TY  - JOUR
A1  - Miesbach, Wolfgang
A1  - Chowdary, Pratima
A1  - Coppens, Michiel
A1  - Hart, Daniel P.
A1  - Jiménez-Yuste, Victor
A1  - Klamroth, Robert
A1  - Makris, Michael
A1  - Noone, Declan
A1  - Peyvand, Flora
T1  - 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
T2  - Haemophilia
N2  - 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.
KW  - gene therapy
KW  - haemophilia care
KW  - haemophilia treatment
KW  - treatment centres
Y1  - 2021
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/64105
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-641057
SN  - 1365-2516
N1  - Open Access funding enabled and organized by Projekt DEAL.
N1  - Early View: Online Version before inclusion in an issue
VL  - 2021
IS  - online version before inclusion in an issue
SP  - 1
EP  - 7
PB  - Wiley-Blackwell
CY  - Oxford [u.a.]
ER  -