Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: Implications for dosing in prophylaxis

European Journal of Clinical Pharmacology

Volumn 68, Issue 6, 2012, Pages 969-977

  Björkman, Sven ^a   Åhlén, Victor ^a  

^a UPPSALA UNIVERSITY   (Sweden)

Author keywords

Dosing; Factor IX; Haemophilia B; Pharmacokinetics; Prophylaxis

Indexed keywords

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

ADOLESCENT; ADULT; AGED; ARTICLE; BLOOD CLOTTING; CHEMOPROPHYLAXIS; CLINICAL ARTICLE; CONTINUOUS INFUSION; DISEASE SEVERITY; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG ELIMINATION; DRUG HALF LIFE; HEMOPHILIA B; HUMAN; MALE; POPULATION RESEARCH; PRIORITY JOURNAL;

ADOLESCENT; ADULT; AGED; COHORT STUDIES; FACTOR IX; HALF-LIFE; HEMOPHILIA B; HUMANS; MIDDLE AGED; MODELS, BIOLOGICAL; YOUNG ADULT;

EID: 84864280808     PISSN: 00316970     EISSN: 14321041     Source Type: Journal    
DOI: 10.1007/s00228-012-1211-z     Document Type: Article

References (38)

1. Escobar MA (2003) Treatment on demand - in vivo dose finding studies. Haemophilia 9:360-367 (Pubitemid 36874382)
2. World Federation of Hemophilia (2008) Protocols for the treatment of hemophilia and von Willebrand disease, 3rd edn. Treatment of Hemophilia monograph no 14. Available at: http://www.wfh.org/2/docs/Publications Accessed 16 June 2010.
3. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H (1992) Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Int Med 232:25-32
4. Björkman S (2003) Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia 9 [Suppl 1]:101-110 (Pubitemid 36622057)
5. Collins PW, Fischer K, Morfini M, Blanchette VS, Björkman S (2011) Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia. Haemophilia 17:2-10
6. Björkman S, Carlsson M (1997) The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications. Haemophilia 3:1-8 (Pubitemid 27033930)
7. Björkman S, Berntorp E (2001) Pharmacokinetics of coagulation factors: clinical relevance for patients with haemophilia. Clin Pharmacokinet 40:815-832 (Pubitemid 33095998)
8. Björkman S (2011) A commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosing. Haemophilia 17:179-184
9. Björkman S, Carlsson M, Berntorp E (1994) Pharmacokinetics of factor IX in patients with haemophilia B: methodological aspects and physiological interpretation. Eur J Clin Pharmacol 46:325-332 (Pubitemid 24167380)
10. The European Agency for the Evaluation of Medicinal Products (EMEA), Committee for Proprietary Medicinal Products (CPMP) (2000). Note for guidance on the clinical investigation of human plasma derived factor VIII and IX products (CPMP/BPWG/198/95 rev. 1). Available at: http://www.ema.europa.eu/docs/en-GB/ document-library/Scientific-guideline/2009/09/WC500003621.pdf Accessed 12 July 2011
11. Hoag MS, Johnson FF, Robinson JA, Aggeler PM (1969) Treatment of hemophilia B with a new clotting factor concentrate. New Engl J Med 280:581-586
12. Berntorp E, Björkman S, Carlsson M, Lethagen S, Nilsson IM (1993) Biochemical and in vivo properties of high purity factor IX concentrates. Thrombos Haemostas 70:768-773 (Pubitemid 23339450)
13. White GC, Beebe A, Nielsen B (1997) Recombinant factor IX. Thromb Haemost 78:261-265 (Pubitemid 27289251)
14. Goudemand J, Peynet J, Chambost H, Négrier C, Briquel ME, Claeyssens S, Derlon-Borel A, Guérois C, Caron C, Scherrmann JM, Debray M, Bridey F (1998) 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 80:919-924 (Pubitemid 28556693)
15. Aznar JA, Cabrera N, Matysiak M, Zawilska K, Gercheva L, Antonov A, Montañés M, Páez AM, Lissitchkov T (2009) 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 15:1243-1248
16. Lissitchkov T, Matysiak M, Zawilska K, & Lstrok;aguna P, Gercheva L, Antonov A, Cabrera N, Aznar JA, Woodward MK, Páez A (2011) A clinical study assessing the pharmacokinetics, efficacy and safety of AlphaNine, a high-purity factor IX concentrate, in patients with severe haemophilia B. Haemophilia 17:590-596
17. Serban M, Skotnicki AB, Colovic M, Jinca C, Klukowska A, Laguna P, Wolf DM (2011) Clinical efficacy, safety and pharmacokinetic properties of the plasma-derived factor IX concentrate Haemonine in previously treated patients with severe haemophilia B. Haemophilia 17. doi:10.1111/j.1365-2516.2011.02624.x
18. Fijnvandraat K, Berntorp E, ten Cate JW, Johnsson H, Peters M, Savidge G, Tengborn L, Spira J, Stahl C (1997) Recombinant, Bdomain deleted factor VIII (r-VIII SQ): Pharmacokinetics and initial safety aspects in hemophilia A patients. Thromb Haemost 77:298-302 (Pubitemid 27084919)
19. Björkman S, Folkesson A, Jönsson S (2009) Pharmacokinetics and dose requirements of factor VIII over the age range 3-74 years: A population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol 65:989-998
20. Björkman S, Blanchette VS, Fischer K, Oh M, Spotts G, Schroth P, Fritsch S, Patrone L, Ewenstein BW, for the ADVATE Clinical Program Group, Collins PW (2010) Comparative pharmacokinetics of plasma- and albumin-free recombinant factor VIII in children and adults: the influence of blood sampling schedule on observed age-related differences and implications for dose tailoring. J Thrombos Haemostas 8:730-736
21. Carlsson M, Björkman S, Berntorp E (1998) Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia 4:83-88 (Pubitemid 28165684)
22. Björkman S, Shapiro AD, Berntorp E (2001) Pharmacokinetics of recombinant factor IX in relation to age of the patient: implications for dosing in prophylaxis. Haemophilia 7:133-139 (Pubitemid 32243794)
23. Ahnström J, Berntorp E, Lindvall K, Björkman S (2004) A sixyear follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia. Haemophilia 10:689-697 (Pubitemid 40028584)
24. Giddings JC (1980) Hereditary coagulation disorders: laboratory techniques. In: Thomson JM (ed) Blood coagulation and haemostasis. Churchill Livingstone, New York, pp 117-157
25. Karlsson MO, Sheiner LB (1993) The importance of modeling interoccasion variability in population pharmacokinetic analyses. J Pharmacokinet Biopharm 21:735-750 (Pubitemid 24052760)
26. Jonsson EN, Karlsson MO (1999) Xpose - an S-PLUS based population pharmacokinetic-pharmacodynamic model building aid for NONMEM. Comput Methods Programs Biomed 58:51-64 (Pubitemid 29049052)
27. Meibohm B, Läer S, Panetta JC, Barrett JS (2005) Population pharmacokinetic studies in pediatrics: issues in design and analysis. AAPS J 7:475-787
28. Thomson AH, Whiting B (1992) Bayesian parameter estimation and population pharmacokinetics. Clin Pharmacokin 22:447-467
29. Upton RN (2004) Calculating the hybrid (macro) rate constants of a three-compartment mammillary pharmacokinetic model from known micro-rate constants. J Pharmacol Toxicol Method 49:65-68 (Pubitemid 37506056)
30. Karlsson MO, Savic RM (2007) Diagnosing model diagnostics. Clin Pharmacol Ther 82:17-20 (Pubitemid 46944205)
31. Savic RM, Karlsson MO (2009) Importance of shrinkage in empirical Bayes estimates for diagnostics: problems and solutions. AAPS J 11:558-569
32. Karlsson MO, Holford N. A tutorial on visual predictive checks. PAGE 17 (2008) Abstract 1434. Available at: http://www.pagemeeting.org/default.asp? abstract01434. Accessed 12 July 2011 © Springer-Verlag 2012.
33. Stonebraker JS, Bolton-Maggs PHB, Soucie JM, Walkers I, Brooker M (2011) A study of variations in the reported haemophilia B prevalence around the world. Haemophilia 17. doi:10.1111/j.13652516.2011.02588.x
34. Chitlur M, Warrier I, Rajpurkar M, Lusher JM (2009) Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006). Haemophilia 15:1027-1031
35. Holford NHG (1996) A size standard for pharmacokinetics. Clin Pharmacokinet 30:329-332 (Pubitemid 26177179)
36. Collins PW, Björkman S, Fischer K, Blanchette V, Oh M, Schroth P, Fritsch S, Casey K, Spotts G, Ewenstein BW (2010) 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 Thrombos Haemostas 8:269-275
37. Schimpf K, Baumann P (1976) Die ambulante Dauerbehandling der Hämophilie B; eine kontrollierte Studie (A controlled study of long-term treatment of haemophilia B on an out-patient basis). Dtsch Med Wochenschr 101:233-238
38. Björkman S (2010) Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia 16:597-605