Evolution of Hemophilia Management in Italy: Results from a Delphi Consensus Study

Background/Objectives: The clinical management of hemophilia has evolved with the introduction of innovative therapies that are reshaping treatment paradigms. Real-world implementation of these approaches may vary across centers. This modified Delphi consensus study aimed to explore current clinical perspectives on hemophilia management in Italy among healthcare professionals. Methods: Eight hemophilia experts developed 38 statements covering seven domains, including therapeutic personalization, adherence and patient engagement, follow-up strategies, inhibitor management, comorbidity management in patients, musculoskeletal monitoring, and organizational aspects of care. Statements were evaluated through an anonymous web-based survey in a modified single-round Delphi process using a 5-point Likert scale. Consensus was defined a priori as ≥70% agreement or disagreement after exclusion of neutral responses. Results: Thirty-four clinicians from specialized hemophilia centers participated in the survey. Strong agreement emerged regarding the importance of personalized treatment strategies, active patient involvement in therapeutic decisions, and multidisciplinary management, particularly in ageing patients with comorbidities. Panelists also supported the role of joint ultrasound in monitoring musculoskeletal health and highlighted adherence as a key determinant of treatment outcomes. Areas of heterogeneity included criteria for transitioning between intravenous and subcutaneous therapies, the implementation of structured transition programs from pediatric to adult care, and the frequency of musculoskeletal imaging. Limited availability of psychological support and variable integration of patient-reported outcomes were also identified. Conclusions: Italian hemophilia specialists showed substantial agreement across key domains; however, future work should address remaining organizational and clinical gaps and incorporate patient perspectives to support more integrated and patient-centered management strategies for people with hemophilia.

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Publication Details

Journal
Journal of Clinical Medicine
Published
2026-07-22
DOI
https://doi.org/10.3390/jcm15145741
Primary Topic
Hemophilia Treatment and Research
Type
article
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article

Evolution of Hemophilia Management in Italy: Results from a Delphi Consensus Study

Ezio Zanon, C. Santoro, Francesca Gatto, Giancarlo Castaman et al.
Journal of Clinical Medicine
Hemophilia Treatment and Research
article

Evolution of Hemophilia Management in Italy: Results from a Delphi Consensus Study

Ezio Zanon, C. Santoro, Francesca Gatto, Giancarlo Castaman, Raimondo De Cristofaro, Rita Santoro, Matteo Nicola Dario Di Minno, Angelo Claudio Molinari, Mariasanta Napolitano, P Mariani
article en

Abstract

Background/Objectives: The clinical management of hemophilia has evolved with the introduction of innovative therapies that are reshaping treatment paradigms. Real-world implementation of these approaches may vary across centers. This modified Delphi consensus study aimed to explore current clinical perspectives on hemophilia management in Italy among healthcare professionals. Methods: Eight hemophilia experts developed 38 statements covering seven domains, including therapeutic personalization, adherence and patient engagement, follow-up strategies, inhibitor management, comorbidity management in patients, musculoskeletal monitoring, and organizational aspects of care. Statements were evaluated through an anonymous web-based survey in a modified single-round Delphi process using a 5-point Likert scale. Consensus was defined a priori as ≥70% agreement or disagreement after exclusion of neutral responses. Results: Thirty-four clinicians from specialized hemophilia centers participated in the survey. Strong agreement emerged regarding the importance of personalized treatment strategies, active patient involvement in therapeutic decisions, and multidisciplinary management, particularly in ageing patients with comorbidities. Panelists also supported the role of joint ultrasound in monitoring musculoskeletal health and highlighted adherence as a key determinant of treatment outcomes. Areas of heterogeneity included criteria for transitioning between intravenous and subcutaneous therapies, the implementation of structured transition programs from pediatric to adult care, and the frequency of musculoskeletal imaging. Limited availability of psychological support and variable integration of patient-reported outcomes were also identified. Conclusions: Italian hemophilia specialists showed substantial agreement across key domains; however, future work should address remaining organizational and clinical gaps and incorporate patient perspectives to support more integrated and patient-centered management strategies for people with hemophilia.

Journal of Clinical MedicineVol. 15(14)
University of Padua (IT), Istituto Giannina Gaslini (IT), University Hospital Foundation (CA), Azienda Ospedaliero-Universitaria Careggi (IT), Presidio Ospedaliero (IT), Pfizer (Italy) (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), University of Milano-Bicocca (IT), University of Naples Federico II (IT), Sapienza University of Rome (IT), University of Palermo (IT)
Reduced inequalities
Openalex Percentile: Top 9%
Hemophilia Treatment and Research
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