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New products for the treatment of haemophilia
Author(s) -
Laffan Mike
Publication year - 2016
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.13797
Subject(s) - haemophilia , medicine , factor ix , clinical practice , clinical trial , haemophilia a , intensive care medicine , surgery , physical therapy
Summary 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.