Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.

ABSTRACT:

Background

More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.

Objectives

We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.

Methods

Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.

Design</

SUBMITTER: Chambost H 

PROVIDER: S-EPMC11770721 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature