title: New products for the treatment of haemophilia.
abstract: Current developments in haemophilia therapy are directed at two therapeutic targets: reduction of injection frequency and reduction or bypassing of inhibitors. A variety of new molecules addressing these aims are now completing clinical trials and are ready to enter clinical use. First amongst these are modified Factor VIII (FVIII) and Factor IX (FIX) molecules with extended half-lives. FIX modifications have achieved 5-fold prolongation of half-life whilst effects on FVIII have been more modest, at less than two-fold. We now face the problem of integrating these into clinical practice. Other approaches have generated chemically modified FVIII molecules with altered activation profiles. An alternative way of correcting the haemophilia defect is to reduce the activity of natural anticoagulants in an attempt to restore the balance of haemostasis. These methods are also giving promising results but, as with all new approaches, it will be some while before they all find their place in practice.
authors.affiliation: Centre for Haematology, Imperial College London, London, UK.
authors.forename: Mike
authors.initials: M
authors.lastname: Laffan
mesh_terms: D001779:Blood Coagulation Factors / Q000493:pharmacokinetics / Q000627:therapeutic use*; D005164:Factor IX / Q000493:pharmacokinetics / Q000627:therapeutic use; D005169:Factor VIII / Q000493:pharmacokinetics / Q000627:therapeutic use; D006207:Half-Life; D006467:Hemophilia A / Q000097:blood / Q000188:drug therapy*; D006801:Humans; D008297:Male; D015202:Protein Engineering / Q000379:methods; D011994:Recombinant Proteins / Q000097:blood / Q000627:therapeutic use
chemical_list: D001779:Blood Coagulation Factors; D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX
keywords: bleeding disorders; coagulation; coagulation factors; haemophilia