“I’d go on a hike with my kids… that would make me feel so happy.” Recognising what people with haemophilia B identify as meaningful when considering personal goals and the potential for gene therapy
Historically people with haemophilia B (PwHB) have experienced adverse clinical outcomes including joint bleeds, pain and arthropathy. The current management of severe and moderate haemophilia B in the UK is largely through the use of factor replacement therapy often given as prophylaxis. Despite th...
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| Main Authors: | , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Sciendo
2025-03-01
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| Series: | The Journal of Haemophilia Practice |
| Online Access: | https://doi.org/10.2478/jhp-2025-0001 |
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| Summary: | Historically people with haemophilia B (PwHB) have experienced adverse clinical outcomes including joint bleeds, pain and arthropathy. The current management of severe and moderate haemophilia B in the UK is largely through the use of factor replacement therapy often given as prophylaxis. Despite this, research reveals reduced quality of life (QoL) and psychological burden in PwHB of all severities, highlighting unmet need. Contemporary therapeutic developments including extended half-life factor IX products, novel non-factor agents, and gene therapy offer opportunities to address treatment-related issues and improve QoL. We investigated what PwHB identify as meaningful when considering their personal goals and the potential for gene therapy. |
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| ISSN: | 2055-3390 |