PB0189 - Knowledge and attitudes of haemophilic patients toward gene therapy

Background: Although gene therapy (GT) is recently become a new treatment option for haemophilia A (HA) and B (HB), very little is known about patients’ knowledge and attitudes toward it. Some previous studies found a general lack of knowledge and education, but to our knowledge no study investigated patients’ attitudes toward GT, their concerns, information needs, nor how these factors might impact on their willingness to accept it.

Aims: This study intends to evaluate the Italian haemophilic patients’ knowledge and attitudes toward GT.

Methods: Participants were enrolled among HA and HB patients referring to Angelo Bianchi Bonomi Hemophilia Thrombosis Center (Milan, Italy). Participants were asked to fill a questionnaire assessing: (1) Clinical Data; (2) GT knowledge; (3) Willingness to accept GT, perceived benefits and concerns, information needs.

Results: Eighty-five patients participated in the study. 87% of participants had HA; 80% had severe hemophilia. 93% were satisfied with their current treatment that is, in 80% of participants, regular prophylaxis. Patients show a poor or very poor knowledge of GT, with a few exceptions (almost 30% of patients gave less than 4 correct answers out of 17, and 75% less than 9). Percentages of correct answers for each question are shown in Table 1.
Table 2 reports the most appealing benefits and the most frequent concerns for GT.
As regards willingness to undergo GT, 51% of participants would not accept it even when offered by doctors. Factors that most influenced the willingness not to undergo GT were: higher fear that the treatment would lose effectiveness over time; higher fear of short- and long-time collateral effects; lower knowledge of GT.

Conclusion(s): Overall, patients showed a poor knowledge of GT. As a greater knowledge corresponds to an increase in willingness to undergo GT, there is a need for a widespread information campaign on GT.