The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.

ABSTRACT:

Background

Gene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families. The aim of this study was to capture real-life experiences of gene therapy in People with haemophilia and their families.

Results

Sixteen participants with severe haemophilia (11 haemophilia A, five haemophilia B), mean age 41.4 years (range 23-75 years), took part in a single qualitative interview; ten were accompanied by a family member. Mean time since transfection was 3.56 years (range 1-10 years). Participants saw their involvement in gene therapy as a positive experience, freeing them from the personal burden of haemophilia and furthering treatment options f

SUBMITTER: Fletcher S 

PROVIDER: S-EPMC8981747 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature