Hamostaseologie 2025; 45(S 01): S55
DOI: 10.1055/s-0044-1801628
Abstracts
Topics
T-07 Hereditary bleeding disorders

Real-World Effectiveness and Usage of Recombinant Factor IX Fc: Interim Analysis in Paediatric Patients from the 24-Month, Prospective, Non-Interventional B-MORE Study

Authors

  • H Glosli

    1   Oslo University Hospital, Centre for Rare Disorders, Oslo, Norway

  • I Wieland

    2   Hannover Medical School, Department of Pediatric Hematology and Oncology, Hannover, Germany

  • H Pergantou

    3   Aghia Sophia Children’s Hospital, Paediatric Haemophilia Centre/Haemostasis and Thrombosis Unit, Athens, Greece

  • B Nolan

    4   Children's Health Ireland at Crumlin, Children’s Coagulation Centre, Dublin, Ireland

  • R Berrueco

    5   Sant Joan de Déu Hospital, Pediatric Hematology Department, Barcelona, Spain

  • S Ranta

    6   Karolinska University Hospital, Astrid Lindgren Children’s Hospital, Paediatric Coagulation Centre, Stockholm, Sweden

  • M Al Saleh

    7   King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

  • S Lauer

    8   Sobi, Stockholm, Sweden

  • E Bednar

    8   Sobi, Stockholm, Sweden

  • E Gresko

    9   Sobi, Basel, Switzerland

  • E Santagostino

    9   Sobi, Basel, Switzerland
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Introduction: B-MORE (NCT03901755) is an ongoing, 24-month prospective, non-interventional, international study evaluating the real-world effectiveness and usage of extended half-life recombinant factor IX Fc (rFIXFc; Alprolix®​) in patients with haemophilia B (PwHB).

Method: PwHB were prescribed rFIXFc on demand (OD) or prophylaxis (PPX) prior to/at B-MORE study entry. Interim data from the first 12 months of rFIXFc treatment (retrospective/prospective periods) were analysed for paediatric PwHB (age grouped by<12/12–<18 years) with≥6 months on the same rFIXFc regimen.

Results: At data cut-off (6 Oct 2022), a total of 151 PwHB were enrolled in B-MORE; 69 paediatric PwHB were included in this analysis (62 of 69 initiated rFIXFc before enrolment) with a median (range) age of 8.3 (1–<18) years (no inhibitor history). Of these paediatric PwHB, 54 were<12 years (all males; 42 severe, 11 moderate, 1 mild disease; 10 were previously untreated) and 15 were 12–<18 years (1 female; 12 severe, 3 moderate disease). At enrolment, 1 patient (12–<18 years) had joint surgery history and no patients had target joints. Overall, 67 PwHB received rFIXFc PPX (64 remained on PPX from rFIXFc initiation and 3 PwHB<12 years switched from rFIXFc OD at initiation) and 2 PwHB (n=1<12 years, mild disease; n=1 12–<18 years, severe) remained on rFIXFc OD from initiation. Median (range) duration on rFIXFc PPX was 33.5 (7–71) months for PwHB<12 years (n=53) and 30.2 (15–65) months for PwHB 12–<18 years (n=14). An intra-patient comparison of 31 PwHB (with≥6 months prior FIX PPX and≥9 months rFIXFc PPX) showed the mean (SD) total annualised bleeding rate reduced from 2.9 (4.8) pre-rFIXFc initiation to 1.0 (1.7) during the first 12 months in PwHB<12 years (n=22) and from 0.8 (1.0) to 0.4 (0.7) in PwHB 12–<18 years (n=9). Mean (SD) weekly factor consumption and injection frequency were reduced by 26 (29) IU/kg and 0.7 (0.4) injections in PwHB<12 years, and 25 (20) IU/kg and 1.2 (0.4) injections in PwHB 12–<18 years. No inhibitor development or serious adverse events related to rFIXFc were reported at data cut-off.

Conclusion: Interim real-world B-MORE data indicate rFIXFc is effective and well tolerated in paediatric PwHB, with improved bleed protection and reduced factor usage versus prior FIX PPX.

B-MORE is funded by Sobi.



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Artikel online veröffentlicht:
13. Februar 2025

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