An analysis of attitudes toward gene therapy in people with severe hemophilia in Germany, a survey-based cross-sectional study.

Abstract

Background: Severe hemophilia traditionally requires lifelong prophylactic treatment, which cannot fully prevent joint damage and significantly impacts patients' quality of life. In recent years, gene therapy has been extensively researched as a potential cure with a single infusion, but it has not yet gained widespread acceptance.

Objectives: This study examines the knowledge and attitudes of people with severe hemophilia regarding gene therapy to provide guidance to practitioners in the context of shared decision-making.

Design: Cross-sectional observational study based on a standardized questionnaire.

Methods: A questionnaire developed by Cutica, Ilaria et al. was evaluated in a self-reported, anonymized survey for a German cohort of 59 people with severe hemophilia. It covered sociodemographic and clinical characteristics, knowledge, attitudes, risk tolerance, and decision-making preferences.

Results: Among 59 people with severe hemophilia A and 10 with severe hemophilia B, 59% expressed a predominantly rejective attitude toward gene therapy. Most had only general knowledge of gene therapy. Concerns about long-term side effects (e.g., cancer development) significantly contribute to a negative attitude toward gene therapy (p = 0.005). People with hemophilia and an annual bleeding rate ⩾1 show a significantly higher willingness to consider or accept gene therapy (p = 0.001). Rejection of gene therapy is significantly associated with high expectations regarding its duration of efficacy and the minimum clotting factor level.

Conclusion: Further studies are needed to refine educational concepts for people with hemophilia and clear out misconceptions about gene therapy.