Abstract Number: PB1153
Meeting: ISTH 2022 Congress
Background: The extended half life rFIXFc has a well-established safety and efficacy profile demonstrated by phase 3 trials in patients with haemophilia B (HB; ≤2 IU/dL FIX) across all age-groups. The B-MORE study is prospectively evaluating real-world effectiveness and usage of rFIXFc in HB patients in Europe and Middle East.
Aims: To describe interim data for patients treated with rFIXFc in the ongoing B-MORE study.
Methods: This 24-month prospective, multicentre, non-interventional study includes patients prescribed with prophylactic or on-demand rFIXFc treatment prior to or at study enrolment. Baseline characteristics, 12-month retrospective data on previous FIX treatment prior to rFIXFc switch, rFIXFc prescribed dose and annualised bleeding rate (ABR) are reported.
Results: At interim data cut-off (22nd October 2021), 117 patients (2 female) from 25 centres were included in the effectiveness analysis. Baseline patient characteristics are shown in Table 1. Sixty patients were aged < 18 years (median 8.3, range 1-18 years). Patients with ≥6 months documented prophylaxis with standard half-life FIX prior to rFIXFc had a median (IQR) of 2.0 injections with FIX per week (2.0-2.0) (n=69), with a median (IQR) factor consumption of 74.9 (58.4-92.1) IU/kg/week (n=65) and a median (IQR) overall ABR of 1.0 (0.0-2.0) (n=69). Mean (range) overall duration of rFIXFc treatment from initiation to data cut-off was 824 (42-3650) and 457 (15-1207) days for prophylactically (n=106) and on-demand treated subjects (n=11), respectively. Dosing and ABR on rFIXFc prophylaxis are shown in Table 2.
Conclusion(s): Interim data from the ongoing B-MORE study indicate that rFIXFc prophylaxis can provide and maintain excellent bleed protection in this HB population including a large number of paediatric patients. These real-world data show a median ABR close to zero on rFIXFc prophylaxis with a low factor consumption mainly based on a once-weekly regimen.
To cite this abstract in AMA style:Glosli H, Ranta S, Allsup D, Ricca I, Saleh M, Carlsheimer Å, Falk A, Santagostino E. Interim Analysis from B-MORE, a 24-month Prospective, Multicentre, Non-interventional Study on Effectiveness and Usage of Recombinant Factor IX Fc (rFIXFc) in Haemophilia B [abstract]. https://abstracts.isth.org/abstract/interim-analysis-from-b-more-a-24-month-prospective-multicentre-non-interventional-study-on-effectiveness-and-usage-of-recombinant-factor-ix-fc-rfixfc-in-haemophilia-b/. Accessed September 29, 2023.
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