title: New and Emerging Agents for the Treatment of Hemophilia: Focus on Extended Half-Life Recombinant Clotting Proteins.
abstract: Hemophilia A and B are X-linked disorders caused by deficient or defective clotting factor VIII (FVIII) or IX factor (FIX) proteins, and characterized by spontaneous or traumatic bleeding into joints and muscles. Previous use of plasma and plasma-derived clotting factors that lacked appropriate viral inactivation steps in manufacturing led to significant morbidity associated with transfusion-transmitted HIV and hepatitis C virus (HCV). The development of recombinant proteins revolutionized their treatment, and, with no new HIV or HCV infection via clotting proteins for nearly 30 years, greatly improved their lifespan, which now approaches that of the general population, and with the same risks for aging complications. Novel long-acting factor proteins are being licensed to extend FVIII and FIX half-life, thereby reducing infusion frequency and potentially bleed frequency and associated morbidity. Further, novel therapeutics which take advantage of new technologies, including siRNA, monoclonal antibody, and small peptide inhibition technologies, have the potential to simplify treatment and improve outcomes for those with inhibitors.
authors: Division Hematology/Oncology, Department of Medicine, Hemophilia Center of Western Pennsylvania, University of Pittsburgh, 3636 Boulevard of the Allies, Pittsburgh, PA, 15213-4306, USA. ragni@pitt.edu.
authors: Margaret V
authors: MV
authors: Ragni
mesh_terms: D005164:Factor IX / Q000008:administration & dosage / Q000378:metabolism* / Q000627:therapeutic use*; D005169:Factor VIII / Q000008:administration & dosage / Q000378:metabolism* / Q000627:therapeutic use*; D006207:Half-Life; D006467:Hemophilia A / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D011994:Recombinant Proteins / Q000378:metabolism*
chemical_list: D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX