The patient gene therapy journey: Findings from qualitative interviews with trial participants at one UK haemophilia centre

Abstract

Abstract Introduction Gene therapy for haemophilia is in late-stage clinical development and has the potential to become a therapeutic option in clinical practice. Aims To enhance the understanding of the perspectives of people with haemophilia around gene therapy, and to highlight their concerns about and motivations for having gene therapy. Method Structured, qualitative interviews were conducted and recorded with six people who had received an investigational gene therapy product. The recordings were transcribed and thematically analysed. Results Most of those interviewed were under the age of 40, and the mean time out from their gene therapy infusion was 10 months. Adverse events were the main concerns pre-infusion, and impact on quality of life was the main motivating factor for choosing to go ahead. Pre-infusion, the treating centre and the health care professionals working there were the main source of information regarding gene therapy; only two participants looked elsewhere for information to support their decision. None of the respondents expressed concerns about the infusion day itself, and all found the infusion to be simple or uneventful. Post-infusion, four found the frequency of follow-up appointments difficult, with time and travel the main issues. Conclusion Although participants' perspectives on gene therapy were generally positive, there remains a need for education and support. Nurses will play an important role in the delivery of gene therapy for haemophilia, but all staff within the haemophilia treatment centre should be armed with the knowledge and confidence to answer questions about gene therapy.