Gene therapy for hemophilia: results of ISTH global survey on current knowledge, attitudes, and preparedness of the hemophilia care team.

Background: Gene therapy has emerged as a transformative treatment for hemophilia, offering the potential for durable factor expression after a single administration. Since 2019, multiple gene therapies have been approved for adults with hemophilia A and B. However, the adoption of gene therapy into clinical practice has been slow, and provider preparedness remains uncertain. Objective: To assess current knowledge, perceptions, and clinical preparedness regarding hemophilia gene therapy among international healthcare professionals. Methods: An anonymous online survey was distributed internationally from August 24, 2023 to January 24, 2024 through four professional organizations. The 24-question survey included items on demographics, perceptions of gene therapy, and knowledge-based questions. Descriptive statistics were used to analyze responses. Results: A total of 327 participants from 66 countries responded. Top concerns with current therapies included dosing burden, inhibitor risk, and venous access. Only 7.5% of respondents were extremely familiar with regulatory guidance on gene therapy, and 35.6% were not comfortable answering patient questions about trials. Perceived barriers to adoption included uncertainty about durability, immune responses, eligibility, and cost. Conclusion: While clinical integration of gene therapy for hemophilia is advancing, this survey highlights persistent gaps in provider knowledge and confidence, especially related to regulatory guidance and clinical trial data. Targeted education and training initiatives are needed to support informed implementation of gene therapy in diverse clinical settings.