Abstract Number: PB0660
Meeting: ISTH 2021 Congress
Background: Gene therapy for haemophilia has the potential to change the life experience of individuals and their families. Few studies have sought to explore the impact of gene therapy on individuals and their families.
Aims: The aim of the Exigency study is to capture the real-life experience of gene therapy in people with haemophilia and their families.
Methods: Participants who had undergone gene therapy were recruited by specialist centres, social media and word of mouth referral. Each participant and a family member when available took part in a semi-structured qualitative interview which was recorded, transcribed verbatim and analysed thematically.
All elements of the study were reviewed by local statutory authorities and informed consent was sought in all cases.
Results: To date, nine participants (eight with haemophilia A and one with haemophilia B), mean age 43.1 years (range 23-68 years), along with a family member, took part in a single qualitative interview online (n=8), or in person (n=1). Mean time since transfection was 1.89 years (range 1-5 years).
Five major themes have emerged: freedom; altruism; side effects of immunosuppression; loss of factor expression; and loss of control.
Conclusions: Most of those interviewed saw participation in gene therapy as a positive experience: it had freed them from the personal burden of haemophilia and furthered treatment options for the wider haemophilia community. Participants however reported being unprepared for the side effects of immunosuppression. Some also reported feeling unsupported and as having little control over what was happening as their factor levels became the focus of the process.
The results so far suggest that psychosocial support needs to be integrated into gene therapy programmes to help participants and families process their experiences both positive and negative.
Interviews are ongoing to confirm these early data and to identify further themes.
To cite this abstract in AMA style:Fletcher S, Chaplin S, Holland M, Jenner K, Khair K. Exigency: The Real-world Impact of Gene Therapy on the Lives of People with Haemophilia and their Families. An Interim Report [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/exigency-the-real-world-impact-of-gene-therapy-on-the-lives-of-people-with-haemophilia-and-their-families-an-interim-report/. Accessed December 6, 2023.
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