Beliefs and Values about gene therapy and gene editing in patients with hemophilia and their family members

Abstract

Hemophilia is an inherited lifelong debilitating disease for which current treatment is non curative. Currently, the promise of gene therapy and gene editing as potential cures are undergoing research. However, we don’t know whether or how the hemophilia community will accept them. In this study, we explored the beliefs and values about these new therapies in patients with hemophilia and their family members in order to bring up important research questions as the therapies are being developed. This is a qualitative study that uses semi structured interviews on patients with hemophilia A or B and their family members. The interview guide is an adaptation of Persaud et al 2018. Grounded theory was used to analyze the data. Participants perceived gene therapy and gene editing as a potential curative alternative to hemophilia. Gene therapy, as we described it, is the most accepted however patients and their family members would like to see more data first. On the other hand, they might not accept gene editing because of safety and ethical concerns. These data provide directions for supportive interventions to be delivered at the point of choice about gene therapy for hemophiliac patients and family and also make sure the questions that the patients have are answered during the research phase.