Optimization of hemophilia B treatment via population PK modeling of rIX-FP, including a 3-week regimen.

ABSTRACT:

Background

Recombinant factor IX-albumin fusion protein (rIX-FP) enables extended-interval prophylaxis for hemophilia B. While higher trough levels with reduced dosing frequency have been shown vs. standard FIX products, age-related differences in pharmacokinetics (PK) may constrain extended intervals in children. We aimed to refine a population PK (popPK) model of rIX-FP to inform clinically practical dosing across pediatric, adolescent, and adult patients.

Methods

Pooled FIX activity data from 113 previously treated patients (1-63 years) in five clinical studies were analyzed with a two-compartment popPK model. Covariates included body weight, age, weight-adjusted dose, and ethnicity (Japanese vs. non-Japanese). Model performance was evaluated by standard diagnostics and p

SUBMITTER: Terasaka N 

PROVIDER: S-EPMC12745456 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature