Parameters influencing FVIII pharmacokinetics in patients with severe and moderate haemophilia A

Haemophilia

Volumn 21, Issue 3, 2015, Pages 343-350

  Kepa, S ^a   Horvath, B ^a   Reitter Pfoertner, S ^a   Schemper, M ^a   Quehenberger, P ^a   Grundbichler, M ^b   Heistinger, M ^c   Neumeister, P ^d   Mannhalter, C ^a   Pabinger, I ^a  

^a MEDICAL UNIVERSITY OF VIENNA   (Austria)

^b UNIVERSITY OF SALZBURG   (Austria)

^c Department of Internal Medicine I   (Austria)

^d MEDICAL UNIVERSITY OF GRAZ   (Austria)

Author keywords

Haemophilia A; Influences on FVIII pharmacokinetics; LRP1; LRP1 polymorphisms; Protein C

Indexed keywords

BLOOD CLOTTING FACTOR 8; LOW DENSITY LIPOPROTEIN RECEPTOR RELATED PROTEIN; PROTEIN C; RECOMBINANT BLOOD CLOTTING FACTOR 8; BLOOD GROUP ABO SYSTEM; RECOMBINANT PROTEIN; VON WILLEBRAND FACTOR;

ADULT; AGE; ARTICLE; BLOOD GROUP O; CLINICAL ARTICLE; DRUG CLEARANCE; DRUG HALF LIFE; GENE FREQUENCY; HEMOPHILIA A; HUMAN; INTRON; POINT MUTATION; PRIORITY JOURNAL; BLOOD; BLOOD CLOTTING; BLOOD GROUP ABO SYSTEM; BODY MASS; GENETICS; HALF LIFE TIME; MALE; MUTATION; SEVERITY OF ILLNESS INDEX; SINGLE NUCLEOTIDE POLYMORPHISM; TREATMENT OUTCOME; YOUNG ADULT;

ABO BLOOD-GROUP SYSTEM; ADULT; AGE FACTORS; BLOOD COAGULATION; BODY MASS INDEX; FACTOR VIII; HALF-LIFE; HEMOPHILIA A; HUMANS; LOW DENSITY LIPOPROTEIN RECEPTOR-RELATED PROTEIN-1; MALE; MUTATION; POLYMORPHISM, SINGLE NUCLEOTIDE; RECOMBINANT PROTEINS; SEVERITY OF ILLNESS INDEX; TREATMENT OUTCOME; VON WILLEBRAND FACTOR; YOUNG ADULT;

EID: 84928828967     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.12592     Document Type: Article

References (36)

1. Collins PW, Björkman S, Fischer K et al. Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens. J Thromb Haemost 2010; 8: 269-75.
2. 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.
3. Manco-Johnson MJ, Abshire TC, Shapiro AD et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med 2007; 357: 535-44.
4. 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.
5. 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.
6. 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.
7. 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.
8. Henrard S, Speybroeck N, Hermans C. Body weight and fat mass index as strong predictors of factor VIII in vivo recovery in adults with hemophilia A. J Thromb Haemost 2011; 9: 1784-90.
9. Henrard S, Speybroeck N, Hermans C. Impact of being underweight or overweight on factor VIII dosing in hemophilia A patients. Haematologica 2013; 98: 1481-6.
10. Björkman S, Folkesson A, Jönsson S. 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 2009; 65: 989-98.
11. Fischer K, Pendu R, van Schooten CJ et al. Models for prediction of factor VIII half-life in severe haemophiliacs: distinct approaches for blood group O and non-O patients. PLoS ONE 2009; 4: e6745.
12. Bovenschen N, van Dijk KW, Havekes LM, Mertens K, van Vlijmen BJM. Clearance of coagulation factor VIII in very low-density lipoprotein receptor knockout mice. Br J Haematol 2004; 126: 722-5.
13. Saenko EL, Yakhyaev AV, Mikhailenko I, Strickland DK, Sarafanov AG. Role of the low density lipoprotein-related protein receptor in mediation of factor VIII catabolism. J Biol Chem 1999; 274: 37685-92.
14. Quinn KA, Pye VJ, Dai YP, Chesterman CN, Owensby DA. Characterization of the soluble form of the low density lipoprotein receptor-related protein (LRP). Exp Cell Res 1999; 251: 433-41.
15. Bovenschen N, Mertens K, Hu L, Havekes LM, van Vlijmen BJM. LDL receptor cooperates with LDL receptor-related protein in regulating plasma levels of coagulation factor VIII in vivo. Blood 2005; 106: 906-12.
16. Bovenschen N, Herz J, Grimbergen JM et al. Elevated plasma factor VIII in a mouse model of low-density lipoprotein receptor-related protein deficiency. Blood 2003; 101: 3933-9.
17. Bovenschen N, van Stempvoort G, Voorberg J, Mertens K, Meijer AB. Proteolytic cleavage of factor VIII heavy chain is required to expose the binding-site for low-density lipoprotein receptor-related protein within the A2 domain. J Thromb Haemost 2006; 4: 1487-93.
18. Marchetti G, Lunghi B, Legnani C et al. Contribution of low density lipoprotein receptor-related protein genotypes to coagulation factor VIII levels in thrombotic women. Haematologica 2006; 91: 1261-3.
19. Morange PE, Tregouet DA, Frere C et al. Biological and genetic factors influencing plasma factor VIII levels in a healthy family population: results from the Stanislas cohort. Br J Haematol 2005; 128: 91-9.
20. Fulcher CA, Gardiner JE, Griffin JH, Zimmerman TS. Proteolytic inactivation of human factor VIII procoagulant protein by activated human protein C and its analogy with factor V. Blood 1984; 63: 486-9.
21. Gennari LC, Blanco AN, Domínguez MP, Grosso SH, Lazzari MA. Endogenous or exogenous coagulation factor level and the response to activated protein C. Thromb Res 2006; 118: 269-73.
22. Lee M, Morfini M, Negrier C, Chamouard V. The pharmacokinetics of coagulation factors. Haemophilia 2006; 12(Suppl 3): 1-7.
23. Lee M, Morfini M, Schulman S, Ingerslev J and the FVIII/FIX Scientific and Standardization Committee. Scientific and Standardization Committee Communication: the Design and Analysis of Pharmacokinetic Studies of Coagulation Factors. Available at http://c.ymcdn.com/sites/www.isth.org/resource/group/d4a6f49a-f4ec-450f-9e0f-7be9f0c2ab2e/official_communications/fviiipharmaco.pdf. Accessed April 02, 2014.
24. 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.
25. Björkman S. Evaluation of the TCIWorks Bayesian computer program for estimation of individual pharmacokinetics of FVIII. Haemophilia 2011; 17: e239-40.
26. Björkman S, Oh M, Spotts G et al. Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight. Blood 2012; 119: 612-8.
27. Verbruggen B, Novakova I, Wessels H, Boezeman J, van den Berg M, Mauser-Bunschoten E. The Nijmegen modification of the Bethesda assay for factor VIII: C inhibitors: improved specificity and reliability. Thromb Haemost 1995; 73: 247-51.
28. Fijnvandraat K, Peters M, ten Cate JW. Inter-individual variation in half-life of infused recombinant factor VIII is related to pre-infusion von Willebrand factor antigen levels. Br J Haematol 1995; 91: 474-6.
29. Vlot AJ, Koppelman SJ, van den Berg MH, Bouma BN, Sixma JJ. The affinity and stoichiometry of binding of human factor VIII to von Willebrand factor. Blood 1995; 85: 3150-7.
30. Nogami K, Shima M, Nishiya K et al. A novel mechanism of factor VIII protection by von Willebrand factor from activated protein C-catalyzed inactivation. Blood 2002; 99: 3993-8.
31. Deitcher SR, Tuller J, Johnson JA. Intranasal DDAVP induced increases in plasma von Willebrand factor alter the pharmacokinetics of high-purity factor VIII concentrates in severe haemophilia A patients. Haemophilia 1999; 5: 88-95.
32. Tang L, Leong L, Sim D et al. von Willebrand factor contributes to longer half-life of PEGylated factor VIII in vivo. Haemophilia 2013; 19: 539-45.
33. Carcao M. Changing paradigm of prophylaxis with longer acting factor concentrates. Haemophilia 2014; 20(Suppl 4): 99-105.
34. Shapiro AD. Long-lasting recombinant factor VIII proteins for hemophilia A. Hematology Am SocHematolEduc Program. 2013; 2013: 37-43.
35. Takeyama M, Wintermute JM, Manithody C, Rezaie AR, Fay PJ. Variable contributions of basic residues forming an APC exosite in the binding and inactivation of factor VIIIa. Biochemistry 2013; 52: 2228-35.
36. Cramer TJ, Gale AJ. Function of the activated protein C (APC) autolysis loop in activated FVIII inactivation. Br J Haematol 2011; 153: 644-54.