Many factor VIII products available in the treatment of hemophilia a: An embarrassment of riches?

Journal of Blood Medicine

Volumn 8, Issue , 2017, Pages 67-73

  Lieuw, Kenneth ^a,b  

^a WALTER REED ARMY MEDICAL CENTER   (United States)

^b UNIFORMED SERVICES UNIVERSITY OF THE HEALTH SCIENCES   (United States)

Author keywords

Bleeding disorder; Extended half life factor VIII; Factor VIII; Hemophilia; Hemophilia A; Immunogenicity; Inhibitor development; Recombinant factor VIII single chain

Indexed keywords

BLOOD CLOTTING FACTOR 8;

REVIEW;

EID: 85020911947     PISSN: None     EISSN: 11792736     Source Type: Journal    
DOI: 10.2147/JBM.S103796     Document Type: Review

References (47)

1. Hoyer LW. Hemophilia A. N Engl J Med. 1994;330(1):38-47.
2. Coppola A, Di Capua M, Di Minno MN, et al. Treatment of hemophilia: a review of current advances and ongoing issues. J Blood Med. 2010;1:183-195.
3. Orlova NA, Kovnir SV, Vorobiev II, Gabibov AG, Vorobiev AI. Blood clotting factor VIII: from evolution to therapy. Acta Naturae. 2013;5(2):19-39.
4. Pipe SW. New therapies for hemophilia. Hematology Am Soc Hematol Educ Program. 2016;2016(1):650-656.
5. Pipe SW, Montgomery RR, Pratt KP, Lenting PJ, Lillicrap D. Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A. Blood. 2016;128(16):2007-2016.
6. Shapiro AD. Long-lasting recombinant factor VIII proteins for hemophilia A. Hematology Am Soc Hematol Educ Program. 2013;2013:37-43.
7. Bensen-Kennedy D. Bringing new therapy options to the hemophilia community. Thromb Res. 2013;131(suppl 2):S15-S18.
8. Mahlangu J, Kuliczkowski K, Karim FA, et al. Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A. Blood. 2016;128(5):630-637.
9. Pabinger-Fasching I. The story of a unique molecule in hemophilia A: recombinant single-chain factor VIII. Thromb Res. 2016;141 (suppl 3):S2-S4.
10. 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.
11. Zollner SB, Raquet E, Müller-Cohrs J, et al. Preclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIII. Thromb Res. 2013;132(2):280-287.
12. Miao HZ, Sirachainan N, Palmer L, et al. Bioengineering of coagulation factor VIII for improved secretion. Blood. 2004;103(9):3412-3419.
13. Santagostino E. A new recombinant factor VIII: from genetics to clinical use. Drug Des Devel Ther. 2014;8:2507-2515.
14. Kavakli K, Yang R, Rusen L, et al. Prophylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II). J Thromb Haemost. 2015;13(3):360-369.
15. Sandberg H, Kannicht C, Stenlund P, et al. Functional characteristics of the novel, human-derived recombinant FVIII protein product, human-cl rhFVIII. Thromb Res. 2012;130(5):808-817.
16. Viuff D, Barrowcliffe T, Saugstrup T, Ezban M, Lillicrap D. International comparative field study of N8 evaluating factor VIII assay performance. Haemophilia. 2011;17(4):695-702.
17. Kannicht C, Ramström M, Kohla G, et al. Characterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIII. Thromb Res. 2012;131(1):78-88.
18. Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glyco-PEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013;11(4):670-678.
19. Kaufman RJ, Powell JS. Molecular approaches for improved clotting factors for hemophilia. Hematology Am Soc Hematol Educ Program. 2013;2013:30-36.
20. Chowdary P, Fosbury E, Riddell A, Mathias M. Therapeutic and routine prophylactic properties of rFactor VIII Fc (efraloctocog alfa, Eloctate(R)) in hemophilia A. J Blood Med. 2016;7:187-198.
21. Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317-325.
22. Klukowska A, Szczepański T, Vdovin V, Knaub S, Jansen M, Liesner R. Novel, human cell line-derived recombinant factor VIII (Human-cl rhFVIII, Nuwiq(®)) in children with severe haemophilia A: efficacy, safety and pharmacokinetics. Haemophilia. Epub 2015 Sep 14.
23. Schmidbauer S, Witzel R, Robbel L, et al. Physicochemical characterization of rVIII-SingleChain, a novel recombinant single-chain factor VIII. Thromb Res. 2015;136(2):388-395.
24. Schulte S. Pioneering designs for recombinant coagulation factors. Thromb Res. 2011;128(suppl 1):S9-S12.
25. Delignat S, Repessé Y, Navarrete AM, et al. Immunoprotective effect of von Willebrand factor towards therapeutic factor VIII in experimental haemophilia A. Haemophilia. 2011;18(2):248-254.
26. Sorvillo N, Hartholt RB, Bloem E, et al. von Willebrand factor binds to the surface of dendritic cells and modulates peptide presentation of factor VIII. Haematologica. 2016;101(3):309-318.
27. Brand B, Gruppo R, Wynn TT, et al. Efficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patients. Haemophilia. 2016;22(4):e251-e258.
28. Wynn TT, Gumuscu B. Potential role of a new PEGylated recombinant factor VIII for hemophilia A. J Blood Med. 2016;7:121-128.
29. Scharrer I, Bray GL, Neutzling O. Incidence of inhibitors in haemophilia A patients - a review of recent studies of recombinant and plasmaderived factor VIII concentrates. Haemophilia. 1999;5(3):145-154.
30. Viel KR, Ameri A, Abshire TC, et al. Inhibitors of factor VIII in black patients with hemophilia. N Engl J Med. 2009;360(16):1618-1627.
31. Gunasekera D, Ettinger RA, Nakaya Fletcher S, et al. Factor VIII gene variants and inhibitor risk in African American hemophilia A patients. Blood. 2015;126(7):895-904.
32. Miller CH, Benson J, Ellingsen D, et al. F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicity. Haemophilia. 2012;18(3):375-382.
33. Gouw SC, van der Bom JG, Ljung R, et al. Factor VIII products and inhibitor development in severe hemophilia A. N Engl J Med. 2013;368(3):231-239.
34. Calvez T, Chambost H, Claeyssens-Donadel S, et al. Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A. Blood. 2014;124(23):3398-3408.
35. Borel Y. Induction of immunologic tolerance by a hapten (DNP) bound to a non-immunogenic carrier. Nat New Biol. 1971;230(14):180.
36. Groomes CL, Gianferante DM, Crouch GD, Parekh DS, Scott DW, Lieuw K. Reduction of factor VIII inhibitor titers during immune tolerance induction with recombinant factor VIII-Fc fusion protein. Pediatr Blood Cancer. 2016;63(5):922-924.
37. Malec LM, Journeycake J, Ragni MV. Extended half-life factor VIII for immune tolerance induction in haemophilia. Haemophilia. 2016;22(6):e552-e554.
38. Iorio A, Puccetti P, Makris M. Clotting factor concentrate switching and inhibitor development in hemophilia A. Blood. 2012;120(4): 720-727.
39. Ing M, Gupta N, Teyssandier M, et al. Immunogenicity of long-lasting recombinant factor VIII products. Cell Immunol. 2016;301:40-48.
40. Hasan H, Shaikh OM, Rassekh SR, Howard AF, Goddard K. Comparison of hypersensitivity rates to intravenous and intramuscular PEG-asparaginase in children with acute lymphoblastic leukemia: a meta-analysis and systematic review. Pediatr Blood Cancer. 2016;64(1): 81-88.
41. 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. 2015;301:30-39.
42. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054-2064.
43. Ettingshausen CE, Kreuz W. Recombinant vs. plasma-derived products, especially those with intact VWF, regarding inhibitor development. Haemophilia. 2006;12(suppl 6):102-106.
44. MASAC. MASAC Recommendation on SIPPET (Survey of Inhibitors in Plasma-Product-Exposed Toddlers): REsults and Recommendations for Treatment Products for Previously Untreated Patients with Hemophilia A. MASAC; 2016.
45. Shima M, Hanabusa H, Taki M, et al. Factor VIII-mimetic function of humanized bispecific antibody in hemophilia A. N Engl J Med. 2016;374(21):2044-2053.
46. Drager D, Patarroyo-White S, Chao H, et al. Recombinant FVIIIFc-VWF-XTEN demonstrates significant bioavailability following subcutaneous administration in hemophilia A mice. Blood. 2015; 126:3492.
47. Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371(21):1994-2004.