Original Articles
Vol. 5 No. 2 (2026)
Development of a shared decision-making tool for gene therapy in hemophilia A in Italy
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All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
Published: 22 June 2026
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Background: Gene therapy offers a transformative treatment option for hemophilia A (HA) by restoring endogenous factor VIII production. Shared decision-making (SDM) is a collaborative process that allows patients and clinicians to weigh the risks and benefits of complex treatment options such as gene therapy. However, no SDM tools exist for the specific needs of Italian persons with HA.
Materials and Methods: An Expert Advisory Board comprising nine experts (including hematologists, a psychologist, a methodology expert, a linguist and patient advocacy group representatives) met three times between March and July 2024 to discuss gene therapy for HA in Italy, and develop an SDM tool to determine the suitability of gene therapy for HA.
Results: The resulting modular SDM tool allows for structured interactions between clinicians and patients, while remaining sensitive to each patient’s level of readiness and understanding. The tool includes three components: Role Definition, Agenda Alignment, and Intention Setting, with supporting materials to guide patient-clinician dialogue and various resources (questionnaires, brochures, and visual aids).
Conclusions: Despite requiring confirmation in further studies, this SDM tool may represent a useful component of patient-centered care for HA in the Italian healthcare setting.
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Citations
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Ethics Approval
GDM was a speaker or a member of a speaker bureau for BioMarin and CSL Behring; and was an ad-hoc speaker/consultant for BioMarin and CSL Behring. SBi and SBa have acted as consultants for BioMarin. EFG participated in speakers’ bureaus on behalf of a patient organization for BioMarin, CSL Behring, Novo Nordisk, Roche, and Sobi; participated in advisory boards on behalf of a patient organization for BioMarin, Pfizer, Roche, and Sobi; and participated in patient councils on behalf of a patient organization for BioMarin, Novo Nordisk, and Roche. MF has no competing interests to declare. MFM has acted as consultant and discus-sant for BioMarin, Novo Nordisk, Roche, and Sobi. MN has acted as consultant for Amgen, Bayer, CSL Behring, and Novo Nordisk; and received speaker fees from Amgen, Bayer, CSL Behring, Novartis, Novo Nordisk, Sanofi Genzyme, Sobi, and Takeda. RCS has acted as con-sultant for Bayer, Novo Nordisk, Roche, Sobi, and Takeda; and received speaker fees from Bayer, BioMarin, CSL Behring, Novo Nordisk, Kedrion, Roche, Sobi, and Takeda. GC received pay-ment or honoraria for lectures, presentations, speakers’ bureaus or educational events from Bi-oMarin, Bioviiix, CSL Behring, Kedrion, LFB, Novo Nordisk, Roche, Sanofi, Sobi, and Takeda, and participated on a Data Safety Monitoring Board or Advisory Board for Bayer, BioMarin, CSL Behring, Kedrion, LFB, Novo Nordisk, Pfizer, Roche, Sanofi, and TakedaCRediT authorship contribution
GDM, SBi, SBa, and GC contributed to developing the manuscript concept and plan; EFG, MF, MFM, MN, and RCS contributed to manuscript writing and editing. All the au-thors read and approved the final version of the manuscript and agreed to be accountable for all aspects of the work. Artificial intelligence (AI)-assisted technologies were not used in the creation of the submitted work.
Supporting Agencies
This project, the development of the manuscript and the open access publishing fee were supported by BioMarin, without conditions.How to Cite
1.
Di Minno G, Bigi S, Barello S, Ferri Grazzi E, Follino M, Mansueto MF, et al. Development of a shared decision-making tool for gene therapy in hemophilia A in Italy. Bleeding Thromb Vasc Biol [Internet]. 2026 Jun. 22 [cited 2026 Jun. 23];5(2). Available from: https://www.btvb.org/btvb/article/view/426
Copyright (c) 2026 The Author(s)
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