Laying the foundations for gene therapy in Italy for patients with haemophilia A: A Delphi consensus study.

Giancarlo Castaman Christian Carulli Raimondo De CristofaroMarco FollinoAngelo LupiMaria Elisa Mancuso Maria Francesca Mansueto Angelo Claudio MolinariPietro PasquettiCristina Santoro Rita Carlotta Santoro Sergio Siragusa Luigi Piero Solimeno Armando Tripodi Ezio Zanon Giovanni Di Minno

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2022)

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.

Keyphrases

gene therapy
case report
gene expression
adipose tissue
dna methylation
copy number
quality improvement
network analysis