Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia
2011
The pharmacokinetic (PK) response to factor
VIII (FVIII) and factor IX varies between patients and
this has important clinical implications for treatment.
Although PK is affected by patient characteristics, this
relationship is too weak to infer a result for an
individual and, if required, PK must be measured. An
important determinant of the efficacy of prophylaxis is
the length of time an individual spends with a low level
of coagulation factor. This time is more dependent on
the patient's coagulation factor half-life and the frequency
of dosing than in vivo recovery and dose
infused. Improved understanding of the effect of PK
and dose frequency on factor levels in patients on
prophylaxis will help tailor regimens to individuals
better and allow more cost effective use of coagulation
factor concentrates. Calculations suggest that adults
need less FVIII per kg body weight than children. The
effect of half-life on trough levels questions the logic of
Monday, Wednesday, Friday dosing and suggests a role
for innovative regimens including low-dose daily treatment
which leads to either higher trough levels or
decreased FVIII requirement. This may expand access to
prophylaxis in healthcare systems with limited resources
and potentially improve patient outcomes. The ideal
trough level will vary between individuals and at
different times of their lives and may be <1 IU dL)1. If
PK is to be used in routine clinical practice, a simplified
method for its measurement is required and this
methodology is becoming available.
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