Evaluation of One-Stage Assays for the Monitoring of Recombinant Human Factor IX Padua Activity After Etranacogene Dezaparvovec Gene Therapy.

ABSTRACT:

Introduction

Accurate and reproducible measures of factor activity are required to guide clinical decision-making following gene therapy for haemophilia B (HB). Highly significant discrepancies have been observed in measurements of various factor IX (FIX) concentrates that carry molecular modifications to extend their half-life, arguing for the need for careful analysis of new HB treatment modalities with respect to FIX assay performance.

Aim

To further characterise variability in FIX activity measured using different one-stage assays (OSAs) and chromogenic assays (CAs) in patients with HB receiving gene therapy utilising the FIX Padua variant and to assess whether assay differences were due to the FIX-Padua variant.

Methods

FIX activity was assessed centrally (OSA an

SUBMITTER: Astermark J 

PROVIDER: S-EPMC12311899 | biostudies-literature | 2025 Jul

REPOSITORIES: biostudies-literature