haemophilia A and B start? - The German experience
    • Kreuz W., Escuriola-Ettingshausen C., Funk M., Schmidt H., Kornhuber B. When should prophylactic treatment in patients with haemophilia A and B start? - The German experience. Haemophilia: 1998; 4 4 413 417
    • (1998) Haemophilia , vol.4 , Issue.4 , pp. 413-417
    • Kreuz, W.1    Escuriola-Ettingshausen, C.2    Funk, M.3    Schmidt, H.4    Kornhuber, B.5
  • 30
    • 77951681450 scopus 로고    scopus 로고
    • A United Kingdom Haemophilia Centre Doctors' Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A
    • Richards M., Williams M., Chalmers E., et al. Paediatric Working Party of the United Kingdom Haemophilia Doctors' Organisation. A United Kingdom Haemophilia Centre Doctors' Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A. Br J Haematol: 2010; 149 4 498 507
    • (2010) Br J Haematol , vol.149 , Issue.4 , pp. 498-507
    • Richards, M.1    Williams, M.2    Chalmers, E.3
  • 31
    • 84920286350 scopus 로고    scopus 로고
    • Definitions in hemophilia: communication from the SSC of the ISTH
    • Blanchette V. S., Key N. S., Ljung L. R., Manco-Johnson M. J., van den Berg H. M., Srivastava A.; Subcommittee on Factor VIII, Factor IX and Rare Coagulation Disorders of the Scientific and Standardization Committee of the International Society on Thrombosis and Hemostasis. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost: 2014; 12 11 1935 1939
    • (2014) J Thromb Haemost , vol.12 , Issue.11 , pp. 1935-1939
    • Blanchette, V.S.1    Key, N.S.2    Ljung, L.R.3    Manco-Johnson, M.J.4    Van Den Berg, H.M.5    Srivastava, A.6
  • 33
    • 78649903536 scopus 로고    scopus 로고
    • Venous access in the management of hemophilia
    • Valentino L. A., Kawji M., Grygotis M. Venous access in the management of hemophilia. Blood Rev: 2011; 25 1 11 15
    • (2011) Blood Rev , vol.25 , Issue.1 , pp. 11-15
    • Valentino, L.A.1    Kawji, M.2    Grygotis, M.3
  • 34
    • 33646759553 scopus 로고    scopus 로고
    • Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study
    • Feldman B. M., Pai M., Rivard G. E., et al. Association of Hemophilia Clinic Directors of Canada Prophylaxis Study Group. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost: 2006; 4 6 1228 1236
    • (2006) J Thromb Haemost , vol.4 , Issue.6 , pp. 1228-1236
    • Feldman, B.M.1    Pai, M.2    Rivard, G.E.3
  • 35
    • 84874967483 scopus 로고    scopus 로고
    • Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) Study
    • Hilliard P., Zourikian N., Blanchette V., et al. Musculoskeletal health of subjects with hemophilia A treated with tailored prophylaxis: Canadian Hemophilia Primary Prophylaxis (CHPS) Study. J Thromb Haemost: 2013; 11 3 460 466
    • (2013) J Thromb Haemost , vol.11 , Issue.3 , pp. 460-466
    • Hilliard, P.1    Zourikian, N.2    Blanchette, V.3
  • 36
    • 84871073400 scopus 로고    scopus 로고
    • Magnetic resonance imaging and joint outcomes in boys with severe hemophilia A treated with tailored primary prophylaxis in Canada
    • Kraft J., Blanchette V., Babyn P., et al. Magnetic resonance imaging and joint outcomes in boys with severe hemophilia A treated with tailored primary prophylaxis in Canada. J Thromb Haemost: 2012; 10 12 2494 2502
    • (2012) J Thromb Haemost , vol.10 , Issue.12 , pp. 2494-2502
    • Kraft, J.1    Blanchette, V.2    Babyn, P.3
  • 37
    • 22144490270 scopus 로고    scopus 로고
    • Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands
    • van Dijk K., Fischer K., van der Bom J. G., Scheibel E., Ingerslev J., van den Berg H. M. Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands. Br J Haematol: 2005; 130 1 107 112
    • (2005) Br J Haematol , vol.130 , Issue.1 , pp. 107-112
    • Van Dijk, K.1    Fischer, K.2    Van Der Bom, J.G.3    Scheibel, E.4    Ingerslev, J.5    Van Den Berg, H.M.6
  • 38
    • 73049092113 scopus 로고    scopus 로고
    • Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study
    • Collins P., Faradji A., Morfini M., Enriquez M. M., Schwartz L. Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study. J Thromb Haemost: 2010; 8 1 83 89
    • (2010) J Thromb Haemost , vol.8 , Issue.1 , pp. 83-89
    • Collins, P.1    Faradji, A.2    Morfini, M.3    Enriquez, M.M.4    Schwartz, L.5
  • 39
    • 84883053954 scopus 로고    scopus 로고
    • Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study
    • Manco-Johnson M. J., Sanders J., Ewing N., Rodriguez N., Tarantino M., Humphries T.; TEEN/TWEN Study Group. Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study. Haemophilia: 2013; 19 5 727 735
    • (2013) Haemophilia , vol.19 , Issue.5 , pp. 727-735
    • Manco-Johnson, M.J.1    Sanders, J.2    Ewing, N.3    Rodriguez, N.4    Tarantino, M.5    Humphries, T.6
  • 40
    • 84883798278 scopus 로고    scopus 로고
    • Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART)
    • Manco-Johnson M. J., Kempton C. L., Reding M. T., et al. Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART). J Thromb Haemost: 2013; 11 6 1119 1127
    • (2013) J Thromb Haemost , vol.11 , Issue.6 , pp. 1119-1127
    • Manco-Johnson, M.J.1    Kempton, C.L.2    Reding, M.T.3
  • 41
    • 84928826150 scopus 로고    scopus 로고
    • Switching from current factor VIII (FVIII) to longer acting FVIII concentrates--what is the real potential benefit?
    • Carcao M. Switching from current factor VIII (FVIII) to longer acting FVIII concentrates--what is the real potential benefit? Haemophilia: 2015; 21 3 297 299
    • (2015) Haemophilia , vol.21 , Issue.3 , pp. 297-299
    • Carcao, M.1
  • 42
    • 0038779248 scopus 로고    scopus 로고
    • A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres
    • 01, discussion 26
    • Blanchette V. S., McCready M., Achonu C., Abdolell M., Rivard G., Manco-Johnson M. J. A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres. Haemophilia: 2003; 9 01 19 26, discussion 26
    • (2003) Haemophilia , vol.9 , pp. 19-26
    • Blanchette, V.S.1    McCready, M.2    Achonu, C.3    Abdolell, M.4    Rivard, G.5    Manco-Johnson, M.J.6
  • 43
    • 84865558828 scopus 로고    scopus 로고
    • Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States
    • Duncan N., Shapiro A., Ye X., Epstein J., Luo M. P. Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States. Haemophilia: 2012; 18 5 760 765
    • (2012) Haemophilia , vol.18 , Issue.5 , pp. 760-765
    • Duncan, N.1    Shapiro, A.2    Ye, X.3    Epstein, J.4    Luo, M.P.5
  • 44
    • 84863186626 scopus 로고    scopus 로고
    • Current prescription of prophylactic factor infusions and perceived adherence for children and adolescents with haemophilia: a survey of haemophilia healthcare professionals in the United States
    • Thornburg C. D., Carpenter S., Zappa S., Munn J., Leissinger C. Current prescription of prophylactic factor infusions and perceived adherence for children and adolescents with haemophilia: a survey of haemophilia healthcare professionals in the United States. Haemophilia: 2012; 18 4 568 574
    • (2012) Haemophilia , vol.18 , Issue.4 , pp. 568-574
    • Thornburg, C.D.1    Carpenter, S.2    Zappa, S.3    Munn, J.4    Leissinger, C.5
  • 45
    • 0034913232 scopus 로고    scopus 로고
    • Barriers to compliance with prophylaxis therapy in haemophilia
    • Hacker M. R., 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
  • 46
    • 84930274386 scopus 로고    scopus 로고
    • Role of enhanced half-life factor VIII and IX in the treatment of haemophilia
    • Mahdi A. J., Obaji S. G., Collins P. W. Role of enhanced half-life factor VIII and IX in the treatment of haemophilia. Br J Haematol: 2015; 169 6 768 776
    • (2015) Br J Haematol , vol.169 , Issue.6 , pp. 768-776
    • Mahdi, A.J.1    Obaji, S.G.2    Collins, P.W.3
  • 47
    • 84899562558 scopus 로고    scopus 로고
    • Changing paradigm of prophylaxis with longer acting factor concentrates
    • 04
    • Carcao M. Changing paradigm of prophylaxis with longer acting factor concentrates. Haemophilia: 2014; 20 04 99 105
    • (2014) Haemophilia , vol.20 , pp. 99-105
    • Carcao, M.1
  • 48
    • 84920427011 scopus 로고    scopus 로고
    • Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels
    • Shapiro A. D., Ragni M. V., Kulkarni R., et al. Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost: 2014; 12 11 1788 1800
    • (2014) J Thromb Haemost , vol.12 , Issue.11 , pp. 1788-1800
    • Shapiro, A.D.1    Ragni, M.V.2    Kulkarni, R.3
  • 49
    • 11844250025 scopus 로고    scopus 로고
    • A passionate kiss, then run: exocytosis and recycling of IgG by FcRn
    • Lencer W. I., Blumberg R. S. A passionate kiss, then run: exocytosis and recycling of IgG by FcRn. Trends Cell Biol: 2005; 15 1 5 9
    • (2005) Trends Cell Biol , vol.15 , Issue.1 , pp. 5-9
    • Lencer, W.I.1    Blumberg, R.S.2
  • 50
    • 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 A. D., Ragni M. V., Valentino L. A., 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
  • 51
    • 84889769562 scopus 로고    scopus 로고
    • Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B
    • Powell J. S., Pasi K. J., Ragni M. V., 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
  • 52
    • 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
  • 53
    • 84916232822 scopus 로고    scopus 로고
    • Long-acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B-LONG study
    • Powell J. S., Apte S., Chambost H., et al. Long-acting recombinant factor IX Fc fusion protein (rFIXFc) for perioperative management of subjects with haemophilia B in the phase 3 B-LONG study. Br J Haematol: 2015; 168 1 124 134
    • (2015) Br J Haematol , vol.168 , Issue.1 , pp. 124-134
    • Powell, J.S.1    Apte, S.2    Chambost, H.3
  • 54
    • 84928756980 scopus 로고    scopus 로고
    • Pharmacokinetics of recombinant factor IX Fc fusion protein (rFIXFc) in pediatric subjects with hemophilia B: an interim analysis of the Kids B-LONG Study
    • Fischer K., Kulkarni R., Bradbury M., et al. Pharmacokinetics of recombinant factor IX Fc fusion protein (rFIXFc) in pediatric subjects with hemophilia B: an interim analysis of the Kids B-LONG Study. Blood: 2013; 122 21
    • (2013) Blood , vol.122 , Issue.21
    • Fischer, K.1    Kulkarni, R.2    Bradbury, M.3
  • 55
    • 85028484090 scopus 로고    scopus 로고
    • Safety, efficacy and pharmacokinetics of recombinant factor IX FC fusion protein in children with haemophilia B (KIDS B-LONG)
    • Fischer K., Kulkarni R., Nolan B., et al. Safety, efficacy and pharmacokinetics of recombinant factor IX FC fusion protein in children with haemophilia B (KIDS B-LONG). J Thromb Haemost: 2015; 13 87 87
    • (2015) J Thromb Haemost , vol.13 , pp. 87
    • Fischer, K.1    Kulkarni, R.2    Nolan, B.3
  • 56
    • 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., Moss 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    Moss, J.5
  • 57
    • 84919491430 scopus 로고    scopus 로고
    • Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial
    • Collins P. W., Young G., Knobe K., et al. paradigm 2 Investigators. 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
  • 58
    • 84930275756 scopus 로고    scopus 로고
    • Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously treated pediatric hemophilia B patients
    • Carcao M., Zak M., Karim F. A., et al. Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously treated pediatric hemophilia B patients. Blood: 2014; 124 21
    • (2014) Blood , vol.124 , Issue.21
    • Carcao, M.1    Zak, M.2    Karim, F.A.3
  • 59
    • 70449461639 scopus 로고    scopus 로고
    • Genetic fusion to albumin improves the pharmacokinetic properties of factor IX
    • Metzner H. J., 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
  • 60
    • 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 12 2405 2411
    • (2012) Blood , vol.120 , Issue.12 , pp. 2405-2411
    • Santagostino, E.1    Negrier, C.2    Klamroth, R.3
  • 61
    • 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. doi:10.1111/hae.12721
    • (2015) Haemophilia , vol.21 , Issue.6 , pp. 784-790
    • Martinowitz, U.1    Lissitchkov, T.2    Lubetsky, A.3
  • 62
    • 84942502471 scopus 로고    scopus 로고
    • Pharmacokinetic results of two phase III clinical studies of coagulation factor ix (recombinant) albumin fusion protein (rIX-FP) in previously treated patients with hemophilia B (PROLONG-9FP)
    • Santagostino E., Jacobs I. C., Voigt C., Feussner A., Limsakun T. Pharmacokinetic results of two phase III clinical studies of coagulation factor ix (recombinant) albumin fusion protein (rIX-FP) in previously treated patients with hemophilia B (PROLONG-9FP). Blood: 2014; 124 21
    • (2014) Blood , vol.124 , Issue.21
    • Santagostino, E.1    Jacobs, I.C.2    Voigt, C.3    Feussner, A.4    Limsakun, T.5
  • 63
    • 84859197329 scopus 로고    scopus 로고
    • Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
    • Powell J. S., Josephson N. C., 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
  • 64
    • 84893123337 scopus 로고    scopus 로고
    • Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
    • Mahlangu J., Powell J. S., Ragni M. V., 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
  • 65
    • 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
  • 66
    • 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
  • 67
    • 84992382294 scopus 로고    scopus 로고
    • Clinical evaluation of novel recombinant glycopegylated FVIII (turoctocog alfa pegol, N8-GP): efficacy and safety in previously treated patients with severe hemophilia A--results of pathfinder (TM) 2 international trial
    • Giangrande P., Chowdary P., Enhrenforth S., et al. Clinical evaluation of novel recombinant glycopegylated FVIII (turoctocog alfa pegol, N8-GP): efficacy and safety in previously treated patients with severe hemophilia A--results of pathfinder (TM) 2 international trial. J Thromb Haemost: 2015; 13 176 176
    • (2015) J Thromb Haemost , vol.13 , pp. 176
    • Giangrande, P.1    Chowdary, P.2    Enhrenforth, S.3
  • 68
    • 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 T. E., Reding M. T., Lin J. C., Michaels L. A., 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
  • 69
    • 84930271775 scopus 로고    scopus 로고
    • Bleeding Phenotype with Various Bay 94-9027 Dosing Regimens: Subanalyses from the Protect VIII Study
    • Boggio L. N., Hong W., Wang M., Eyster M. E., Michaels L. A. Bleeding Phenotype with Various Bay 94-9027 Dosing Regimens: Subanalyses from the Protect VIII Study. Blood: 2014; 124 21
    • (2014) Blood , vol.124 , Issue.21
    • Boggio, L.N.1    Hong, W.2    Wang, M.3    Eyster, M.E.4    Michaels, L.A.5
  • 70
    • 84873625664 scopus 로고    scopus 로고
    • BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation
    • 01
    • Turecek P. L., Bossard M. J., Graninger M., et al. BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional and structural characterisation. Hamostaseologie: 2012; 32 01 S29 S38
    • (2012) Hamostaseologie , vol.32 , pp. S29-S38
    • Turecek, P.L.1    Bossard, M.J.2    Graninger, M.3
  • 71
    • 84942546645 scopus 로고    scopus 로고
    • Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A
    • Konkle B. A., 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
  • 72
    • 84938556132 scopus 로고    scopus 로고
    • Physicochemical characterisation of rVIII-SingleChain, a novel recombinant single-chain factor VIII
    • Schmidbauer S., Witzel R., Robbel L., et al. Physicochemical characterisation of rVIII-SingleChain, a novel recombinant single-chain factor VIII. Thromb Res: 2015; 136 2 388 395
    • (2015) Thromb Res , vol.136 , Issue.2 , pp. 388-395
    • Schmidbauer, S.1    Witzel, R.2    Robbel, L.3
  • 73
    • 84970937919 scopus 로고    scopus 로고
    • RVIII-SingleChain, results of the pivotal phase I/III PK, efficacy and safety clinical trial in adults and adolescents with severe hemophilia A
    • Mahlangu J., Kuliczkowski K., Stasyshyn O., et al. RVIII-SingleChain, results of the pivotal phase I/III PK, efficacy and safety clinical trial in adults and adolescents with severe hemophilia A. J Thromb Haemost: 2015; 13 86 86
    • (2015) J Thromb Haemost , vol.13 , pp. 86
    • Mahlangu, J.1    Kuliczkowski, K.2    Stasyshyn, O.3
  • 74
    • 84974537434 scopus 로고    scopus 로고
    • Efficacy and safety of RVIII-SingleChain in surgical prophylaxis
    • Khayat C. D., Mahlangu J., Leisinger C., et al. Efficacy and safety of RVIII-SingleChain in surgical prophylaxis. J Thromb Haemost: 2015; 13 602 602
    • (2015) J Thromb Haemost , vol.13 , pp. 602
    • Khayat, C.D.1    Mahlangu, J.2    Leisinger, C.3
  • 75
    • 84941260214 scopus 로고    scopus 로고
    • Haemophilia gene therapy: progress and challenges
    • Lheriteau E., Davidoff A. M., Nathwani A. C. Haemophilia gene therapy: progress and challenges. Blood Rev: 2015; 29 5 321 328
    • (2015) Blood Rev , vol.29 , Issue.5 , pp. 321-328
    • Lheriteau, E.1    Davidoff, A.M.2    Nathwani, A.C.3
  • 76
    • 84993736172 scopus 로고    scopus 로고
    • New treatments in hemophilia: insights for the clinician
    • Knobe K., Berntorp E. New treatments in hemophilia: insights for the clinician. Ther Adv Hematol: 2012; 3 3 165 175
    • (2012) Ther Adv Hematol , vol.3 , Issue.3 , pp. 165-175
    • Knobe, K.1    Berntorp, E.2
  • 77
    • 0021318961 scopus 로고
    • Development of factor VIII:C antibodies in dogs with hemophilia A (factor VIII:C deficiency)
    • Giles A. R., Tinlin S., Hoogendoorn H., Greenwood P., Greenwood R. Development of factor VIII:C antibodies in dogs with hemophilia A (factor VIII:C deficiency). Blood: 1984; 63 2 451 456
    • (1984) Blood , vol.63 , Issue.2 , pp. 451-456
    • Giles, A.R.1    Tinlin, S.2    Hoogendoorn, H.3    Greenwood, P.4    Greenwood, R.5
  • 79
    • 84911483252 scopus 로고    scopus 로고
    • Costs and utilization of hemophilia A and B patients with and without inhibitors
    • Armstrong E. P., Malone D. C., Krishnan S., Wessler M. J. Costs and utilization of hemophilia A and B patients with and without inhibitors. J Med Econ: 2014; 17 11 798 802
    • (2014) J Med Econ , vol.17 , Issue.11 , pp. 798-802
    • Armstrong, E.P.1    Malone, D.C.2    Krishnan, S.3    Wessler, M.J.4
  • 81
    • 84877704167 scopus 로고    scopus 로고
    • Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
    • McIntosh J., Lenting P. J., Rosales C., et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood: 2013; 121 17 3335 3344
    • (2013) Blood , vol.121 , Issue.17 , pp. 3335-3344
    • McIntosh, J.1    Lenting, P.J.2    Rosales, C.3
  • 82
    • 84910633509 scopus 로고    scopus 로고
    • Our journey to successful gene therapy for hemophilia B
    • Nathwani A. C., Nienhuis A. W., Davidoff A. M. Our journey to successful gene therapy for hemophilia B. Hum Gene Ther: 2014; 25 11 923 926
    • (2014) Hum Gene Ther , vol.25 , Issue.11 , pp. 923-926
    • Nathwani, A.C.1    Nienhuis, A.W.2    Davidoff, A.M.3
  • 83
    • 84911383748 scopus 로고    scopus 로고
    • Long-term safety and efficacy of factor IX gene therapy in hemophilia B
    • Nathwani A. C., Reiss U. M., Tuddenham E. GD., 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
  • 84
    • 84855161388 scopus 로고    scopus 로고
    • Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    • Nathwani A. C., Tuddenham E. GD, 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
  • 85
    • 0141679053 scopus 로고    scopus 로고
    • Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion
    • Powell J. S., Ragni M. V., White G. C. II., et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood: 2003; 102 6 2038 2045
    • (2003) Blood , vol.102 , Issue.6 , pp. 2038-2045
    • Powell, J.S.1    Ragni, M.V.2    White, I.I.G.C.3
  • 86
    • 0030275393 scopus 로고    scopus 로고
    • Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients
    • Qiu X., Lu D., Zhou J., et al. Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients. Chin Med J (Engl): 1996; 109 11 832 839
    • (1996) Chin Med J (Engl) , vol.109 , Issue.11 , pp. 832-839
    • Qiu, X.1    Lu, D.2    Zhou, J.3
  • 87
    • 0035822005 scopus 로고    scopus 로고
    • Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
    • Roth D. A., Tawa N. E. Jr, O'Brien J. M., Treco D. A., Selden R. F.; Factor VIII Transkaryotic Therapy Study Group. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med: 2001; 344 23 1735 1742
    • (2001) N Engl J Med , vol.344 , Issue.23 , pp. 1735-1742
    • Roth, D.A.1    Tawa, N.E.2    O'Brien, J.M.3    Treco, D.A.4    Selden, R.F.5
  • 88
    • 4043181040 scopus 로고    scopus 로고
    • Implantation of non-viral ex vivo genetically modified autologous dermal fibroblasts that express B-domain deleted human factor VIII in 12 severe hemophilia A study subjects
    • Roth D. A., Tawa N. E., Proper J., et al. Implantation of non-viral ex vivo genetically modified autologous dermal fibroblasts that express B-domain deleted human factor VIII in 12 severe hemophilia A study subjects. Blood: 2002; 100 11 116a 117a
    • (2002) Blood , vol.100 , Issue.11 , pp. 116a-117a
    • Roth, D.A.1    Tawa, N.E.2    Proper, J.3
  • 89
    • 0043033652 scopus 로고    scopus 로고
    • Report of a second serious adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID)
    • Gansbacher B.; European Society of Gene Therapy; Position of the European Society of Gene Therapy (ESGT). Report of a second serious adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID). J Gene Med: 2003; 5 3 261 262
    • (2003) J Gene Med , vol.5 , Issue.3 , pp. 261-262
    • Gansbacher, B.1
  • 90
    • 0037448352 scopus 로고    scopus 로고
    • A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
    • Hacein-Bey-Abina S., von Kalle C., Schmidt M., et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med: 2003; 348 3 255 256
    • (2003) N Engl J Med , vol.348 , Issue.3 , pp. 255-256
    • Hacein-Bey-Abina, S.1    Von Kalle, C.2    Schmidt, M.3
  • 92
    • 0034050904 scopus 로고    scopus 로고
    • Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
    • Kay M. A., Manno C. S., Ragni M. V., et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet: 2000; 24 3 257 261
    • (2000) Nat Genet , vol.24 , Issue.3 , pp. 257-261
    • Kay, M.A.1    Manno, C.S.2    Ragni, M.V.3
  • 93
    • 0013442744 scopus 로고    scopus 로고
    • AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    • Manno C. S., Chew A. J., Hutchison S., et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood: 2003; 101 8 2963 2972
    • (2003) Blood , vol.101 , Issue.8 , pp. 2963-2972
    • Manno, C.S.1    Chew, A.J.2    Hutchison, S.3
  • 94
    • 4043057757 scopus 로고    scopus 로고
    • Immune responses to AAV and to factor IX in a phase I study of AAV-mediated, liver-directed gene transfer for hemophilia B
    • High K. A., Manno C. S., Sabatino D. E., et al. Immune responses to AAV and to factor IX in a phase I study of AAV-mediated, liver-directed gene transfer for hemophilia B. Blood: 2003; 102 11 154a 155a
    • (2003) Blood , vol.102 , Issue.11 , pp. 154a-155a
    • High, K.A.1    Manno, C.S.2    Sabatino, D.E.3
  • 95
    • 84970916883 scopus 로고    scopus 로고
    • Administration of adenovirus months after transduction of the liver by recombinant adeno-associated virus (rAAV) significantly enhances stable rAAV transgene expression by mechanism independent of second strand synthesis
    • Davidoff A. M., Ng C. YC, Zhou J. F., Spence Y., Nathwani A. C. Administration of adenovirus months after transduction of the liver by recombinant adeno-associated virus (rAAV) significantly enhances stable rAAV transgene expression by mechanism independent of second strand synthesis. Mol Ther: 2003; 7 5 S45 S45
    • (2003) Mol Ther , vol.7 , Issue.5 , pp. S45-S45
    • Davidoff, A.M.1    Ng, C.Y.2    Zhou, J.F.3    Spence, Y.4    Nathwani, A.C.5
  • 96
    • 0037015049 scopus 로고    scopus 로고
    • Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
    • Gao G. P., Alvira M. R., Wang L., Calcedo R., Johnston J., Wilson J. M. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A: 2002; 99 18 11854 11859
    • (2002) Proc Natl Acad Sci U S A , vol.99 , Issue.18 , pp. 11854-11859
    • Gao, G.P.1    Alvira, M.R.2    Wang, L.3    Calcedo, R.4    Johnston, J.5    Wilson, J.M.6
  • 97
    • 0141482003 scopus 로고    scopus 로고
    • Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy
    • Grimm D., Zhou S., Nakai H., et al. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood: 2003; 102 7 2412 2419
    • (2003) Blood , vol.102 , Issue.7 , pp. 2412-2419
    • Grimm, D.1    Zhou, S.2    Nakai, H.3
  • 99
    • 84944215188 scopus 로고    scopus 로고
    • The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals
    • Mimuro J., Mizukami H., Shima M., et al. The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals. J Med Virol: 2014; 86 11 1990 1997
    • (2014) J Med Virol , vol.86 , Issue.11 , pp. 1990-1997
    • Mimuro, J.1    Mizukami, H.2    Shima, M.3
  • 100
    • 84899567922 scopus 로고    scopus 로고
    • Novel products for haemostasis - current status
    • 04
    • Oldenburg J., Albert T. Novel products for haemostasis - current status. Haemophilia: 2014; 20 04 23 28
    • (2014) Haemophilia , vol.20 , pp. 23-28
    • Oldenburg, J.1    Albert, T.2
  • 101
    • 84930337835 scopus 로고    scopus 로고
    • Lasting power of new clotting proteins
    • Powell J. S. Lasting power of new clotting proteins. Hematology Am Soc Hematol Educ Program: 2014; 2014 1 355 363
    • (2014) Hematology Am Soc Hematol Educ Program , vol.2014 , Issue.1 , pp. 355-363
    • Powell, J.S.1
  • 102
    • 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
  • 103
    • 84872032297 scopus 로고    scopus 로고
    • A complex substitute: antibody therapy for hemophilia
    • Lillicrap D. A complex substitute: antibody therapy for hemophilia. Nat Med: 2012; 18 10 1460 1461
    • (2012) Nat Med , vol.18 , Issue.10 , pp. 1460-1461
    • Lillicrap, D.1
  • 104
    • 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
  • 105
    • 84970921305 scopus 로고    scopus 로고
    • The potential of bispecific antibodies for treatment of hemophilia A
    • Shima M. The potential of bispecific antibodies for treatment of hemophilia A. J Thromb Haemost: 2015; 13 5 6
    • (2015) J Thromb Haemost , vol.13 , pp. 5-6
    • Shima, M.1
  • 106
    • 84970937901 scopus 로고    scopus 로고
    • Long-term safety and prophylactic efficacy of once-weekly subcutaneous administration of ACE910, in Japanese hemophilia A patients with and without FVIII inhibitors: interim results of the extension study of a phase 1 study
    • Shima M., Hanabusa H., Taki M., et al. Long-term safety and prophylactic efficacy of once-weekly subcutaneous administration of ACE910, in Japanese hemophilia A patients with and without FVIII inhibitors: interim results of the extension study of a phase 1 study. J Thromb Haemost: 2015; 13 6 7
    • (2015) J Thromb Haemost , vol.13 , pp. 6-7
    • Shima, M.1    Hanabusa, H.2    Taki, M.3
  • 107
    • 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., Sorensen B. B., 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    Sorensen, B.B.3
  • 108
    • 84859798949 scopus 로고    scopus 로고
    • Hemostatic properties of a TFPI antibody
    • 02
    • Petersen L. C. Hemostatic properties of a TFPI antibody. Thromb Res: 2012; 129 02 S44 S45
    • (2012) Thromb Res , vol.129 , pp. S44-S45
    • Petersen, L.C.1
  • 109
    • 84870238164 scopus 로고    scopus 로고
    • Effect of BAX499 aptamer on tissue factor pathway inhibitor function and thrombin generation in models of hemophilia
    • Gissel M., Orfeo T., Foley J. H., Butenas S. Effect of BAX499 aptamer on tissue factor pathway inhibitor function and thrombin generation in models of hemophilia. Thromb Res: 2012; 130 6 948 955
    • (2012) Thromb Res , vol.130 , Issue.6 , pp. 948-955
    • Gissel, M.1    Orfeo, T.2    Foley, J.H.3    Butenas, S.4
  • 110
    • 84970916882 scopus 로고    scopus 로고
    • Pharmacodynamics and pharmacokinetics of TFPI-neutralizing antibody (BAY1093884) in cynomolgus monkeys and prediction of human dose
    • Gu J. M., Ho E., Zhao X. Y., et al. Pharmacodynamics and pharmacokinetics of TFPI-neutralizing antibody (BAY1093884) in cynomolgus monkeys and prediction of human dose. J Thromb Haemost: 2015; 13 7 7
    • (2015) J Thromb Haemost , vol.13 , pp. 7
    • Gu, J.M.1    Ho, E.2    Zhao, X.Y.3
  • 111
    • 84976864737 scopus 로고    scopus 로고
    • Thrombin generation is increased in plasma from healthy males who have received concizumab, an antibody against tissue factor pathway inhibitor (ExplorerTM2)
    • Waters E. K., Sigh J., Ezban M., Hilden I. Thrombin generation is increased in plasma from healthy males who have received concizumab, an antibody against tissue factor pathway inhibitor (ExplorerTM2). J Thromb Haemost: 2015; 13 7 7
    • (2015) J Thromb Haemost , vol.13 , pp. 7
    • Waters, E.K.1    Sigh, J.2    Ezban, M.3    Hilden, I.4
  • 112
    • 84902303698 scopus 로고    scopus 로고
    • Clinical presentation and molecular basis of congenital antithrombin deficiency in children: a cohort study
    • Kumar R., Chan A. KC, Dawson J. E., Forman-Kay J. D., Kahr W. H., Williams S. Clinical presentation and molecular basis of congenital antithrombin deficiency in children: a cohort study. Br J Haematol: 2014; 166 1 130 139
    • (2014) Br J Haematol , vol.166 , Issue.1 , pp. 130-139
    • Kumar, R.1    Chan, A.K.2    Dawson, J.E.3    Forman-Kay, J.D.4    Kahr, W.H.5    Williams, S.6
  • 113
    • 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


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