An Analysis of Attitudes Toward Gene Therapy in People with Severe Haemophilia in Germany

Introduction Severe haemophilia 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. This study examines the knowledge and attitudes of people with severe haemophilia regarding gene therapy to provide guidance to practitioners in the context of shared decision-making. Fig. 1 Patient Knowledge on Gene Therapy. Fig. 1 displays the percentage of patients who answered knowledge questions on the topic of gene therapy for heamophilia correctly. It illustrates the need for educational interventions to bridge the information gaps among the affected population. Method A questionnaire developed by Cutica, Ilaria et al. was evaluated for a German cohort of people with severe haemophilia. It covered socio-demographic and clinical characteristics, knowledge, attitudes, risk tolerance, and decision-making preferences. Results Among 59 people with severe haemophilia A and 10 with severe haemophilia B, 59% expressed a predominantly rejective attitude towards 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 haemophilia and an ABR≥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. Fig. 2 Perceived Risks of Gene Therapy. The group of study participants who rejected gene therapy rated potential risks of the therapy slightly higher compared to those who were willing to accept it. Concerns about long-term side effects reached a high level of statistical significance. Conclusion Further studies are needed to refine educational concepts for people with haemophilia and clear out misconceptions about gene therapy [ 1 ] [ 2 ] [ 3 ] [ 4 ] [ 5 ] [ 6 ]. Publication History Article published online: 17 February 2026 © 2026. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany