메뉴 건너뛰기




Volumn 7, Issue , 2016, Pages 187-198

Therapeutic and routine prophylactic properties of rFactor VIII Fc (efraloctocog alfa, Eloctate®) in hemophilia A

Author keywords

Efraloctocog alfa; Extended half life factors; FVIII; Hemophilia A; Prophylaxis; rFVIIIFc

Indexed keywords

ALBUMIN; BLOOD CLOTTING FACTOR 8; EFRALOCTOCOG ALFA; IMMUNOGLOBULIN; NEUTRALIZING ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 8; UNCLASSIFIED DRUG;

EID: 84992754529     PISSN: None     EISSN: 11792736     Source Type: Journal    
DOI: 10.2147/JBM.S80814     Document Type: Article
Times cited : (20)

References (49)
  • 2
    • 0035822038 scopus 로고    scopus 로고
    • The hemophilias-from royal genes to gene therapy
    • Mannucci PM, Tuddenham EG. The hemophilias-from royal genes to gene therapy. N Engl J Med. 2001; 344(23): 1773-1779.
    • (2001) N Engl J Med , vol.344 , Issue.23 , pp. 1773-1779
    • Mannucci, P.M.1    Tuddenham, E.G.2
  • 3
    • 0035077234 scopus 로고    scopus 로고
    • Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis
    • White GC 2nd, Rosendaal F, Aledort LM, 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
  • 4
    • 84992727332 scopus 로고    scopus 로고
    • Front matter
    • New York, NY: John Wiley and Sons, Ltd
    • Lee CA. Front matter. Textbook of Hemophilia. New York, NY: John Wiley and Sons, Ltd; 2014: i-xxi.
    • (2014) Textbook of Hemophilia
    • Lee, C.A.1
  • 5
    • 67649840909 scopus 로고    scopus 로고
    • Survival in a cohort of patients with haemophilia at the haemophilia care center in Vienna, Austria, from 1983 to 2006
    • Reitter S, Waldhoer T, Vutuc C, Lechner K, Pabinger I. Survival in a cohort of patients with haemophilia at the haemophilia care center in Vienna, Austria, from 1983 to 2006. Haemophilia. 2009; 15(4): 888-893.
    • (2009) Haemophilia , vol.15 , Issue.4 , pp. 888-893
    • Reitter, S.1    Waldhoer, T.2    Vutuc, C.3    Lechner, K.4    Pabinger, I.5
  • 6
    • 0034913232 scopus 로고    scopus 로고
    • Barriers to compliance with prophylaxis therapy in haemophilia
    • Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia. 2001; 7(4): 392-396.
    • (2001) Haemophilia , vol.7 , Issue.4 , pp. 392-396
    • Hacker, M.R.1    Geraghty, S.2    Manco-Johnson, M.3
  • 7
    • 84939120787 scopus 로고    scopus 로고
    • Profile of efraloctocog alfa and its potential in the treatment of hemophilia A
    • George LA, Camire RM. Profile of efraloctocog alfa and its potential in the treatment of hemophilia A. J Blood Med. 2015; 6: 131-141.
    • (2015) J Blood Med , vol.6 , pp. 131-141
    • George, L.A.1    Camire, R.M.2
  • 8
    • 37749043474 scopus 로고    scopus 로고
    • Physicians’ perceptions of adherence to prophylactic clotting factor infusions
    • Thornburg CD. Physicians’ perceptions of adherence to prophylactic clotting factor infusions. Haemophilia. 2008; 14(1): 25-29.
    • (2008) Haemophilia , vol.14 , Issue.1 , pp. 25-29
    • Thornburg, C.D.1
  • 9
    • 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 CD, 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
  • 10
    • 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
  • 12
    • 84859596442 scopus 로고    scopus 로고
    • Modern haemophilia care
    • Berntorp E, Shapiro AD. 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
  • 13
    • 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. 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
  • 14
    • 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 PM. 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
  • 15
    • 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 MJ, Kempton CL, Reding MT, 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
  • 16
    • 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 MM, 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
  • 17
    • 0035106558 scopus 로고    scopus 로고
    • Long-term outcome of individualized prophylactic treatment of children with severe haemophilia
    • van den Berg HM, Fischer K, Mauser-Bunschoten EP, et al. Long-term outcome of individualized prophylactic treatment of children with severe haemophilia. Br J Haematol. 2001; 112(3): 561-565.
    • (2001) Br J Haematol , vol.112 , Issue.3 , pp. 561-565
    • van den Berg, H.M.1    Fischer, K.2    Mauser-Bunschoten, E.P.3
  • 18
    • 0026635406 scopus 로고
    • Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B
    • Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years’ experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992; 232(1): 25-32.
    • (1992) J Intern Med , vol.232 , Issue.1 , pp. 25-32
    • Nilsson, I.M.1    Berntorp, E.2    Lofqvist, T.3    Pettersson, H.4
  • 19
    • 84887192427 scopus 로고    scopus 로고
    • Intermediate-dose versus high-dose prophylaxis for severe hemophilia: Comparing outcome and costs since the 1970s
    • Fischer K, Carlsson K, Petrini P, et al. Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s. Blood. 2013; 122(7): 1129-1136.
    • (2013) Blood , vol.122 , Issue.7 , pp. 1129-1136
    • Fischer, K.1    Carlsson, K.2    Petrini, P.3
  • 20
    • 70349961526 scopus 로고    scopus 로고
    • Prophylactic therapy in haemophilia
    • Ljung R. Prophylactic therapy in haemophilia. Blood Rev. 2009; 23(6): 267-274.
    • (2009) Blood Rev , vol.23 , Issue.6 , pp. 267-274
    • Ljung, R.1
  • 21
    • 84859612152 scopus 로고
    • Prophylaxis in haemophilia
    • van Creveld S. Prophylaxis in haemophilia. Lancet. 1971; 1(7696): 450.
    • (1971) Lancet , vol.1 , Issue.7696 , pp. 450
    • van Creveld, S.1
  • 22
    • 0026633813 scopus 로고
    • Prophylaxis: The next haemophilia treatment
    • Aledort LM. Prophylaxis: the next haemophilia treatment. J Intern Med. 1992; 232(1): 1-2.
    • (1992) J Intern Med , vol.232 , Issue.1 , pp. 1-2
    • Aledort, L.M.1
  • 23
    • 34547757915 scopus 로고    scopus 로고
    • Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia
    • Manco-Johnson MJ, Abshire T, Shapiro A, 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.2    Shapiro, A.3
  • 24
    • 85027949267 scopus 로고    scopus 로고
    • Considerations in individualizing prophylaxis in patients with haemophilia A
    • Valentino LA. Considerations in individualizing prophylaxis in patients with haemophilia A. Haemophilia. 2014; 20(5): 607-615.
    • (2014) Haemophilia , vol.20 , Issue.5 , pp. 607-615
    • Valentino, L.A.1
  • 26
    • 84859192874 scopus 로고    scopus 로고
    • Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs
    • Dumont JA, Liu T, Low SC, et al. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood. 2012; 119(13): 3024-3030.
    • (2012) Blood , vol.119 , Issue.13 , pp. 3024-3030
    • Dumont, J.A.1    Liu, T.2    Low, S.C.3
  • 27
    • 34548229364 scopus 로고    scopus 로고
    • FcRn: The neonatal Fc receptor comes of age
    • Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 2007; 7(9): 715-725.
    • (2007) Nat Rev Immunol , vol.7 , Issue.9 , pp. 715-725
    • Roopenian, D.C.1    Akilesh, S.2
  • 28
    • 0037362655 scopus 로고    scopus 로고
    • Effect of pegylation on pharmaceuticals
    • Harris JM, Chess RB. Effect of pegylation on pharmaceuticals. Nat Rev Drug Discov. 2003; 2(3): 214-221.
    • (2003) Nat Rev Drug Discov , vol.2 , Issue.3 , pp. 214-221
    • Harris, J.M.1    Chess, R.B.2
  • 29
    • 84861749627 scopus 로고    scopus 로고
    • State of the art in PEGylation: The great versatility achieved after forty years of research
    • Pasut G, Veronese FM. State of the art in PEGylation: the great versatility achieved after forty years of research. J Control Release. 2012; 161(2): 461-472.
    • (2012) J Control Release , vol.161 , Issue.2 , pp. 461-472
    • Pasut, G.1    Veronese, F.M.2
  • 30
    • 79952115967 scopus 로고    scopus 로고
    • The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia
    • Yatuv R, Robinson M, Dayan-Tarshish I, Baru M. The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia. Int J Nanomedicine. 2010; 5: 581-591.
    • (2010) Int J Nanomedicine , vol.5 , pp. 581-591
    • Yatuv, R.1    Robinson, M.2    Dayan-Tarshish, I.3    Baru, M.4
  • 31
    • 77949884124 scopus 로고    scopus 로고
    • Importance of neonatal FcR in regulating the serum half-life of therapeutic proteins containing the Fc domain of human IgG1: A comparative study of the affinity of monoclonal antibodies and Fc-fusion proteins to human neonatal FcR
    • Suzuki T, Ishii-Watabe A, Tada M, et al. Importance of neonatal FcR in regulating the serum half-life of therapeutic proteins containing the Fc domain of human IgG1: A comparative study of the affinity of monoclonal antibodies and Fc-fusion proteins to human neonatal FcR. J Immunol. 2010; 184(4): 1968-1976.
    • (2010) J Immunol , vol.184 , Issue.4 , pp. 1968-1976
    • Suzuki, T.1    Ishii-Watabe, A.2    Tada, M.3
  • 32
    • 84873043409 scopus 로고    scopus 로고
    • Biochemical and functional characterization of a recombinant monomeric factorVIII-Fc fusion protein
    • Peters RT, Toby G, Lu Q, et al. Biochemical and functional characterization of a recombinant monomeric factorVIII-Fc fusion protein. J Thromb Haemost. 2013; 11(1): 132-141.
    • (2013) J Thromb Haemost , vol.11 , Issue.1 , pp. 132-141
    • Peters, R.T.1    Toby, G.2    Lu, Q.3
  • 33
    • 84859197329 scopus 로고    scopus 로고
    • Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients
    • Powell JS, Josephson NC, Quon D, et al. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012; 119(13): 3031-3037.
    • (2012) Blood , vol.119 , Issue.13 , pp. 3031-3037
    • Powell, J.S.1    Josephson, N.C.2    Quon, D.3
  • 34
    • 84893123337 scopus 로고    scopus 로고
    • Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A
    • Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014; 123(3): 317-325.
    • (2014) Blood , vol.123 , Issue.3 , pp. 317-325
    • Mahlangu, J.1    Powell, J.S.2    Ragni, M.V.3
  • 35
    • 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
  • 39
    • 4944263724 scopus 로고    scopus 로고
    • Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: Pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A
    • Tarantino MD, Collins PW, Hay CR, et al. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A. Haemophilia. 2004; 10(5): 428-437.
    • (2004) Haemophilia , vol.10 , Issue.5 , pp. 428-437
    • Tarantino, M.D.1    Collins, P.W.2    Hay, C.R.3
  • 40
    • 84992761460 scopus 로고    scopus 로고
    • Treatment of bleeding with recombinant factor VIII FC fusion protein in previously-treated pediatric subjects with hemophilia A in the phase 3 kids a-long study
    • Paper presented at, June 20-25, Toronto, Canada
    • Chalmers EYG, Mahlangu J, Recht M, et al. Treatment of bleeding with recombinant factor VIII FC fusion protein in previously-treated pediatric subjects with hemophilia A in the phase 3 kids a-long study. Paper presented at: XXV Congress of the International Society on Thrombosis and Haemostasis, June 20-25, 2015, Toronto, Canada.
    • (2015) XXV Congress of the International Society on Thrombosis and Haemostasis
    • Chalmers, E.Y.G.1    Mahlangu, J.2    Recht, M.3
  • 41
    • 84992698382 scopus 로고    scopus 로고
    • Antihemophilic factor [recombinant Fc fusion protein]
    • FDA. antihemophilic factor [recombinant Fc fusion protein]-FDA Clinical Pharmacology BLA Review. 2013.
    • (2013) FDA Clinical Pharmacology BLA Review
  • 42
    • 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
  • 43
    • 0038383605 scopus 로고    scopus 로고
    • The epidemiology of inhibitors in haemophilia A: A systematic review
    • Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: A systematic review. Haemophilia. 2003; 9(4): 418-435.
    • (2003) Haemophilia , vol.9 , Issue.4 , pp. 418-435
    • Wight, J.1    Paisley, S.2
  • 44
    • 84899539775 scopus 로고    scopus 로고
    • Monitoring of modified factor VIII and IX products
    • Kitchen S, Gray E, Mertens K. Monitoring of modified factor VIII and IX products. Haemophilia. 2014; 20(Suppl 4): 36-42.
    • (2014) Haemophilia , vol.20 , pp. 36-42
    • Kitchen, S.1    Gray, E.2    Mertens, K.3
  • 45
    • 0031857499 scopus 로고    scopus 로고
    • In vivo recovery with products of very high purity-assay discrepancies
    • Lusher JM, Hillman-Wiseman C, Hurst D. In vivo recovery with products of very high purity-assay discrepancies. Haemophilia. 1998; 4(4): 641-645.
    • (1998) Haemophilia , vol.4 , Issue.4 , pp. 641-645
    • Lusher, J.M.1    Hillman-Wiseman, C.2    Hurst, D.3
  • 46
    • 79959505463 scopus 로고    scopus 로고
    • International comparative field study of N8 evaluating factor VIII assay performance
    • Viuff D, Barrowcliffe T, Saugstrup T, Ezban M, Lillicrap D. International comparative field study of N8 evaluating factor VIII assay performance. Haemophilia. 2011; 17(4): 695-702.
    • (2011) Haemophilia , vol.17 , Issue.4 , pp. 695-702
    • Viuff, D.1    Barrowcliffe, T.2    Saugstrup, T.3    Ezban, M.4    Lillicrap, D.5
  • 47
    • 4844226443 scopus 로고    scopus 로고
    • Collaborative field study on the utility of a BDD factor VIII concentrate standard in the estimation of BDDr Factor VIII: C activity in hemophilic plasma using one-stage clotting assays
    • Ingerslev J, Jankowski MA, Weston SB, Charles LA. Collaborative field study on the utility of a BDD factor VIII concentrate standard in the estimation of BDDr Factor VIII: C activity in hemophilic plasma using one-stage clotting assays. J Thromb Haemost. 2004; 2(4): 623-628.
    • (2004) J Thromb Haemost , vol.2 , Issue.4 , pp. 623-628
    • Ingerslev, J.1    Jankowski, M.A.2    Weston, S.B.3    Charles, L.A.4
  • 48
    • 0036017368 scopus 로고    scopus 로고
    • Coagulation and chromogenic assays of factor VIII activity: General aspects, standardization, and recommendations
    • Barrowcliffe TW, Raut S, Sands D, Hubbard AR. Coagulation and chromogenic assays of factor VIII activity: general aspects, standardization, and recommendations. Semin Thromb Hemost. 2002; 28(3): 247-256.
    • (2002) Semin Thromb Hemost , vol.28 , Issue.3 , pp. 247-256
    • Barrowcliffe, T.W.1    Raut, S.2    Sands, D.3    Hubbard, A.R.4
  • 49
    • 84894260830 scopus 로고    scopus 로고
    • Comparative field study evaluating the activity of recombinant factor VIII Fc fusion protein in plasma samples at clinical haemostasis laboratories
    • Sommer JM, Moore N, McGuffie-Valentine B, et al. Comparative field study evaluating the activity of recombinant factor VIII Fc fusion protein in plasma samples at clinical haemostasis laboratories. Haemophilia. 2014; 20(2): 294-300.
    • (2014) Haemophilia , vol.20 , Issue.2 , pp. 294-300
    • Sommer, J.M.1    Moore, N.2    McGuffie-Valentine, B.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.