Titre
Gentherapie der Hämophilie: Empfehlung der Gesellschaft für Thrombose- und Hämostaseforschung (GTH) [Gene therapy of Hemophilia: Recommendations from the German, Austrian, and Swiss Society for Thrombosis and Haemostasis Research (GTH)]
Type
article
Institution
UNIL/CHUV/Unisanté + institutions partenaires
Périodique
Auteur(s)
Miesbach, W.
Auteure/Auteur
Oldenburg, J.
Auteure/Auteur
Klamroth, R.
Auteure/Auteur
Eichler, H.
Auteure/Auteur
Koscielny, J.
Auteure/Auteur
Holzhauer, S.
Auteure/Auteur
Holstein, K.
Auteure/Auteur
Hovinga, JAK
Auteure/Auteur
Alberio, L.
Auteure/Auteur
Olivieri, M.
Auteure/Auteur
Knöfler, R.
Auteure/Auteur
Male, C.
Auteure/Auteur
Tiede, A.
Auteure/Auteur
Liens vers les personnes
Liens vers les unités
ISSN
2567-5761
Statut éditorial
Publié
Date de publication
2023-06
Volume
43
Numéro
3
Première page
196
Dernière page/numéro d’article
207
Peer-reviewed
Oui
Langue
allemand
Notes
Publication types: English Abstract ; Journal Article
Publication Status: ppublish
Publication Status: ppublish
Résumé
Gene therapy has recently become a realistic treatment perspective for patients with hemophilia. Reviewing the literature and our personal experience from clinical trials, we discuss key aspects of hemophilia A and B gene therapy with vectors derived from adeno-associated virus, including predictable results, risks, adverse events, and patient-reported outcomes. Patient selection, informed consent, administration, and monitoring of gene therapy as well as data collection are explained. We also discuss the need for interdisciplinary cooperation with hepatology and other specialties. We emphasize structural and organizational requirements for treatment centers according to the hub-and-spoke model and recommend the use of electronic diaries to ensure safe and timely collection and exchange of data. Electronic diaries will play a key role as a primary source of data for pharmacovigilance, postmarketing clinical studies, national and international registries, as well as health technology and benefit assessment. Reimbursement aspects and the future of gene therapy in adolescents and children are also considered. In a rapidly evolving scientific environment, these recommendations aim to support treatment providers and payers to prepare for the implementation of gene therapy following marketing authorization.
PID Serval
serval:BIB_796C30B0331E
PMID
Open Access
Oui
Date de création
2022-12-19T08:51:15.507Z
Date de création dans IRIS
2025-05-21T00:04:31Z
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Nom
36516966_BIB_796C30B0331E.pdf
Version du manuscrit
published
Licence
https://creativecommons.org/licenses/by/4.0
Taille
643.11 KB
Format
Adobe PDF
PID Serval
serval:BIB_796C30B0331E.P001
URN
urn:nbn:ch:serval-BIB_796C30B0331E6
Somme de contrôle
(MD5):fd7bf3643ad99ab9790c9cbf14672e46