Gene Therapy Decision-Making in Adults with Sickle Cell Anaemia in England

Introduction: Sickle cell disease is a genetic blood condition associated with significant physical health complications, which can progress and be potentially life-threatening. These complications can affect quality of life and adjustment across the lifespan. Recent advances in gene therapy offer curative potential; however, limited qualitative research has explored how adults understand and experience decision-making regarding gene therapy. This research aimed to address this gap.

Methods: Eight adults with sickle cell anaemia participated in semi-structured interviews. Data were analysed using Interpretative Phenomenological Analysis.

Results: Five group experiential themes and 14 sub-themes were developed through the interpretative process. Sickle cell anaemia had shaped who participants had become, yet they were clear that it did not define them. Participants approached gene therapy differently, influenced by their relationships, media representations, experiences of living with sickle cell anaemia, and the futures they imagined for themselves. While some participants were drawn towards gene therapy because of the possibility of relief from the challenges associated with sickle cell anaemia, others remained uncertain about moving away from a life they had learned to navigate, recognising that both options carried potential risks. Nevertheless, participants did not describe gene therapy as an opportunity to start again, but as a continuation of a life already shaped by living with the condition.

Conclusion: The findings highlight the importance of understanding gene therapy decisions within the biographies of adults with sickle cell anaemia. For participants, cure had implications for identity, relationships, and belonging, as well as for their health. Hope for relief therefore coexisted with uncertainty about what cure might mean. These findings can inform clinical conversations by recognising that decisions about gene therapy involve not only clinical risk and eligibility, but also what cure represents within an individual’s life.