Exploration of the impact of gene therapy on the lives of people with haemophilia and their families: a protocol for the mixed-methods exigency study

Abstract

Introduction Gene therapy has the potential to change the life experience of people with haemophilia and their families. A growing number of studies have examined the experience for those who have had gene therapy. A few studies have examined the process with other gene therapy among a wider cross-section of the haemophilia community. Exigency is a nested group of studies investigating the experience and understanding of the haemophilia community to identify what place gene therapy is likely to have in haemophilia care. Five groups have been identified: those who have already undergone gene therapy, those who do not want it, those who wanted to have it but withdrew or were withdrawn before dosing, those who have not yet been offered it and parents of children with haemophilia. Methods A qualitative, mixed-methods process will identify what each group understands about gene therapy and what it might mean for the haemophilia community in the future. Analysis All of the transcripts will be analysed by the lead and coinvestigator using a grounded theory approach. The texts will be coded into themes for further analysis. The data will be summarised and synthesised, and the views expressed will be represented descriptively. Ethics and dissemination Written consent will be required, and participants will be anonymised. All elements of the study will be reviewed by UK statutory bodies. The study findings will be submitted for publication in peer-reviewed journals, and at haemophilia conferences and symposia. The study results will also be disseminated directly to study participants. Each participant will receive a copy of any publication and a summary report at the end of the study. Trial registration number NCT04723680