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Peptides, Proteins & Antisense
Haemophilia therapies
Yesim Dargaud† & Claude Negrier
†Hôpital Edouard Herriot, Comprehensive Haemophilia Treatment Centre, Lyon, France
In the last few decades dramatic improvements in the management of
haemophilia patients have occurred. Haemophilia has moved from a fatal or
disabling disease to a hereditary disorder with available treatment and much
better clinical outcomes. The safety of antihaemophilic factor concentrates
has been dramatically improved and, in a multidisciplinary environment
including haematologists, orthopaedic surgeons, paediatrics, infectiologists,
specialised nurses and physiotherapists, complications related to haemophilia
are now limited, markedly improving the quality of life of haemophiliacs.
One can even think that the cure of haemophilia through gene therapy
migth occur in the next decades. Keeping this ultimate aim in mind, efforts at
present are mainly focused on bioengineered Factor VIII/Factor IX
concentrates with increased efficacy or longer half-life or decreased
immunogenicity. In addition, several preclinical and clinical studies are being
carried out for optimising and individually tailoring the therapeutic regimens
of antihaemophilic therapies using global haemostasis tests in combination
with the routine coagulation assays.
Keywords: bypassing agents, FIX, FVIII, gene therapy, global haemostasis tests, haemophilia
Expert Opin. Biol. Ther. (2007) 7(5):651-663
原文下载：血友病治疗进展
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