Background: Qualitative studies have explored why people with haemophilia (PwH) might consider or forgo gene therapy, the impact it has had on those who have received it, and what support might be needed throughout the process. No studies have yet examined what withdrawal prior to transfection might mean for PwH and their families.
Aims: To understand the experiences of PwH and their families of withdrawal from gene therapy and to understand what support mechanisms might be required.
Methods: Qualitative interviews were conducted with people with severe haemophilia who consented to take part in a gene therapy study in the UK but either withdrew or were withdrawn before transfection.
Results: Nine PwH and a family member were invited to this sub-study. Eight participants were recruited: six PwH (five haemophilia A and one haemophilia B) and two family members. Four participants were excluded from a study after consenting but before transfection, having failed to meet all the inclusion criteria; two withdrew after consenting but before transfection due to concerns including duration of factor expression and the time commitment involved in follow-up. The mean age of participants was 40.5 years (range 25-63 years). Two major themes emerged during the interviews: Expectation and loss.
Conclusions: PwH have many expectations about the difference gene therapy can make to their lives. Studies show that these expectations may not be fully realised. For those who have either withdrawn or been withdrawn from gene therapy, any expectations they had may now be unachievable. The nature of these expectations and the loss expressed by the participants indicate that support needs to be provided to help them and their families manage it.
Keywords: clinical trials; decision making; gene therapy; grief and loss; haemophilia.
© 2023 John Wiley & Sons Ltd.