Abstract Number: PB1051
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: In clinical trials, rIX-FP, a long-acting fusion protein, led to low annualized bleeding rates with prophylaxis regimens up to every 21 days. Data on the effectiveness and safety of rIX-FP in routine clinical practice are required.
Aims: Prospective, non-interventional, multicenter studies are ongoing to gather data on effectiveness, safety and health-related quality of life outcomes in patients treated with rIX-FP during routine clinical practice in Europe.
Methods: Three non-interventional studies have been initiated in Italy, Belgium and Germany in September 2017, May 2017, and March 2018, respectively. All patients with hemophilia B are eligible for enrollment; however, the Italian study is not enrolling patients with mild hemophilia B. Patients are treated with rIX-FP up to every 14 days and routinely monitored every 3-12 months. Patients are followed for 2-3 years or until 100 exposure days. Patient data is collected, stored and anonymized in accordance with General Data Protection Regulations.
Results: In total, 114 patients have been enrolled in Italy (n=60), Belgium (n=16), and Germany (n=38) up to December 31, 2019. Patients range in age from 1-80 years, have between 0 and 646 exposure days to rIX-FP, and 64 patients (59%) have severe hemophilia B. At the data cut-off, 16 adverse events have been observed; one in the Italian cohort was considered related to rIX-FP and led to discontinuation of treatment. To date, no patients have developed inhibitors. Two patients in both Italy and Germany have discontinued rIX-FP treatment, one patient was lost to follow-up in Belgium. Efficacy and safety data collected will be reported after completion of the studies.
Conclusions: Initial data from these studies indicate that rIX-FP is well tolerated and leads to few adverse events in routine clinical use. Further data will be collected to assess the effectiveness and safety profile of rIX-FP in the real-world clinical setting.
To cite this abstract in AMA style:
Tagliaferri A, Oldenburg J, Peerlinck K, Castaman G. Real-world Evidence on the Use of rIX-FP in Patients with Hemophilia B: Interim Results from Three Multinational Prospective, Non-interventional, Post-market Surveillance Studies [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/real-world-evidence-on-the-use-of-rix-fp-in-patients-with-hemophilia-b-interim-results-from-three-multinational-prospective-non-interventional-post-market-surveillance-studies/. Accessed September 27, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/real-world-evidence-on-the-use-of-rix-fp-in-patients-with-hemophilia-b-interim-results-from-three-multinational-prospective-non-interventional-post-market-surveillance-studies/