The extravascular distribution of endogenous factor IX may be a relevant consideration for infused factor IX therapies
Extravascular distribution may have implications on how we evaluate PK activity in hemophilia B, including trough levels1,2
- Multiple PK parameters should be considered, including half-life, clearance, and volume of distribution (VOD)3
Trough levels do not account for infused factor IX that has moved outside of the plasma4,5
Data in hemophilia B patients show that plasma factor IX levels decrease quickly following infusion6
Additional research is needed to understand the clinical implications of VOD
Though PK is important, factor IX replacement therapy should ultimately be evaluated based on outcomes, including bleed prevention and control. PK parameters, such as trough levels, are not patient outcomes3,7
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Preclinical data are not correlated with outcomes. Animal studies may
not apply to humans. Additional research for preclinical data and research in humans is
needed to confirm these findings
PK=pharmacokinetic.