PB0686 - 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

Monday, July 11, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

BP

Brigitte Pan-Petesch

MD
CRC-MHC Brest – Service Hématologie, Unité Hémostase Clinique
BREST, Bretagne, France
MF

Marc Fouassier

Doctor
Haemostasis Consultations, CRC-MHC, CHU Hôtel-Dieu, Nantes, France
Nantes, Pays de la Loire, France
BG

Benoît Guillet

Doctor
CRC-MHC, CHU de Rennes – Hôpital Pontchaillou, Rennes, France
Rennes, Bretagne, France
AH

Annie Harroche

Pediatric Hematologist
CRC-MHC Hôpital Necker-Enfants malades, Paris, France
Paris, Ile-de-France, France
RD

Roseline D'Oiron

Hematologist
Haemophilia Treatment Centre, Bicêtre Hospital, University Hospital of Paris (APHP), Kremlin-Bicêtre, France.
paris, Centre, France
YD

Yesim Dargaud

Professor
CRC-MHC, Hôpital Edouard Herriot Pavillon, Lyon, France
Lyon, Rhone-Alpes, France

Presenting Author(s)

Sophie Susen, MD, PhD

Professor, MD, PhD
CRC-MHC, Lille University Hospital, Lille, France
Lille, Nord-Pas-de-Calais, France

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 (N=100) 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.