Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX-FP Prophylaxis in Hemophilia B: A Repeated Time-to-Event Analysis.

ABSTRACT: In hemophilia B, pharmacokinetic (PK)-guided dosing of extended half-life factor IX (EHL-FIX) concentrates can secure targeted FIX exposure. Target FIX activity levels in plasma should be individually set primarily taking bleeding tendency into account, alongside the presence of target joints, physical activity, and preferred dosing schedules. In other words, both PK and pharmacodynamics (PD) are relevant when individualizing therapy. Our objective was to examine the relationship between dose, FIX activity levels, and bleeding specifically for EHL-FIX concentrate recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP). Data from hemophilia B patients with endogenous FIX activity level ≤ 2 IU/dL from five clinical trials were combined. Bleeding probability was describ

SUBMITTER: Koopman SF 

PROVIDER: S-EPMC12439015 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature