Summary
Current therapy for haemophilia A is guided by severity of the disease, which in turn
is best reflected in patients' endogenous factor VIII activity levels. For patients
with severe haemophilia (particularly children), prophylaxis with continuous routine
factor replacement has become standard of care in developed countries and is gradually
becoming the standard of care in developing countries. The question arises then: what
is an appropriate prophylaxis regimen to prevent bleeding events and arthropathy,
while also maximizing patient quality of life and taking into consideration the costs
of prophylaxis? Should all patients be treated with one standard, fixed prophylaxis
regimen, or should prophylaxis be individualised for each patient? If so, what factors
need to be considered in choosing the appropriate dose and frequency of factor administration?
If prophylaxis is tailored to the individual patient, then patient-related factors
(bleeding phenotype, activity profiles, age, joint status) and product-specific factors
(half-life of the replacement factor in the individual patient) will determine the
choice of regimen, whether it be a fixed-regimen prophylaxis or prophylaxis that is
tailored to patient activity and bleeding risk. Regardless of the choice of prophylaxis
regimen, for any regimen to be effective, adherence to therapy is key to optimising
outcomes.
Keywords
FVIII - prophylaxis - phenotype - dose - frequency
