The improved factor concentrate.
The current treatment of haemophilia with coagulation protein replacement therapy is both effective and safe. Nevertheless, this therapy requires frequent, repeated intravenous infusions and approximately 25% of treated haemophilia A patients develop antibodies to the replacement protein. Furthermore, the cost and limited availability of current concentrates has restricted access to therapy to less than 30% of the global haemophilia population. With this background, efforts are now underway to develop coagulation concentrates with enhanced biological properties that further improve the quality of care for haemophiliacs. The specific areas of enhancement that are being explored include improved biosynthetic processes, prolonging the circulating half-life and reducing concentrate immunogenicity. Coincident with these approaches, it is hoped that there will be more widespread availability of these concentrates and that their cost will be contained.
Lillicrap
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Department of Pathology and Molecular Medicine, Richardson Laboratory, Queen's University, Kingston, Canada K7L 3N6. lillicrap@cliff.path.queensu.ca
D001779:Blood Coagulation Factors / Q000235:genetics / Q000592:standards / Q000627:therapeutic use*; D005169:Factor VIII / Q000235:genetics / Q000627:therapeutic use; D006467:Hemophilia A / Q000188:drug therapy; D006801:Humans; D011993:Recombinant Fusion Proteins / Q000276:immunology / Q000627:therapeutic use; D011994:Recombinant Proteins / Q000627:therapeutic use; D012449:Safety
D001779:Blood Coagulation Factors; D011993:Recombinant Fusion Proteins; D011994:Recombinant Proteins; D005169:Factor VIII