Value contribution of etranacogene dezaparvovec gene therapy in moderately severe and severe haemophilia B through multi‐criteria decision analysis
ED brings added value in the treatment of moderately severe and severe HB (sHB) compared to current EHL rFIX, addressing the severity of the disease and increasing efficacy and patients' QoL especially related to the single dose and low bleeding rate. Concerns about long-term safety need to be addressed.
Authors
- Olga Benítez (ORCID: https://orcid.org/0000-0002-2883-9049)
- Xavier Badía (ORCID: https://orcid.org/0000-0001-7568-2550)
- José Luís Trillo (ORCID: https://orcid.org/0000-0002-5851-9038)
- Ramiro Núñez (ORCID: https://orcid.org/0009-0001-6055-1037)
- Víctor Jiménez‐Yuste (ORCID: https://orcid.org/0000-0003-3937-3499)
- Daniel‐Anibal Garcia Diego (ORCID: https://orcid.org/0000-0001-9410-3825)
- Joan‐Antoni Vallés
- J.C. Juárez Giménez (ORCID: https://orcid.org/0000-0001-5859-986X)
- José Luís Poveda (ORCID: https://orcid.org/0000-0003-1800-6198)
Institutions
- Hospital Universitario La Paz (ES)
- Hospital Universitari i Politècnic La Fe (ES)
- Oblikue Consulting (Spain) (ES)
- Federación Española de Enfermedades Raras (ES)
- Vall d'Hebron Hospital Universitari (ES)
- Hospital Universitario Virgen del Rocío (ES)
- Institut Català de la Salut (ES)
Publication Details
- Journal
- Haemophilia
- Published
- 2024-09-28
- DOI
- https://doi.org/10.1111/hae.15096
- Citations
- 4
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 1.82