title: Emerging drugs for the treatment of hemophilia A and B.
abstract: BACKGROUND
  Replacement therapy with clotting factor concentrates is the most appropriate and effective way to treat bleedings of Hemophilia A&B to prevent chronic arthropathy. Unfortunately, the short half-life (HL) of FVIII/IX concentrates obliges the patients to receive frequent infusions, a big concern for children. The development of inhibitors in about 30-45% of hemophilia A and in 3-5% of hemophilia B patient is the major adverse event of replacement therapy.
  
  
  METHODS
  In the last few years, new rFIX have been developed with HL. New rFVIII concentrates are displaying small increase of PK characteristics. The new bio-engineering methods allowed the production of molecules fused with Fc fragment of IgG or Albumin or linked to PEG. A new approach to improve hemostasis is represented by Mab against TFPI and small RNA interfering with Antithrombin synthesis. Another innovative drug seems to be the new bi-specific antibody which mimics FVIII function in linking FXa and FX to tenase production.
  
  
  CONCLUSIONS
  The emerging drugs for hemophilia treatment seem to be very promising. The extended half-life will improve the adherence of patients to therapy. Accurate post-marketing surveillance studies will be necessary to check the efficacy, safety and immunogenicity of these new molecules.
authors.affiliation: a Past President of Italian Association of Haemophilia Centres , Firenze , Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Morfini
authors.affiliation: b Internal Medicine Department, University of Padua Medical School , Padua , Italy.
authors.forename: Ezio
authors.initials: E
authors.lastname: Zanon
mesh_terms: D000818:Animals; D002648:Child; D015195:Drug Design; D005164:Factor IX / Q000008:administration & dosage / Q000009:adverse effects / Q000493:pharmacokinetics; D005169:Factor VIII / Q000008:administration & dosage / Q000009:adverse effects / Q000493:pharmacokinetics; D006207:Half-Life; D006467:Hemophilia A / Q000188:drug therapy* / Q000503:physiopathology; D002836:Hemophilia B / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D010349:Patient Compliance
chemical_list: D005169:Factor VIII; D005164:Factor IX
keywords: ACE910; ANL-AT3; Concizumab; N8-G; N9-GP; pharmacokinetics; rFIXFc; rFVIIIFc; rIX-FP; rVIII-SingleChain