Potential role of a new PEGylated recombinant factor VIII for hemophilia A

Journal of Blood Medicine

Volumn 7, Issue , 2016, Pages 121-128

  Wynn, Tung Thanh ^a   Gumuscu, Burak ^b,c  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^b St Mary s Hospital   (United States)

^c UNIVERSITY OF VIRGINIA   (United States)

Author keywords

Extended half life; Factor VIII deficiency; Hemophilia A; PEG

Indexed keywords

PEGYLATED RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 8; UNCLASSIFIED DRUG;

BLEEDING; BLOOD CLOTTING; CLINICAL TRIAL (TOPIC); DRUG APPROVAL; DRUG HALF LIFE; HEMOPHILIA A; HUMAN; PROTEIN FUNCTION; PROTEIN STRUCTURE; REVIEW;

EID: 85006207092     PISSN: None     EISSN: 11792736     Source Type: Journal    
DOI: 10.2147/JBM.S82457     Document Type: Review

References (39)

1. Hoyer LW. Hemophilia A. N Engl J Med. 1994;330:38–45.
2. Mannucci PM, Tuddenham EGD. The hemophilias from royal genes to gene therapy. N Engl J Med. 2001;344:1773–1779.
3. Antonarakis SE, Rossiter JP, Young M, et al. Factor VIII inversions in severe hemophilia A: results from an international consortium. Blood. 1995;86:2206–2212.
4. Paola JD, Montgomery RR, Gill JC, Flood V. Hemophilia and Von Willebrand disease. In: Orkin SH, Fisher D, Ginsburg D, Look AT, Lux SE, Nathan DG, editors. Nathan and Oski’s Hematology and Oncology of Infancy and Childhood. 8th ed. Philadelphia, PA: Elsevier; 2015:1028–1039.
5. Ragni MV. Targeting antithrombin to treat hemophilia. N Engl J Med. 2015;373:389–391.
6. Howard TE, Yanover C, Mahlangu J, et al. Haemophilia management: time to get personal? Haemophilia. 2011;17:721–728.
7. Toole JJ, Knopf JL, Wozney JM, et al. Molecular cloning of a cDNA encoding human antihaemophilic factor. Nature. 1984;312:342–347.
8. Medical and Scientific Advisory Council (MASAC) Recommendations Concerning Prophylaxis. Medical bulletin: 193. New York: National Hemophilia Foundation; 1994.
9. 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(6):535–544.
10. Oladapo AO, Epstein JD, Williams E, Ito D, Gringeri A, Valentino LA. Health-related quality of life assessment in haemophilia patients on prophylaxis therapy: a systematic review of results from prospective clinical trials. Haemophilia. 2015;21(5):e344–358.
11. Gouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013;121(20):4046–4055.
12. Fischer K, Lassila R, Peyvandi F; EUHASS participants. Inhibitor development in haemophilia according to concentrate. Four-year results from the European Haemophilia Safety Surveillance (EUHASS) project. Thromb Haemost. 2015; 113(5):911–1157.
13. Hay CR, DiMichele DM. The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012; 119(6):1335–1344.
14. Rocino A, Cortesi PA, Scalone L, Mantovani LG, Crea R, Gringeri A. Immune tolerance induction in patients with haemophilia a and inhibitors: effectiveness and cost analysis in an European Cohort (The ITERStudy). Haemophilia. 2016; 22(1):96–102.
15. Fay PJ, Chavin SI, Schroeder et al. Purification and characterization of a highly purified human factor VIII consisting of a single type of polypeptide chain. Proc Natl Acad Sci U S A. 1982;79:7200–7204.
16. Gitschier J, Wood WI, Goralka G, et al. Characterization of the human factor VIII gene. Nature. 1984;312:326–330.
17. Wood WI, Capon DJ, Simonsen CC, et al. Expression of active human factor VIII from recombinant DNA clones. Nature. 1984;312:330–337.
18. Tantravahi U, Murty VV, Jhanwar SC, et al. Physical mapping of the factor VIII gene proximal to two polymorphic DNA probes in human chromosome band Xq28: implications for factor VIII gene segregation analysis. Cytogenet Cell Genet. 1986;42:75–79.
19. Lenting PJ, Van Mourik J, Mertens K. The life cycle of coagulation factor VIII in view of its structure and function. Blood. 1998;92:3983–3996.
20. Kumaran V, Benten D, Follenzi A, Joseph B, Sarkar R, Gupta S. Transplantation of endothelial cells corrects the phenotype in hemophilia A mice. J Thromb Haemost. 2005;3(9):2022–2031.
21. Follenzi A, Benten D, Novikoff P, Faulkner L, Raut S, Gupta S. Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice. J Clin Invest. 2008;118(3):935–945.
22. Kemball-Cook G, Gomez K. Molecular basis of hemophilia A. In: Lee CA, Berntorp EE, Keith Hoots W, editors. Textbook of Hemophilia. Hoboken, NJ: Wiley Blackwell; 2014:23–31.
23. Gouw SC, Van der Bom JG, Ljung R. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013;368:231–239.
24. Astermark J. FVIII inhibitors: pathogenesis and avoidance. Blood. 2015;125(13):2045–2051.
25. Mahdi AJ, Obaji SG, Collins PW. Role of enhanced half-life factor VIII AND ix in the treatment of haemophilia. Br J Haematol. 2015;169:768–776.
26. Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe haemophilia A. Blood. 2014;123(3):317–325.
27. Zollner S, Raquet E, Claar P, et al. Non-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant singlechain factor VIII. Thromb Res. 2014;134(1):125–131.
28. Konkle BA, Stasyshyn O, Chowdary P, et al. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015;126(9):1078–1085.
29. Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013;11(4):670–678.
30. Coyle TE, Reding MT, Lin JC, Michaels LA, Shah A, Powell J. Phase I study of BAY 94-9027, a pegylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. J Thromb Haemost. 2014;12:488–496.
31. Krishnan S, Vietri J, Furlan R, Duncan N. Adherence to prophylaxis is associated with better outcomes in moderate and severe haemophilia: results of a patient survey. Haemophilia. 2015;21(1):64–70.
32. Shapiro AD, Donfield SM, Lynn HS, et al. Defining the impact of hemophilia: the Academic Achievement in Children with Hemophilia Study. Pediatrics. 2001;108(6):E105.
33. Duncan N, Shapiro A, Ye X, Epstein J, Luo MP. Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States. Haemophilia. 2012;18(5): 760–765.
34. Mannucci PM, Gringeri A, Peyvandi F, Santagostino E. Factor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers). Haemophilia. 2007;13(Suppl 5): S65–S68.
35. Peyvandi F, Mannucci PM, Garagiola I. Source of factor VIII replacement (plasmatic or recombinant) and incidence of inhibitory alloantibodies in previously untreated patients with severe hemophilia a: The Multicenter Randomized Sippet Study. Presented at: ASH 57th Annual Meeting Plenary Session; December 6, 2015; Orlando, FL.
36. Oldenburg J, Jiménez-Yuste V, Peiró-Jordán R, Aledort LM, Santagostino E. Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate. Haemophilia. 2014;20(1): 83–91.
37. Krishnamoorthy S, Liu T, Drager D, et al. Recombinant factor VIII Fc (rFVIIIFc) fusion protein reduces immunogenicity and induces tolerance in hemophilia A mice. Cell Immunol. 2016;301:30–39.
38. Groomes CL, Gianferante DM, Crouch GD, Parekh DS, Scott DW, Liewu K. Reduction of factor VIII inhibitor titers during immune tolerance induction with recombinant factor VIII-Fc fusion protein. Pediatr Blood Cancer. 2016;63:922–924.
39. Blanchette VS, Shapiro AD, Liesner RJ, et al. Plasma and albuminfree recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patients. J Thromb Haemost. 2008; 6(8):1319–1326.