Abstract Number: PB0538
Meeting: ISTH 2021 Congress
Background: rIX-FP, a long-acting fusion protein, previously demonstrated safety and efficacy in patients with hemophilia B in clinical trials. Data from routine clinical practice on the effectiveness and safety of rIX-FP are required.
Aims: To obtain data on clinical outcomes in patients treated with rIX-FP during routine clinical practice in Germany.
Methods: A non-interventional study was initiated in Germany in March 2018. All patients with hemophilia B are eligible for enrolment. Patients are followed for 2–3 years or until 100 exposure days and are treated with rIX-FP up to every 14 days and routinely monitored every 3–12 months.
Results: At the interim data cut-off, 26 October 2020, 63 patients including one female were enrolled across 22 sites. One patient has since withdrawn due to lack of treatment satisfaction and another requested to withdraw but has remained on rIX-FP prophylaxis. Enrolled patients range in age from 1–80 years and have been observed for mean (SD) 7.4 (6.82) months. Mean rIX-FP consumption is lower or within the EU recommended initial dosing range for rIX-FP (35–50 IU/kg/week) for all age groups (adults, 32.3 IU/kg/week; adolescents, 38 IU/kg/week; pediatrics, 43 IU/kg/week). The effectiveness of treatment was rated good or excellent in 87.3% of cases. Mean annualized bleeding rates (ABR) were 1.1 (n=18), 1.1 (n=2), and 2.4 (n=10) for adult, adolescent, and pediatric patients, respectively. Median spontaneous ABR was 0.0 in adult and pediatric patients. Thirty-seven adverse events (AEs) have been reported, none of which were related to rIX-FP. Eight previously untreated patients have been treated; no inhibitors or drug-related AEs have been reported. To date, no patients have developed inhibitors.
Conclusions: Data from this study indicate that rIX-FP prophylaxis is well tolerated in routine clinical practice in Germany. Further studies analyzing patients treated with rIX-FP in other countries are ongoing.
To cite this abstract in AMA style:Oldenburg J, Holzhauer S, Wenning S, Olivieri M, Pfrepper C. Efficacy and Safety Analysis of the Use of rIX-FP in Patients with Hemophilia B: A Prospective, Non-interventional, Surveillance Study from Germany [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/efficacy-and-safety-analysis-of-the-use-of-rix-fp-in-patients-with-hemophilia-b-a-prospective-non-interventional-surveillance-study-from-germany/. Accessed January 23, 2022.
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