How we choose factor VIII to treat hemophilia.

@article{Mannucci2012HowWC,
  title={How we choose factor VIII to treat hemophilia.},
  author={Pier Mannuccio Mannucci and Maria Elisa Elisa Mancuso and Elena Santagostino},
  journal={Blood},
  year={2012},
  volume={119 18},
  pages={
          4108-14
        }
}
In high-income countries, the large availability of coagulation factors for replacement therapy of patients with hemophilia A has raised the life expectancy of these lifelong bleeders to that of males from the general population. The practicing clinician is offered a multitude of choices among several commercial brands of factor VIII extracted from human plasma or engineered from mammalian cell cultures by means of recombinant DNA technology. This article has the goal to offer our opinions on… 
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Interest is currently directed toward the potential for lesser immunogenicity of novel hemostatic agents designed to decrease the dosing frequency or avoid/delay the need of FVIII replacement therapy.
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Plasma-derived factor VIII is less immunogenic, but not entirely safe from the point of view of the possibility of transmitting biological agents, and plasma-derived or recombinant factor VIII products must co-exist on the market for the benefit of haemophilic patients.
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The discovery of "antihemophilic globulin" in the middle of the 20th century paved the way to the production of cryoprecipitate and then of FVIII and FIX concentrates, which revolutionized the treatment of hemophilia through the widespread adoption of home treatment and prophylaxis regimens, which dramatically improved the quality of life and life expectancy of persons with hemophilias during the past decade.
2017 Clinical trials update: Innovations in hemophilia therapy
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The nonfactor replacement strategies provide a promising treatment option for patients with inhibitors, presently the greatest unmet medical need in hemophilia.
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Evolution of the European guidelines for the clinical development of factor VIII products: little progress towards improved patient management
  • P. Mannucci
  • Medicine
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  • 2013
TLDR
This article reviews the evolution of the European regulations on new FVIII products, lists a number of regulatory requirements whose scientific and/or clinical rationale is perhaps questionable and recommends keeping such requirements in reasonable limits of feasibility, without jeopardizing current high standards of efficacy and safety.
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