A commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosing

Haemophilia

Volumn 17, Issue 2, 2011, Pages 179-184

  Bjorkman S ^a  

^a UPPSALA UNIVERSITY   (Sweden)

Author keywords

Dose tailoring; Factor IX; Pharmacokinetics

Indexed keywords

BLOOD CLOTTING FACTOR 9; PROTHROMBIN COMPLEX; RECOMBINANT BLOOD CLOTTING FACTOR 9;

ARTICLE; BLOOD SAMPLING; DRUG CLEARANCE; DRUG HALF LIFE; HEMOPHILIA B; HUMAN; IN VIVO STUDY; PRIORITY JOURNAL; PROPHYLAXIS;

ADOLESCENT; ADULT; AGED; CHILD; CHILD, PRESCHOOL; FACTOR IX; HALF-LIFE; HEMOPHILIA B; HUMANS; MIDDLE AGED; RECOMBINANT PROTEINS; YOUNG ADULT;

EID: 79951789201     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2010.02431.x     Document Type: Article

References (28)

1. White GC, Beebe A, Nielsen B. Recombinant factor IX. Thromb Haemost 1997; 78: 261-5.
2. Kisker CT, Eisberg A, Schwartz B, the Mononine Study Group. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia 2003; 9: 279-84.
3. 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.
4. Martorell M, Altisent C, Parra R. Recovery of recombinant factor IX determined in clinical practice. Haemophilia 2009; 15: 840-2.
5. Santagostino E. Prophylaxis in haemophilia B patients: unresolved issues and pharmacoeconomic implications. Haemophilia 2010; 16(Suppl. 6): 13-7.
6. White G, Shapiro A, Ragni M et al. Clinical evaluation of recombinant factor IX. Semin Haematol 1998; 35: 33-8.
7. Ewenstein BM, Joist JH, Shapiro AD et al. Pharmacokinetic analysis of plasma-derived and recombinant FIX concentrates in previously treated patients with moderate or severe haemophilia B. Transfusion 2002; 42: 190-7.
8. 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.
9. Protocols for the Treatment of Hemophilia and Von Willebrand Disease, 3rd edn. Treatment of Hemophilia monograph no 14, April 2008. Available at Accessed June 16, 2010.
10. Carlsson M, Björkman S, Berntorp E. Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia 1998; 4: 83-8.
11. 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.
12. Björkman S, Carlsson M. The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications. Haemophilia 1997; 3: 1-8.
13. 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.
14. BeneFIX (Wyeth Europe, Maidenhead, UK) Summary of Product Characteristics. Available at Accessed March 25, 2010.
15. 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.
16. Ragni MV, Pasi KJ, White GC et al. Use of recombinant factor IX in subjects with haemophilia B undergoing surgery. Haemophilia 2002; 8: 91-7.
17. Lambert T, Recht M, Valentino LA et al. Reformulated BeneFIX: efficacy and safety in previously treated patients with moderately severe to severe haemophilia B. Haemophilia 2007; 13: 233-43.
18. Chang HH, Yang YL, Hung MH, Tsay W, Shen MC. Pharmacokinetic study of recombinant human factor IX in previously treated patients with hemophilia B in Taiwan. J Formos Med Assoc 2007; 106: 281-7.
19. Hoag MS, Johnson FF, Robinson JA, Aggeler PM. Treatment of hemophilia B with a new clotting factor concentrate. New Engl J Med 1969; 280: 581-6.
20. Berntorp E, Björkman S, Carlsson M, Lethagen S, Nilsson IM. Biochemical and in vivo properties of high purity factor IX concentrates. Thrombos Haemostas 1993; 70: 768-73.
21. 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.
22. Goudemand J, Peynet J, Chambost H et al. A cross-over pharmacokinetic study of a double viral inactivated factor IX concentrate (15 nm filtration and SD) compared to a SD factor IX concentrate. Thromb Haemost 1998; 80: 919-24.
23. Hoots WK, Leissinger C, Stabler S et al. Continuous intravenous infusion of a plasma-derived factor IX concentrate (Mononine) in haemophilia B. Haemophilia 2003; 9: 164-72.
24. Aznar JA, Cabrera N, Matysiak M et al. Pharmacokinetic study of a high-purity factor IX concentrate (Factor IX Grifols) with a 6-month follow up in previously treated patients with severe haemophilia B. Haemophilia 2009; 15: 1243-8.
25. 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.
26. 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.
27. Björkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia 2003; 9(Suppl. 1): 101-10.
28. Collins PW, Björkman S, Fischer K et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe haemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemostas 2009; 8: 269-75.