Delivery of AAV-based gene therapy through haemophilia centres - A need for re-evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC
- Introduction: Adeno-associated virus (AAV)-based gene therapy for haemophilia presents a challenge to the existing structure of haemophilia centres and requires a rethink of current collaboration and information exchange with the aim of ensuring a system that is fit-for-purpose for advanced therapies to maximise benefits and minimise risks. In Europe, a certification process based on the number of patients and facilities is offered to the haemophilia centres by European Haemophilia Network (EUHANET). Aim and methods: This joint European Association for Haemophilia and Allied Disorders (EAHAD) and European Haemophilia Consortium (EHC) publication describes criteria for centres participating in gene therapy care that require a reassessment of the infrastructure of comprehensive care and provides an outlook on how these criteria can be implemented in the future work of haemophilia centres. Results: The core definition of a haemophilia treatment centre remains, but additional roles could be implemented. A modifiable ‘hub-and-spoke’ model addresses all aspects associated with gene therapy, including preparation and administration of the gene therapy product, determination of coagulation and immunological parameters, joint.
Author: | Wolfgang MiesbachORCiDGND, Pratima Chowdary, Michiel Coppens, Daniel P. Hart, Victor Jiménez-Yuste, Robert KlamrothORCiDGND, Michael Makris, Declan Noone, Flora Peyvand |
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URN: | urn:nbn:de:hebis:30:3-641057 |
DOI: | https://doi.org/10.1111/hae.14420 |
ISSN: | 1365-2516 |
Parent Title (English): | Haemophilia |
Publisher: | Wiley-Blackwell |
Place of publication: | Oxford [u.a.] |
Document Type: | Article |
Language: | English |
Date of Publication (online): | 2021/09/22 |
Date of first Publication: | 2021/09/22 |
Publishing Institution: | Universitätsbibliothek Johann Christian Senckenberg |
Release Date: | 2022/01/12 |
Tag: | gene therapy; haemophilia care; haemophilia treatment; treatment centres |
Volume: | 2021 |
Issue: | online version before inclusion in an issue |
Page Number: | 7 |
First Page: | 1 |
Last Page: | 7 |
Note: | Open Access funding enabled and organized by Projekt DEAL. |
Note: | Early View: Online Version before inclusion in an issue |
HeBIS-PPN: | 491319843 |
Institutes: | Medizin |
Dewey Decimal Classification: | 6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit |
Sammlungen: | Universitätspublikationen |
Licence (English): | Creative Commons - Namensnennung-Nicht kommerziell 4.0 |