Recombinant Factor IX for Clinical and Research Use

Department of Pediatrics, Gene Therapy Center, Harold R. Roberts Comprehensive Hemophilia Treatment Center, University or North Carolina at Chapel Hill, Chapel Hill, NC 27599-7236, USA.
Seminars in Thrombosis and Hemostasis (Impact Factor: 3.88). 07/2010; 36(5):498-509. DOI: 10.1055/s-0030-1255444
Source: PubMed


The last significant advance in the therapy of hemophilia B was the introduction of recombinant factor IX (FIX), ensuring an advanced level of safety from potential infectious contaminants of plasma-derived clotting factors. Since that time, recombinant DNA techniques have been applied in research to elucidate the role of FIX and its functional domains within coagulation. At the same time, recombinant DNA technology has been applied to engineer an expanding spectrum of novel FIX therapies that are now being translating into clinical trials. The experience with the existing recombinant FIX product is reviewed with a focus on the novel products and the potential to improve the quality of life for individuals with hemophilia B.

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    • "IX powodują zmiany w jego farmakokinetyce, czego skutkiem jest o około 30% mniejsze odzyskanie rcz. IX w porównaniu z osoczopochodnym [15]. Czas półtrwania obu rodzajów cz. "
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    ABSTRACT: The treatment of patients with haemophilia A and B is based on substitution of factor VIII and factor IX. The half-life of factor VIII (about 11 hours) and factor IX (about 18 hours) are rather short, so frequency of prophylactic infusions of factor VIII and factor IX concentrates are not less than 3 or 2 times a week. The development of new factor VIII and factor IX concentrates with longer half-lives makes possible the prolongation of time between doses and the improvement of prophylaxis efficacy. The fusion of factor IX to polyethylene glycol, Fc fragment of immunoglobulin G or albumin prolonged half-life of this coagulation protein to almost 100 hours. The half-life of factor VIII concentrate, with recombinant factor VIII combined with polyethylene glycol or Fc fragment of immunoglobulin G is prolonged to about 19 hours. Modification of factor VIII structure to produce single-chain protein with increased binding to von Willebrand factor is a new promising project as well. The introduction of coagulation concentrates with prolonged half-life will improve the quality of life of patients with severe type of haemophilia.
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  • No preview · Article · Jul 2010 · Seminars in Thrombosis and Hemostasis
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