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Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study.
- Source :
-
Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2023 Mar; Vol. 29 (2), pp. 435-444. Date of Electronic Publication: 2022 Dec 05. - Publication Year :
- 2023
-
Abstract
- Introduction: Current treatment for haemophilia A involves factor VIII replacement or non-replacement (emicizumab) therapies, neither of which permanently normalise factor VIII levels. Gene therapy using adeno-associated viral (AAV) vectors is an emerging long-term treatment strategy for people with severe haemophilia A (PwSHA) that is likely to be available for clinical use in the near future.<br />Aim: This article proposes practical guidelines for the assessment, treatment, and follow-up of potential PwSHA candidates for AAV-based gene therapy.<br />Method: Using the Delphi method, a working group of Italian stakeholders with expertise in and knowledge of the care of adults with haemophilia A analysed literature for AAV-based gene therapy and drafted a list of statements that were circulated to a panel of Italian peers. During two rounds of voting, panel members voted on their agreement with each statement to reach a consensus.<br />Results: The Delphi process yielded 40 statements regarding haemophilia A gene therapy, across five topics: (1) organisational model; (2) multidisciplinary team; (3) patient engagement; (4) laboratory surveillance; and (5) patient follow-up and gene therapy outcomes. The consensus was reached for all 40 statements, with the second round of voting needed for five statements.<br />Conclusion: Use of the hub-and-spoke organisational model and multidisciplinary teams are expected to optimise patient selection for gene therapy, as well as the management of dosing and patient follow-up, patient engagement, laboratory surveillance, and patient expectations regarding outcomes. This approach should allow the benefits of AAV-based gene therapy for haemophilia A to be maximised.<br /> (© 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.)
Details
- Language :
- English
- ISSN :
- 1365-2516
- Volume :
- 29
- Issue :
- 2
- Database :
- MEDLINE
- Journal :
- Haemophilia : the official journal of the World Federation of Hemophilia
- Publication Type :
- Academic Journal
- Accession number :
- 36469855
- Full Text :
- https://doi.org/10.1111/hae.14709