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Gene therapy for people with hemophilia B: a proposed care delivery model in Italy.
Castaman, Giancarlo; Di Minno, Giovanni; Simioni, Paolo; Molinari, Angelo Claudio; Siragusa, Sergio; Baldacci, Erminia; La Mura, Vincenzo; Lupi, Angelo; Grazzi, Enrico Ferri; Peyvandi, Flora.
Afiliación
  • Castaman G; Center for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.
  • Di Minno G; Department of Clinical Medicine, Federico II University, School of Medicine, Scientific Executive, Governance Headquarters, Campania Network for Inherited Bleeding Disorders, Napoli, Italy.
  • Simioni P; Hemorrhagic and Thrombotic Diseases Unit, Department of Medicine (DIMED), Padova University Hospital, Padova, Italy.
  • Molinari AC; Regional Reference Centre for Haemorrhagic Diseases, Department of Hemato Oncology, Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS) Istituto Giannina Gaslini, Genoa, Italy.
  • Siragusa S; Department of Health Promotion, Mother and Child Care, Internal Medicine and Medical Specialties (ProMISE), Unit of Hematology, University of Palermo and Policlinico Paolo Giaccone, Palermo, Italy.
  • Baldacci E; Haematology, "Umberto I" Policlinico, Department of Translational and Precision Medicine, Sapienza University of Rome, Rome, Italy.
  • La Mura V; Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy.
  • Lupi A; Federation of Haemophilia Associations (FedEmo), Milan, Italy.
  • Grazzi EF; Federation of Haemophilia Associations (FedEmo), Milan, Italy.
  • Peyvandi F; Istituti di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca' Granda Ospedale Maggiore Policlinico Foundation, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milano, Italy; Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy. Electronic address: flora.peyv
J Thromb Haemost ; 2024 Aug 22.
Article en En | MEDLINE | ID: mdl-39173877
ABSTRACT

BACKGROUND:

Gene therapy is designed to provide people with hemophilia B with a steady and elevated factor (F)IX activity, thereby strengthening protection and relieving the burden of frequent replacement therapy infusions. The European Medicines Agency has approved gene therapy for the severe and moderately severe forms of hemophilia B that uses the FIX-Padua variant (etranacogene dezaparvovec).

OBJECTIVES:

The aim was to provide a document dedicated to hemophilia B gene therapy and give a comprehensive overview of the topic.

METHODS:

An Italian group of experts in hemophilia carried out a narrative review of the literature and discussed during a virtual meeting several key aspects of the delivery of this treatment in Italy. The discussion covered the organizational model, the role of the multidisciplinary team, the laboratory surveillance, and the patient's journey, from the follow-up to the identification of safety issues and outcome measures.

RESULTS:

This article highlights the need to follow the Hub and Spoke organizational model and sheds light on the role of each professional figure within the multidisciplinary teams to favor patient engagement, management, and retention. Moreover, this article stresses the need to perform laboratory tests for patient screening and follow-up and proposes a checklist to help patient identification. Finally, the needs of Italian hemophilia centers have been considered to ensure an efficient implementation of the care delivery model.

CONCLUSION:

It is crucial to ensure that centers are appropriately organized, equipped, and trained to adequately select patients, deliver the gene therapy, and perform follow-up.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: J Thromb Haemost Asunto de la revista: HEMATOLOGIA Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: J Thromb Haemost Asunto de la revista: HEMATOLOGIA Año: 2024 Tipo del documento: Article País de afiliación: Italia Pais de publicación: Reino Unido