Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study.
Haemophilia
; 29(2): 435-444, 2023 Mar.
Article
in En
| MEDLINE
| ID: mdl-36469855
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.AIM:
This article proposes practical guidelines for the assessment, treatment, and follow-up of potential PwSHA candidates for AAV-based gene therapy.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.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.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.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Hemophilia A
Type of study:
Guideline
/
Prognostic_studies
Limits:
Humans
Country/Region as subject:
Europa
Language:
En
Journal:
Haemophilia
Journal subject:
HEMATOLOGIA
Year:
2023
Type:
Article
Affiliation country:
Italy