Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: A first human dose trial in patients with hemophilia B

Blood

Volumn 118, Issue 10, 2011, Pages 2695-2701

  Negrier, Claude ^a   Knobe, Karin ^b   Tiede, Andreas ^c   Giangrande, Paul ^d   Møss, Judi ^e  

^a UNIVERSITÉ LYON 1   (France)

^b LUND UNIVERSITY   (Sweden)

^c HANNOVER MEDICAL SCHOOL   (Germany)

^d CHURCHILL HOSPITAL   (United Kingdom)

^e NOVO NORDISK A S   (Denmark)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; CLEMASTINE; CORTISONE; GLYCOPEGYLATED RECOMBINANT FACTOR 9; MACROGOL; RECOMBINANT BLOOD CLOTTING FACTOR 9; UNCLASSIFIED DRUG;

ADULT; ARTICLE; BLEEDING; CLINICAL ARTICLE; CONJUNCTIVAL HYPEREMIA; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DRUG DOSE INCREASE; DRUG HALF LIFE; DRUG HYPERSENSITIVITY; DRUG SAFETY; DRUG TOLERANCE; DRUG WITHDRAWAL; DYSPHAGIA; FACE EDEMA; FATIGUE; HEMOPHILIA B; HUMAN; MALE; MYALGIA; NAUSEA; PARESTHESIA; PRIORITY JOURNAL; RASH; SINGLE DRUG DOSE; SUBSTITUTION THERAPY; SWELLING; TREATMENT RESPONSE; UNSPECIFIED SIDE EFFECT; VERTIGO;

EID: 80052669248     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2011-02-335596     Document Type: Article

References (39)

1. Peyvandi F, Jayandharan G, Chandy M, et al. Genetic diagnosis of haemophilia and other inherited bleeding disorders. Haemophilia. 2006;12(suppl 3):82-89.
2. Carr ME Jr. Future directions in hemostasis: normalizing the lives of patients with hemophilia. Thromb Res. 2010;125(suppl 1):S78-S81.
3. Darby SC, Kan SW, Spooner RJ, et al. Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV. Blood. 2007;110(3):815-825. (Pubitemid 47267416)
4. Lyseng-Williamson KA, Plosker GL. Recombinant factor VIIa (eptacog alfa): a pharmacoeconomic review of its use in haemophilia in patients with inhibitors to clotting factors VIII or IX. Pharmacoeconomics. 2007;25(12):1007-1029. (Pubitemid 350208906)
5. Gringeri A, Von MS, Auerswald G, et al. Health status and health-related quality of life of children with haemophilia from six West European countries. Haemophilia. 2004;10(suppl 1):26-33. (Pubitemid 40045397)
6. Bjorkman 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(2):133-139. (Pubitemid 32243794)
7. 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(3):233-243. (Pubitemid 46746045)
8. Löfqvist T, Nilsson IM, Berntorp E, Pettersson H. Haemophilia prophylaxis in young patients - a long-term follow-up. J Intern Med. 1997;241(5):395-400. (Pubitemid 27244664)
9. Poon MC, 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(3):431-435. (Pubitemid 34232484)
10. Shapiro AD, Korth-Bradley J, Poon MC. Use of pharmacokinetics in the coagulation factor treatment of patients with haemophilia. Haemophilia. 2005;11(6):571-582. (Pubitemid 41631591)
11. Nilsson IM, Berntorp E, Löfqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232(1):25-32.
12. Blanchette VS. Prophylaxis in the haemophilia population. Haemophilia. 2010;16(suppl 5):181-188.
13. Journeycake JM, Quinn CT, Miller KL, Zajac JL, Buchanan GR. Catheter-related deep venous thrombosis in children with hemophilia. Blood. 2001;98(6):1727-1731.
14. Neunert CE, Miller KL, Journeycake JM, Buchanan GR. Implantable central venous access device procedures in haemophilia patients without an inhibitor: systematic review of the literature and institutional experience. Haemophilia. 2008;14(2):260-270. (Pubitemid 351343620)
15. Geraghty S, Dunkley T, Harrington C, et al. Practice patterns in haemophilia A therapy - global progress towards optimal care. Haemophilia. 2006;12(1):75-81.
16. Collins P, Faradji A, Morfini M, Enriquez MM, Schwartz L. Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study. J Thromb Haemost. 2010;8(1):83-89.
17. EMEA. 2009. Committee for medicinal products for human use (CHMP), Guideline on the Clinical Investigation of Recombinant and Human Plasma-derived Factor IX Products, EMEA/CHMP/BPWP/144552/2009. Available at http://www.emea.europa.eu/pdfs/human/bpwg/14455209endraft.pdf. Accessed December 2010.
18. World Medical Association. 2008. World Medical Association Declaration of Helsinki: Ethical principles for medical research involving human subjects. Last amended by the 59th WMA General Assembly, Seoul 2008. Available at http://www.wma.net/en/30publications/10policies/b3/index.html. Accessed December 2010.
19. International Conference on Harmonisation. ICH Harmonised Tripartite Guideline: Guideline for Good Clinical Practice E6 (R1), Step 4, 10-6-1996. Available at http://www.ich.org/LOB/media/MEDIA482.pdf. Accessed December 2010.
20. Bjorkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia. 2003;9(suppl 1):101-110. (Pubitemid 36622057)
21. Bolton-Maggs PH, Perry DJ, Chalmers EA, et al. The rare coagulation disorders-review with guidelines for management from the United Kingdom Haemophilia. Centre Doctors' Organisation. Haemophilia. 2004;10(5):593-628. (Pubitemid 39317772)
22. Giles AR, Verbruggen B, Rivard GE, Teitel J, Walker I. A detailed comparison of the performance of the standard versus the Nijmegen modification of the Bethesda assay in detecting factor VIII:C inhibitors in the haemophilia A population of Canada. Association of Hemophilia Centre Directors of Canada. Factor VIII/IX Subcommittee of Scientific and Standardization Committee of International Society on Thrombosis and Haemostasis. Thromb Haemost. 1998;79(4):872-875. (Pubitemid 28163870)
23. Verbruggen B, Novakova I, Wessels H, et al. The Nijmegen modification of the Bethesda assay for factor VIII:C inhibitors: improved specificity and reliability. Thromb Haemost. 1995;73(2):247-251.
24. Warrier I, Ewenstein BM, Koerper MA, et al. Factor IX inhibitors and anaphylaxis in hemophilia B. J Pediatr Hematol Oncol. 1997;19(1):23-27. (Pubitemid 27124054)
25. Shapiro AD, Di PJ, Cohen A, et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood. 2005;105(2):518-525. (Pubitemid 40070730)
26. Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood. 2001;98(13):3600-3606.
27. Gaberc-Porekar V, Zore I, Podobnik B, Menart V. Obstacles and pitfalls in the PEGylation of therapeutic proteins. Curr Opin Drug Discov Devel. 2008;11(2):242-250. (Pubitemid 351281064)
28. Jevsevar S, Kunstelj M, Porekar VG. PEGylation of therapeutic proteins. Biotechnol J. 2010;5(1):113-128.
29. Fishburn CS. The pharmacology of PEGylation: balancing PD with PK to generate novel therapeutics. J Pharm Sci. 2008;97(10):4167-4183.
30. Lichtenstein G, Thomsen O, Schreiber S, et al. Long-term remission with certolizumab pegol in Crohn's disease over 3.5 years: results from the PRECiSE 3 study. Inflamm Bowel Dis. 2009;15:S12-S13.
31. Sandborn W, Lichtenstein G, Schreiber S, Feagan B. The long-term, 30 months, efficacy and tolerability of certolizumab pegol therapy for Crohn's disease. Am J Gastroenterol. 2008;103:1099.
32. Lillicrap D. Extending half-life in coagulation factors: where do we stand? Thromb Res. 2008;122(suppl 4):S2-S8.
33. Fischer K, van der B, Molho P, et al. Prophylactic versus on-demand treatment strategies for severe haemophilia: a comparison of costs and long-term outcome. Haemophilia. 2002;8(6):745-752.
34. Ljung R. Prophylactic therapy in haemophilia. Blood Rev. 2009;23(6):267-274.
35. Monahan PE, Di PJ. Recombinant factor IX for clinical and research use. Semin Thromb Hemost. 2010;36(5):498-509.
36. 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(2):190-197.
37. Kisker CT, Eisberg A, Schwartz B. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia. 2003;9(3):279-284. (Pubitemid 36622029)
38. White GC, Beebe A, Nielsen B. Recombinant factor IX. Thromb Haemost. 1997;78(1):261-265. (Pubitemid 27289251)
39. Bjorkman S. A commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosing. Haemophilia. 2011;17(2):179-184.