Etranacogene dezaparvovec in people with hemophilia B and without adeno-associated virus serotype 5 neutralizing antibodies: a 4-year subgroup analysis of the Health Outcomes with Padua Gene; Evaluation in Hemophilia B (HOPE-B) trial
Etranacogene dezaparvovec proved to be highly effective, superior to FIX prophylaxis for bleeding protection, and safe for 4 years postinfusion in NAb- persons with severe or moderately severe hemophilia B.
Authors
- Sandra Le Quellec (ORCID: https://orcid.org/0000-0002-6203-3946)
- Richard S. Lemons (ORCID: https://orcid.org/0000-0002-2382-9247)
- Paul E. Monahan (ORCID: https://orcid.org/0000-0003-2110-0740)
- Niamh O’Connell (ORCID: https://orcid.org/0000-0002-7005-5328)
- Peter Verhamme (ORCID: https://orcid.org/0000-0001-8698-2858)
- Peter Kampmann
- Sean E. Gill (ORCID: https://orcid.org/0000-0003-4791-1576)
- Priyanka Raheja
- Fei Wang
- Frank W.G. Leebeek
Institutions
- University of Utah (US)
- Barts Health NHS Trust (GB)
- Erasmus MC (NL)
- Rigshospitalet (DK)
- CSL (United States) (US)
- KU Leuven (BE)
Publication Details
- Journal
- Research and Practice in Thrombosis and Haemostasis
- Published
- 2025-12-30
- DOI
- https://doi.org/10.1016/j.rpth.2025.103321
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- CSL Behring