Safety, Efficacy and Treatment Patterns of rIX-FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan.

ABSTRACT:

Background

Limited real-world data exist on recombinant fusion protein-linking coagulation factor IX (FIX) with albumin (rIX-FP) in paediatric previously untreated patients (PUPs) with haemophilia B, particularly in infants.

Aims

To evaluate the treatment patterns, safety and efficacy of rIX-FP in paediatric PUPs in Japan.

Methods

Retrospective chart review of 21 PUPs who initiated rIX-FP between May 2017 and September 2023, with follow-up to June 2024.

Results

At first administration, 95% (19/20) of patients were <2 years old, and 84% (16/19) weighed <10 kg. The mean treatment duration was 27 months. Prophylaxis was received by 90% (18/20), with 56% (10/18) on every 2 weeks and 44% (8/18) on a weekly regimen. The proportion of patients with ≥25 exposure days w

SUBMITTER: Nogami K 

PROVIDER: S-EPMC12904173 | biostudies-literature | 2026 Jan-Feb

REPOSITORIES: biostudies-literature