Gene therapy for cystic fibrosis: a psychosocial study of trial participants

Abstract

As yet little is known of psychological aspects of gene therapy. This pilot study aimed to investigate the attitudes, expectations, knowledge and psychological functioning of participants in a phase I safety trial of a single application of gene therapy to cystic fibrosis patients. Sixteen patients were assessed before and after the trial and at a 16 week follow-up using a semi-structured qualitative interview. They completed the Family Assessment Scale, the CF-12 quality of life measure and repeat measures of the Hospital Anxiety and Depression Scale and SF-36. Most patients had an emotionally driven optimism about gene therapy and few complaints about the procedures in a nasal trial. The majority of subjects were in good psychological health. A significant minority had anxiety disorders and this group were more likely to have concerns over the safety and promise of gene therapy. A preference was expressed for the potential of gene therapy over heart–lung transplantation. Results suggest that there are unlikely to be major psychological contraindications to proceeding with gene therapy research in this patient group. Patients with anxiety disorders may need special consideration. Qualitative data collected in this pilot study can now be used to develop survey questions for larger scale studies.