Miesbach, Wolfgang; Oldenburg, Johannes; Klamroth, Robert; Eichler, Hermann; Koscielny, Jürgen; Holzhauer, Susanne; Holstein, Katharina; Kremer Hovinga, Johanna A; Alberio, Lorenzo; Olivieri, Martin; Knöfler, Ralf; Male, Christoph; Tiede, Andreas (2023). Gentherapie der Hämophilie: Empfehlung der Gesellschaft für Thrombose- und Hämostaseforschung (GTH). Hämostaseologie, 43(3), pp. 196-207. Thieme 10.1055/a-1957-4477
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Gene therapy has recently become a realistic treatment perspective for patients with haemophilia. Reviewing the literature and our personal experience from clinical trials, we discuss key aspects of haemophilia A and B gene therapy with vectors derived from adeno-associated virus (AAV), 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 centres 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 primary source of data for pharmacovigilance, post-marketing 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.
Item Type: |
Journal Article (Review Article) |
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Division/Institute: |
04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory |
UniBE Contributor: |
Kremer Hovinga Strebel, Johanna Anna |
Subjects: |
600 Technology > 610 Medicine & health 300 Social sciences, sociology & anthropology > 340 Law |
ISSN: |
2567-5761 |
Publisher: |
Thieme |
Language: |
English |
Submitter: |
Pierrette Durand Lüthi |
Date Deposited: |
13 Jan 2023 15:01 |
Last Modified: |
22 Jun 2023 00:12 |
Publisher DOI: |
10.1055/a-1957-4477 |
PubMed ID: |
36516966 |
BORIS DOI: |
10.48350/176932 |
URI: |
https://boris.unibe.ch/id/eprint/176932 |
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