Primary prophylaxis for children with severe congenital factor VII deficiency - Clinical and laboratory assessment

Among our treated patients neither FVII activity nor thrombin generation parameters (both already declined 24 h post rFVIIa administration) were able to predict the impact of prophylaxis, and could not be used as surrogate markers in order to assess the most beneficial treatment frequency. However, the long clinical follow-up and comprehensive laboratory assessment performed, have shown that early primary prophylaxis as administered in our cohort was safe and effective.