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

Giancarlo CastamanChristian CarulliRaimondo De CristofaroMarco FollinoAngelo LupiMaria Elisa MancusoMaria Francesca MansuetoAngelo Claudio MolinariPietro PasquettiCristina SantoroRita Carlotta SantoroSergio SiragusaLuigi Piero SolimenoArmando TripodiEzio ZanonGiovanni 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.