Haemophilia therapies.
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 might 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.
Dargaud
Yesim
Y
Hôpital Edouard Herriot, Comprehensive Haemophilia Treatment Centre, Lyon, France. ydargaud@univ-lyon1.fr
Negrier
Claude
C
D000818:Animals; D001779:Blood Coagulation Factors / Q000008:administration & dosage; D003029:Coagulants / Q000008:administration & dosage* / Q000009:adverse effects / Q000276:immunology; D004334:Drug Administration Schedule; D016903:Drug Monitoring; D005164:Factor IX / Q000008:administration & dosage; D005167:Factor VII / Q000008:administration & dosage; D005169:Factor VIII / Q000008:administration & dosage; D015942:Factor VIIa; D015316:Genetic Therapy / Q000379:methods; D006467:Hemophilia A / Q000188:drug therapy* / Q000235:genetics / Q000628:therapy; D006470:Hemorrhage / Q000139:chemically induced; D006801:Humans; D018579:Patient Selection; D015202:Protein Engineering / Q000379:methods; D011994:Recombinant Proteins / Q000008:administration & dosage; D016896:Treatment Outcome
D001779:Blood Coagulation Factors; D003029:Coagulants; D011994:Recombinant Proteins; C025667:prothrombin complex concentrates; C078147:F8 protein, human; D005167:Factor VII; D005169:Factor VIII; D005164:Factor IX; C103587:recombinant FVIIa; D015942:Factor VIIa