PB1153 - 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

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Co-Author(s)

HG

Heidi Glosli

Medical Doctor
Division of Paediatric and Adolescent Medicine Centre for Rare Disorders Oslo University Hospital Oslo Norway.
Oslo, Oslo, Norway

Presenting Author(s)

Susanna Ranta, MD, PhD

Physician
Karolinska University Hospital
Stockholm, Stockholms Lan, Sweden

Co-Author(s)

DA

David Allsup

Senior Lecturer in Haematology
Hull University Teaching Hospital Trust And Centre for Atherothrombosis and Metabolic Disease
Hull, England, United Kingdom
IR

Irene Ricca

SSD Medicina Trasfusionale Materno-Infantile e Traumatologica
AOU Città della Salute e della Scienza di Torino, Presidio OIRM-S. Anna
Torino, Piemonte, Italy
MS

Mahasen Saleh, MD

Physician
King Faisal Specialist Hospital and Research Centre
Riyadh, Ar Riyad, Saudi Arabia
�C

Åsa Carlsheimer

Statistical Scientist
Swedish Orphan Biovitrum AB
Stockholm, Stockholms Lan, Sweden
AF

Aletta Falk

Director Medical Affairs Haemophilia
Sobi, Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden
ES

Elena Santagostino

VP, Medicine Development Lead Haemophilia
Sobi, Stockholm, Sweden
Stockholm, Stockholms Lan, Sweden

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.