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Volumn 19, Issue 6, 2013, Pages 808-813

Pharmacokinetics of plasma-derived and recombinant factor IX - implications for prophylaxis and on-demand therapy

Author keywords

Dose tailoring; Factor IX; Pharmacokinetics

Indexed keywords

PLASMA DERIVED BLOOD CLOTTING FACTOR IX; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG;

EID: 84886640646     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12216     Document Type: Review
Times cited : (29)

References (37)
  • 2
    • 0031598275 scopus 로고    scopus 로고
    • Call for post-licensing human pharmacokinetic studies of administration of recombinant factor IX
    • Gioia KP, Bouhasin D. Call for post-licensing human pharmacokinetic studies of administration of recombinant factor IX. Haemophilia 1998; 4: 67.
    • (1998) Haemophilia , vol.4 , pp. 67
    • Gioia, K.P.1    Bouhasin, D.2
  • 3
    • 0005619513 scopus 로고    scopus 로고
    • Call for post-licensing human pharmacokinetic studies of administration of recombinant factor IX
    • Courter SG, Beach KJ. Call for post-licensing human pharmacokinetic studies of administration of recombinant factor IX. Haemophilia 1998; 4: 136-7.
    • (1998) Haemophilia , vol.4 , pp. 136-137
    • Courter, S.G.1    Beach, K.J.2
  • 4
    • 0038779279 scopus 로고    scopus 로고
    • Prophylaxis in factor IX deficiency product and patient variation
    • The Mononine Study Group
    • Kisker CT, Eisberg A, Schwartz B and the Mononine Study Group. 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
  • 5
    • 47649125876 scopus 로고    scopus 로고
    • Factor IX pharmacokinetics: differences between plasma-derived and recombinant products and the clinical and economic implications: a meeting report
    • Morfini M, Laguna P, Leissinger C. Factor IX pharmacokinetics: differences between plasma-derived and recombinant products and the clinical and economic implications: a meeting report. Haemophilia 2008; 14: 873-5.
    • (2008) Haemophilia , vol.14 , pp. 873-875
    • Morfini, M.1    Laguna, P.2    Leissinger, C.3
  • 6
    • 77954644923 scopus 로고    scopus 로고
    • Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications
    • Santagostino E. Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications. Haemophilia 2010; 16(Suppl. 6): 13-7.
    • (2010) Haemophilia , vol.16 , Issue.SUPPL. 6 , pp. 13-17
    • Santagostino, E.1
  • 7
    • 79951789201 scopus 로고    scopus 로고
    • A commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosing
    • Björkman S. A commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosing. Haemophilia 2011; 17: 179-84.
    • (2011) Haemophilia , vol.17 , pp. 179-184
    • Björkman, S.1
  • 9
    • 78751521068 scopus 로고    scopus 로고
    • Replacement therapy with recombinant factor IX. A multicentre evaluation of current dosing practices in Italy
    • Rocca A, Pizzinelli S, Oliovecchio E, Santagostino E, Rocino A, Iorio A. Replacement therapy with recombinant factor IX. A multicentre evaluation of current dosing practices in Italy. Blood Transfus 2011; 9: 60-9.
    • (2011) Blood Transfus , vol.9 , pp. 60-69
    • Rocca, A.1    Pizzinelli, S.2    Oliovecchio, E.3    Santagostino, E.4    Rocino, A.5    Iorio, A.6
  • 10
    • 0035895058 scopus 로고    scopus 로고
    • Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates
    • Roth DA, Kessler CM, Pasi KJ, Rup B, Courter SG, Tubridy KL. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001; 98: 3600-6.
    • (2001) Blood , vol.98 , pp. 3600-3606
    • Roth, D.A.1    Kessler, C.M.2    Pasi, K.J.3    Rup, B.4    Courter, S.G.5    Tubridy, K.L.6
  • 11
    • 19944429262 scopus 로고    scopus 로고
    • The safety and efficacy of recombinant blood coagulation factor IX in previously untreated patients with severe and moderately severe hemophilia B
    • Shapiro AD, Di Paola J, Cohen A et al. The safety and efficacy of recombinant blood coagulation factor IX in previously untreated patients with severe and moderately severe hemophilia B. Blood 2005; 105: 518-25.
    • (2005) Blood , vol.105 , pp. 518-525
    • Shapiro, A.D.1    Di Paola, J.2    Cohen, A.3
  • 12
    • 77953578591 scopus 로고    scopus 로고
    • Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B
    • Monahan PE, Liesner R, Sullivan ST, Ramirez ME, Kelly P, Roth DA. Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B. Haemophilia 2010; 16: 460-8.
    • (2010) Haemophilia , vol.16 , pp. 460-468
    • Monahan, P.E.1    Liesner, R.2    Sullivan, S.T.3    Ramirez, M.E.4    Kelly, P.5    Roth, D.A.6
  • 13
    • 0017259368 scopus 로고
    • Evaluation of prophylactic replacement therapy in haemophilia B
    • Morfini M, Mannucci PM, Mariani G et al. Evaluation of prophylactic replacement therapy in haemophilia B. Scand J Haematol 1976; 16: 41-7.
    • (1976) Scand J Haematol , vol.16 , pp. 41-47
    • Morfini, M.1    Mannucci, P.M.2    Mariani, G.3
  • 14
    • 0017234753 scopus 로고
    • Die ambulante Dauerbehandlung der Hämophilie B
    • Schimpf K, Baumann P. Die ambulante Dauerbehandlung der Hämophilie B. Dtsch Med Wochenshr 1976; 101: 233-8.
    • (1976) Dtsch Med Wochenshr , vol.101 , pp. 233-238
    • Schimpf, K.1    Baumann, P.2
  • 15
    • 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 Int Med 1992; 232: 25-32.
    • (1992) J Int Med , vol.232 , pp. 25-32
    • Nilsson, I.M.1    Berntorp, E.2    Löfqvist, T.3    Pettersson, H.4
  • 16
    • 77950191134 scopus 로고    scopus 로고
    • An open clinical study assessing the efficacy and safety of factor IX Grifols, a high-purity factor IX concentrate, in patients with severe haemophilia B
    • Lissitchkov T, Matysiak M, Zawilska K et al. An open clinical study assessing the efficacy and safety of factor IX Grifols, a high-purity factor IX concentrate, in patients with severe haemophilia B. Haemophilia 2010; 16: 240-6.
    • (2010) Haemophilia , vol.16 , pp. 240-246
    • Lissitchkov, T.1    Matysiak, M.2    Zawilska, K.3
  • 17
    • 79959499738 scopus 로고    scopus 로고
    • A clinical study assessing the pharmacokinetics, efficacy and safety of AlphaNine, a high-purity factor IX concentrate, in patients with severe haemophilia B
    • Lissitchkov T, Matysiak M, Zavilska K et al. A clinical study assessing the pharmacokinetics, efficacy and safety of AlphaNine, a high-purity factor IX concentrate, in patients with severe haemophilia B. Haemophilia 2011; 17: 590-6.
    • (2011) Haemophilia , vol.17 , pp. 590-596
    • Lissitchkov, T.1    Matysiak, M.2    Zavilska, K.3
  • 18
    • 0037764674 scopus 로고    scopus 로고
    • Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective
    • Björkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia 2003; 9(Suppl. 1): 101-10.
    • (2003) Haemophilia , vol.9 , Issue.SUPPL. 1 , pp. 101-110
    • Björkman, S.1
  • 19
    • 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, Björkman S. Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia 2011; 17: 2-10.
    • (2011) Haemophilia , vol.17 , pp. 2-10
    • Collins, P.W.1    Fischer, K.2    Morfini, M.3    Blanchette, V.S.4    Björkman, S.5
  • 20
    • 84862872608 scopus 로고    scopus 로고
    • Personalized prophylaxis
    • Collins PW. Personalized prophylaxis. Haemophilia 2012; 18(Suppl. 4): 131-5.
    • (2012) Haemophilia , vol.18 , Issue.SUPPL. 4 , pp. 131-135
    • Collins, P.W.1
  • 21
    • 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
  • 22
    • 84862908041 scopus 로고    scopus 로고
    • Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients
    • Shapiro AD, Ragni MV, Valentino LA et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood 2012; 119: 666-72.
    • (2012) Blood , vol.119 , pp. 666-672
    • Shapiro, A.D.1    Ragni, M.V.2    Valentino, L.A.3
  • 23
    • 84868152441 scopus 로고    scopus 로고
    • Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX
    • Collins PW, Møss J, Knobe K, Groth A, Colberg T, Watson E. Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX. J Thromb Haemost 2012; 10: 2305-12.
    • (2012) J Thromb Haemost , vol.10 , pp. 2305-2312
    • Collins, P.W.1    Møss, J.2    Knobe, K.3    Groth, A.4    Colberg, T.5    Watson, E.6
  • 24
    • 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
  • 25
    • 27744581791 scopus 로고    scopus 로고
    • The design and analysis of pharmacokinetic studies of coagulation factors
    • and the Factor VIII/Factor IX Scientific and Standardization Committee of the International Society for Thrombosis and Haemostasis. Available at. Accessed May 10, 2013.
    • Lee M, Morfini M, Schulman S, Ingerslev J and the Factor VIII/Factor IX Scientific and Standardization Committee of the International Society for Thrombosis and Haemostasis. The design and analysis of pharmacokinetic studies of coagulation factors. 2001. Available at http://www.isth.org/Publications/OfficialCommunications/Factor8and9/Pharmacokinetic.aspx. Accessed May 10, 2013.
    • (2001)
    • Lee, M.1    Morfini, M.2    Schulman, S.3    Ingerslev, J.4
  • 26
    • 79951794547 scopus 로고    scopus 로고
    • Population pharmacokinetic modeling of BeneFIX in pediatric and adult patients with hemophilia B demonstrates weight as an important factor contributing to inter-patient PK variability
    • Abstract 1218.
    • Udata C, Sullivan S, Kelly P, Roth DA, Meng X. Population pharmacokinetic modeling of BeneFIX in pediatric and adult patients with hemophilia B demonstrates weight as an important factor contributing to inter-patient PK variability. Blood 2008; 112: Abstract 1218.
    • (2008) Blood , vol.112
    • Udata, C.1    Sullivan, S.2    Kelly, P.3    Roth, D.A.4    Meng, X.5
  • 27
    • 84864280808 scopus 로고    scopus 로고
    • Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis
    • Björkman S, Åhlén V. Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis. Eur J Clin Pharmacol 2012; 68: 969-77.
    • (2012) Eur J Clin Pharmacol , vol.68 , pp. 969-977
    • Björkman, S.1    Åhlén, V.2
  • 28
    • 84883052367 scopus 로고    scopus 로고
    • Population pharmacokinetics of recombinant factor IX: implications for dose tailoring
    • doi:10.1111/hae.12188.
    • Björkman S. Population pharmacokinetics of recombinant factor IX: implications for dose tailoring. Haemophilia 2013; 19: doi:10.1111/hae.12188.
    • (2013) Haemophilia , vol.19
    • Björkman, S.1
  • 29
    • 0031028921 scopus 로고    scopus 로고
    • The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications
    • Björkman S, Carlsson M. The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications. Haemophilia 1997; 3: 1-8.
    • (1997) Haemophilia , vol.3 , pp. 1-8
    • Björkman, S.1    Carlsson, M.2
  • 30
    • 84873038634 scopus 로고    scopus 로고
    • Collins P; for the Project on Factor VIII/Factor IX Pharmacokinetics of the Factor VIII/Factor IX Scientific and Standardization Committee of the ISTH. Measurement of factor VIII pharmacokinetics in routine clinical practice
    • Björkman S. Collins P; for the Project on Factor VIII/Factor IX Pharmacokinetics of the Factor VIII/Factor IX Scientific and Standardization Committee of the ISTH. Measurement of factor VIII pharmacokinetics in routine clinical practice. J Thromb Haemost 2013; 11: 180-2.
    • (2013) J Thromb Haemost , vol.11 , pp. 180-182
    • Björkman, S.1
  • 31
    • 0028365342 scopus 로고
    • Pharmacokinetics of factor IX in patients with haemophilia B: methodological aspects and physiological interpretation
    • Björkman S, Carlsson M, Berntorp E. Pharmacokinetics of factor IX in patients with haemophilia B: methodological aspects and physiological interpretation. Eur J Clin Pharmacol 1994; 46: 325-32.
    • (1994) Eur J Clin Pharmacol , vol.46 , pp. 325-332
    • Björkman, S.1    Carlsson, M.2    Berntorp, E.3
  • 32
    • 0029043733 scopus 로고
    • Variability of in vivo recovery of factor IX after infusion of monoclonal antibody purified factor IX concentrates in patients with hemophilia B
    • the Mononine Study Group
    • White GC, Shapiro AD, Kurczynski EM, Kim HC, Bergman GE, the Mononine Study Group. Variability of in vivo recovery of factor IX after infusion of monoclonal antibody purified factor IX concentrates in patients with hemophilia B. Thromb Haemost 1995; 73: 779-84.
    • (1995) Thromb Haemost , vol.73 , pp. 779-784
    • White, G.C.1    Shapiro, A.D.2    Kurczynski, E.M.3    Kim, H.C.4    Bergman, G.E.5
  • 33
    • 0036123458 scopus 로고    scopus 로고
    • Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study
    • Poon M-C, Lillicrap D, Hensman C, Card R, Scully MF. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thromb Haemost 2002; 87: 431-5.
    • (2002) Thromb Haemost , vol.87 , pp. 431-435
    • Poon, M.-C.1    Lillicrap, D.2    Hensman, C.3    Card, R.4    Scully, M.F.5
  • 34
    • 33845730827 scopus 로고    scopus 로고
    • In vivo recovery of factor VIII and factor IX: intra- and interindividual variance in a clinical setting
    • Björkman S, Folkesson A, Berntorp E. In vivo recovery of factor VIII and factor IX: intra- and interindividual variance in a clinical setting. Haemophilia 2007; 13: 2-8.
    • (2007) Haemophilia , vol.13 , pp. 2-8
    • Björkman, S.1    Folkesson, A.2    Berntorp, E.3
  • 35
    • 65449121407 scopus 로고    scopus 로고
    • Recovery of recombinant factor IX determined in clinical practice
    • Martorell M, Altisent C, Parra R. Recovery of recombinant factor IX determined in clinical practice. Haemophilia 2009; 15: 840-2.
    • (2009) Haemophilia , vol.15 , pp. 840-842
    • Martorell, M.1    Altisent, C.2    Parra, R.3
  • 36
    • 77953478039 scopus 로고    scopus 로고
    • Atlanta, GA: Clinical Growth Charts. Available at Accessed July 30, 2012.
    • Centers for Disease Control and Prevention. Atlanta, GA: Clinical Growth Charts. Available at http://www.cdc.gov/growthcharts/ Accessed July 30, 2012.
    • Centers for Disease Control and Prevention
  • 37
    • 0035081112 scopus 로고    scopus 로고
    • Pharmacokinetics of recombinant factor IX in relation to age of the patient: implications for dosing in prophylaxis
    • Björkman S, Shapiro AD, Berntorp E. Pharmacokinetics of recombinant factor IX in relation to age of the patient: implications for dosing in prophylaxis. Haemophilia 2001; 7: 133-9.
    • (2001) Haemophilia , vol.7 , pp. 133-139
    • Björkman, S.1    Shapiro, A.D.2    Berntorp, E.3


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