Abstract Number: PB0686
Meeting: ISTH 2022 Congress
Background: rIX-FP is a long-acting fusion protein genetically linking recombinant human coagulation factor IX (FIX) with recombinant human albumin. In clinical trials, rIX-FP provided low annualized bleeding rates with 7-, 14- and 21-day prophylaxis and robust efficacy in bleed treatment and surgery. Long-term data is needed on the effectiveness and safety of rIX-FP in routine clinical practice.
Aims: This post-marketing surveillance study (OrPHEe; Clinicaltrials.gov identifier: NCT05086575) aims to collect real-world effectiveness and safety data on the use of rIX-FP in real-world clinical practice in France.
Methods: The study is being conducted across up to 25 specialist hemophilia centers in France. The study cohort includes patients with hemophilia B, regardless of severity, who are currently or previously receiving rIX-FP for long-term prophylaxis, for short-term prophylaxis to cover periods with a high risk of bleeding, for on-demand treatment, or for treatment during surgery. Patients (Nf100) satisfying the inclusion criteria will be enrolled for a period of two years and each patient will be followed up for three years. Data on safety, efficacy and health-related quality of life will be gathered, in addition to disease and treatment (in the year prior to rIX-FP initiation) history.
Results: Recruitment to the study began in December 2021. Study methodology will be described in detail, and the interim data analysis will be provided by the time of congress presentation.
Conclusion(s): This prospective, non-interventional, multicenter study will provide data on the real-world use of rIX-FP in France to confirm the favorable efficacy and safety of rIX-FP seen in clinical development studies.
To cite this abstract in AMA style:Pan-Petesch B, Fouassier M, Guillet B, Harroche A, d'Oiron R, Dargaud Y, Susen S. Real-world use of recombinant factor IX albumin fusion protein (rIX-FP) in patients with hemophilia B: A prospective, multicenter, non-interventional post-marketing surveillance study in France [abstract]. https://abstracts.isth.org/abstract/real-world-use-of-recombinant-factor-ix-albumin-fusion-protein-rix-fp-in-patients-with-hemophilia-b-a-prospective-multicenter-non-interventional-post-marketing-surveillance-study-in-france/. Accessed March 4, 2024.
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