Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia

Haemophilia

Volumn 17, Issue 1, 2011, Pages 2-10

  Collins, P W ^a   Fischer, K ^b,c   Morfini, M ^d   Blanchette, V S ^e   Bjorkman S ^f  

^a CARDIFF UNIVERSITY   (United Kingdom)

^b Van Creveldkliniek ^*  (United Kingdom)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^d University Hospital   (Italy)

^e HOSPITAL FOR SICK CHILDREN   (Canada)

^f UPPSALA UNIVERSITY   (Sweden)

Author keywords

Break through bleeding; Half life; Pharmacokinetics; Prophylaxis; Trough

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9;

CLINICAL PRACTICE; COST EFFECTIVENESS ANALYSIS; HALF LIFE TIME; HEALTH CARE SYSTEM; HEMOPHILIA; HUMAN; IMMUNOLOGICAL TOLERANCE; PRIORITY JOURNAL; REVIEW; TREATMENT OUTCOME;

ADULT; CHILD; DRUG ADMINISTRATION SCHEDULE; FACTOR IX; FACTOR VIII; HEMOPHILIA A; HEMOSTASIS; HUMANS; IMMUNE TOLERANCE;

EID: 79951472629     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/j.1365-2516.2010.02370.x     Document Type: Review

References (53)

1. Björkman S, Folkesson A, Jonsson S. Pharmacokinetics and dose requirements of factor VIII over the age range of 3-74years: a population analysis based on 50 patients with long-term prophylactic treatment for haemophilia A. Eur J Clin Pharmacol 2009; 65: 989-98.
2. Björkman S, Blanchette VS, Fischer K et al. 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 Thromb Haemost 2010; 8: 730-6.
3. Messori A, Longo G, Morfini M et al. Individualizing of factor VIII dosage. J Clin Hosp Pharm 1984; 9: 95-103.
4. Messori A, Longo G, Matucci M, Morfini M, Rossi Ferrini PL. Clinical pharmacokinetics of factor VIII in patients with classic haemophilia. Clin Pharmacokin 1987; 13: 365-80.
5. Carlsson M, Berntorp E, Björkman S, Lindvall K. Pharmacokinetic dosing in prophylactic treatment of hemophilia A. Eur J Haematol 1993; 51: 247-52.
6. Björkman S, Carlsson M. The pharmacokinetics of factor VIII and factor IX: methodology, pitfalls and applications. Haemophilia 1997; 3: 1-8.
7. Carlsson M, Berntorp E, Björkman S, Lethagen S, Ljung R. Improved cost-effectiveness by pharmacokinetic dosing of factor VIII in prophylactic treatment of haemophilia A. Haemophilia 1997; 3: 96-101.
8. Carlsson M, Björkman S, Berntorp E. Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia 1998; 4: 83-8.
9. 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.
10. Björkman S, Berntorp E. Pharmacokinetics of coagulation factors: clinical relevance for patients with haemophilia. Clin Pharmacokinet 2001; 40: 815-32.
11. Björkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia 2003; 1(Suppl. 9): 101-10.
12. Shapiro AD, Korth-Bradley J, Poon MC. Use of pharmacokinetics in the coagulation factor treatment of patients with haemophilia. Haemophilia 2005; 11: 571-82.
13. 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 Thrombos Haemostas 2010; 8: 269-75.
14. Matucci M, Messori A, Donati-Cori G et al. Kinetic evaluation of four factor VIII concentrates by model-independent methods. Scand J Haematol 1985; 34: 22-8.
15. van Dijk K, van der Bom JG, Lenting PJ et al. Factor VIII half-life and clinical phenotype of severe hemophilia A. Haematologica 2005; 90: 494-8.
16. Blanchette VS, Shapiro AD, Liesner RJ et al. Plasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost 2008; 6: 1319-26.
17. Lee M, Morfini M, Schulman S, Ingerslev J, 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. Available at: Accessed September 22, 2008.
18. Sheiner LB, Beal S, Rosenberg B, Marathe VV. Forecasting individual pharmacokinetics. Clin Pharmacol Ther 1979; 26: 294-305.
19. Ruffo S, Messori A, Grasela TH et al. A calculator program for clinical application of the Bayesian method of predicting plasma drug levels. Comput Progr Biomed 1985; 19: 167-77.
20. Björkman S. Limited blood sampling for pharmacokinetic dose tailoring of FVIII in the prophylactic treatment of haemophilia A. Haemophilia 2010; 16: 597-605.
21. 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: 428-37.
22. Fijnvandraat K, Berntorp E, ten Cate JW et al. Recombinant, B-domain deleted factor VIII (r-VIII SQ): pharmacokinetics and initial safety aspects in hemophilia A patients. Thromb Haemost 1997; 77: 298-302.
23. 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 1965; 77(Suppl.): 5-99.
24. 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.
25. 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: 1109-13.
26. Fischer K, Astermark J, van der Bom JG et al. Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen. Haemophilia 2002; 8: 753-60.
27. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe haemophilia. N Engl J Med 2007; 9: 35-44.
28. den Uijl IE, Fischer K, van der Bom JG, Grobbee DE, Rosendaal FR, Plug I. Clinical outcome of moderate haemophilia compared with severe and mild haemophilia. Haemophilia 2009; 15: 83-90.
29. Ahnström J, Berntorp E, Lindvall K, Björkman S. A 6-year follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia. Haemophilia 2004; 10: 689-97.
30. 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 haemophilia A. J Thromb Haemost 2009; 7: 413-20.
31. Petrini P. What factors should influence the dosage and interval of prophylactic treatment in patients with severe haemophilia A and B. Haemophilia 2001; 7: 99-102.
32. Quick AJ, Hussey CV. Hemophilia B (PTC deficiency, or Christmas disease). AMA Arch Intern Med. 1959; 103: 762-75.
33. Pai KM, Walker I, Almonte T, Chan AK, Sek J, Deker K. Comparing bleed frequency and factor concentrates use between haemophilia A and haemophilia B. J Thromb Haemostas 2005; 3(Suppl. 1): abstract P00807.
34. Biss TT, Chan AK, Blanchette VS et al. The use of prophylaxis in 2663 children and adults with haemophilia: results of the 2006 Canadian national haemophilia prophylaxis survey. Haemophilia 2008; 14: 923-30.
35. Tagariello G, Iorio A, Santagostino E et al. Comparison of the rates of joint arthroplasty in patients with severe factor VIII and IX deficiency: an index of different clinical severity of the 2 coagulation disorders. Blood 2009; 114: 779-84.
36. 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.
37. White G, Shapiro A, Ragni M et al. Clinical evaluation of recombinant factor IX. Semin Hematol 1998; 35: 33-8.
38. Ewenstein BM, Joist JH, Shapiro AD et al. Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B. Transfusion 2002; 42: 190-7.
39. Roth DA, Kessler CM, Pasi KJ, Rup B, Courter SG, Tubridy KL. Human recombinant factor IX: safety and efficacy studies in haemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001; 98: 3600-6.
40. 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.
41. 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.
42. Collins P, Faradji A, Morfini M, Enriquez MM, Schwartz L. Efficacy and safety of secondary prophylactic versus on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia a: results from a 13-month crossover study. J Thromb Haemost 2009; 8: 83-9.
43. Poon MC, Lillicrap D, Hensman C, Card R, Scully MF. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study. Thrombos Haemost 2002; 87: 431-5.
44. van Dijk K, Fischer K, Van der Bom JG, Grobbee DE, Van den Berg HM. Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed. Haemophilia 2005; 11: 438-43.
45. Kasper CK, Dietrich SL, Rapaport SI. Hemophilia prophylaxis with factor VIII concentrate. Arch Int Med 1970; 125: 1004-9.
46. Schimpf K, Fischer B, Rothmann P. Hemophilia A prophylaxis with factor VIII concentrate in a home treatment program: a controlled study. Scand J Haematol Supple 1977; 30: 79-80.
47. Blombäck M, Kjellman H, Allain JP, Hedner U, Schimpf K, Wiechel B. On the unreliability of one-stage factor VIII:C clotting assays after infusion of factor VIII concentrates. Scand J Clin Lab Invest 1987; 47: 561-6.
48. Björkman S, Carlsson M, Berntorp E, Stenberg P. Pharmacokinetics of factor VIII in humans. Obtaining clinically relevant data from comparative studies. Clin Pharmacokinet 1992; 22: 385-95.
49. 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.
50. Longo G, Matucci M, Messori A, Morfini M, Rossi-Ferrini P. Pharmacokinetics of a new heat-treated concentrate of factor VIII estimated by model-independent methods. Thromb Res 1986; 42: 471-6.
51. Longo G, Cinotti S, Filimberti E et al. Single-dose pharmacokinetics of factor IX evaluated by model-independent methods. Eur J Haematol 1987; 39: 426-33.
52. Morfini M. Pharmacokinetics studies: international guidelines for the conduct and interpretation of suc studies. Haemophilia 2006; 12(Suppl. 4): 6-11.
53. Stass H. Determination of minimal sampling time points for reliable pharmacokinetics evaluation of recombinant factor VIII - an exploratory population pharmacokinetic analysis in pediatric patients suffering from severe haemophilia. Haemophilia 2006; 12(Suppl. 4): 50-5.