Spotlight on the human factor: building a foundation for the future of haemophilia A management: report from a symposium on human recombinant FVIII at the World Federation of Hemophilia World Congress, Melbourne, Australia on 12 May 2014.

摘要: Summary Inhibitor development is the most serious and challenging complication in treatment of severe haemophilia A. Up to 38% such patients develop inhibitors with current recombinant factor VIII (rFVIII) products produced hamster cell lines. Human-cl rhFVIII a new generation fully sulfated B-domain-deleted FVIII coagulant glycoprotein, which generated from human line. Thus, there are no non-human epitopes would be potentially immunogenic. This molecule has significantly higher VWF-binding affinity compared existing full-length rFVIII The aim address challenges frequent infusions during prophylaxis. rhFVIII's mean half-life very comparable some newer involve modification to extend circulating half-life. There promising data concerning use personalized prophylaxis regimen rhFVIII. Preliminary indicate median dosing interval 3.5 days 66.7% on twice per week or fewer schedule combined low bleeding rate increased consumption when standard No product-specific laboratory assay required monitor coagulation activity for results registration clinical trials as well ongoing studies previously untreated (NuProtect) and personalized study treated (NuPreviq), will discussed. manufacturer received marketing authorization Europe Canada under name Nuwiq® plans launch it USA globally 2015.