Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline.

Giovanni Di Minno, Gaia Spadarella, Nelson Mauro Maldonato, Natascia De Lucia, Giancarlo Castaman, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Anna Borrelli, Angelo Lupi, Marco Follino, Gerardo Guerrino, Filomena Morisco, Matteo Di Minno

Journal: Blood reviews 2023;62():101118

PMID: 37544828

Full Text on Elsevier Science View on PubMed

Abstract

In clinical medicine, shared decision making (SDM) is a well-recognized strategy to enhance engagement of both patients and clinicians in medical decisions. The success of liver-directed gene therapy (GT) to transform severe congenital haemophilia A (HA) from an incurable to a curable disease has launched a shift beyond current standards of treatment. However, GT acceptance remains low in the community of HA persons. We argue for both persons with haemophilia (PWH) and specialists in HA care including clinicians, as needing SDM-oriented educational programs devoted to GT. Here, we provide an ad hoc outline to implement education to SDM and tailor clinician information on GT to individual PWHs. Based on routine key components of SDM: patient priorities; recommendations based on individual risk reduction; adverse effects; drug-drug interactions; alternatives to GT; and ongoing re-assessment of the objectives as risk factors (and individual priorities) change, this approach is finalized to exploit efficacious communication.

Address: Dipartimento di Medicina Clinica e Chirurgia, Naples, Italy. Electronic address: [email protected].; Dipartimento di Scienze Mediche Traslazionali, Naples, Italy. Electronic address: [email protected].; Dipartimento di Neuroscienze e di Scienze Riproduttive e Odontostomatologiche, "Federico II" University, Naples, Italy.; Dipartimento di Neuroscienze e di Scienze Riproduttive e Odontostomatologiche, "Federico II" University, Naples, Italy. Electronic address: [email protected].; Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy. Electronic address: [email protected].; Section of Haemorrhagic and Thrombotic Diseases, Department of Medicine and Translational Surgery, Sacred Heart University, Rome, Italy.. Electronic address: [email protected].; Ematologia, Azienda Ospedaliero-Universitaria Policlinico Umberto I, Rome, Italy. Electronic address: [email protected].; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, 20122 Milan, Italy; Università degli Studi di Milano, Department of Pathophysiology and Transplantation, Milan, Italy. Electronic address: [email protected].; Direzione Sanitaria, AOU "Federico II" Napoli, Italy.; Federazione delle Associazioni Emofilici (FedEmo), Milan, Italy. Electronic address: [email protected].; Fondazione Paracelso onlus, Milan, Italy. Electronic address: [email protected].; Chair, Campania Association for Haemophlia (ARCE), Italy.; Dipartimento di Medicina Clinica e Chirurgia, Naples, Italy. Electronic address: [email protected].; Dipartimento di Medicina Clinica e Chirurgia, Naples, Italy. Electronic address: [email protected].

DOI: 10.1016/j.blre.2023.101118

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MeSH Terms: Humans, Decision Making, Shared, Hemophilia A, Decision Making, Goals, Genetic Therapy, Liver