Real-World Effectiveness of rFIXFc Prophylaxis in Patients with Haemophilia B Switched from Standard Half-Life Therapy in Three European Countries

Research output: Contribution to journalJournal articleResearchpeer-review

  • Funding, Eva
  • Gillian Lowe
  • Lone H. Poulsen
  • Susan Shapiro
  • Johannes Oldenburg
  • Daniel Eriksson
  • Aletta Falk
  • Carly Rich

Introduction: The current study describes real-world clinical outcomes and factor usage among patients with haemophilia B switching from standard half-life factor IX (SHL FIX) treatment to recombinant factor IX Fc fusion protein (rFIXFc) prophylaxis in European treatment centres. Methods: This non-interventional, retrospective, multicentre chart review evaluated medical records from adult and paediatric patients with haemophilia B in Denmark, Germany and the UK. Patients had documented SHL FIX treatment, on-demand or prophylaxis, for ≥ 6 months before starting rFIXFc prophylaxis, and subsequent data for ≥ 6 months afterwards (up to 24 months). Primary endpoints included annualised bleeding rates (ABRs), prophylactic factor consumption and injection frequency. Results: Data from 30 patients (24/30 [80.0%] with severe disease) showed overall mean (standard deviation, SD) ABRs of 4.7 (6.3) on SHL FIX treatment and 1.7 (2.3) after switching to rFIXFc prophylaxis. The reduction in mean (SD) ABRs was greater when switching from SHL FIX on-demand treatment (n = 6), with a decrease from 10.5 (9.9) to 2.6 (4.5), than when switching from SHL FIX prophylaxis (n = 24), with a decrease from 3.3 (4.3) to 1.5 (1.4). Among prior SHL FIX prophylaxis patients, switching to rFIXFc prophylaxis increased the proportion of those with zero bleeds from 21.7% to 45.8% during the 6 months before and after switching, respectively. In the total population, five of six target joints (83.3%) present when patients started rFIXFc prophylaxis subsequently resolved. In patients switching from SHL FIX prophylaxis to rFIXFc prophylaxis, mean (SD) weekly injection frequency was reduced by 1.0 (0.7) and mean (SD) factor consumption was reduced by 27.7 (49.6) IU/kg/week. Conclusion: This study demonstrates the effectiveness of rFIXFc prophylaxis in real-world clinical practice. Improvements in both clinical effectiveness and factor usage associated with rFIXFc prophylaxis may potentially reduce patient burden and improve quality of life.

Original languageEnglish
JournalAdvances in Therapy
Volume40
Issue number9
Pages (from-to)3770-3783
Number of pages14
ISSN0741-238X
DOIs
Publication statusPublished - 2023

Bibliographical note

Funding Information:
The Kids B-LONG, B-LONG, and B-YOND studies were sponsored by Sobi and Sanofi. This analysis was funded by Sobi, who also funded the journal’s Rapid Service and Open Access Fees. Sobi and Sanofi reviewed and provided feedback on the manuscript.

Funding Information:
Medical writing and editorial support, funded by Sobi, was provided by Andy Lockley, PhD, Bioscript Group, Macclesfield, UK, based on the authors’ input and direction, and in accordance with Good Publication Practice (GPP3) guidelines ( http://www.ismpp.org/gpp3 ).

Publisher Copyright:
© 2023, The Author(s).

    Research areas

  • Annualised bleeding rate, Factor consumption, Factor IX, Haemophilia B, Injection frequency, Prophylaxis, Real-world, Recombinant factor IX Fc fusion protein, Standard half-life therapy

ID: 368136423