Recombinant VWF: is this the answer for treatment of von Willebrand disease?

摘要

The use of replacement protein factors for treatment of blood clotting disorders has a long history that has involved both major advances and setbacks. Replacement therapies can be viewed as the gold standard treatment for the majority of factor deficiencies and, for certain conditions, such treatments are both highly developed and well established. For the common inherited bleeding disorders, hemophilia A and B, replacement FVIII and FIX concentrate has been in clinical use for many years, with recombinant product prophylaxis now in routine use in those countries that can afford it, and further improvements relating to increasing the half-life of these products at a fairly advanced stage [1].