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

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...

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Bibliographic Details
Published in:Orphanet journal of rare diseases Vol. 17; no. 1; pp. 155 - 9
Main Authors: Fletcher, Simon, Jenner, Kathryn, Pembroke, Luke, Holland, Michael, Khair, Kate
Format: Journal Article
Language:English
Published: London BioMed Central 04.04.2022
Springer Nature B.V
BMC
Subjects:
Adult
Aged
Altruism
Clinical trial
Clinical trials
Clinical Trials as Topic
Decision making
Emotions
Factor IX deficiency
Family - psychology
Gene therapy
Genetic Therapy
Haematology
Haemophilia A
Haemophilia B
Hemophilia
Hemophilia A - drug therapy
Hemophilia A - therapy
Human Genetics
Humans
Immunosuppression
Informed consent
Medical research
Medicine
Medicine & Public Health
Middle Aged
Pharmacology/Toxicology
Qualitative Research
Rare diseases
Steroids
Transfection
Young Adult
United Kingdom > UK
Genetic therapy
Informed consent
Clinical trial
Haemophilia A
Haemophilia B
Decision making
ISSN:1750-1172, 1750-1172
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Summary: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 for the wider haemophilia community. However, participants reported being unprepared for the side effects of immunosuppression. Some also reported feeling unsupported and having little control over what was happening as their factor levels became the focus of the process. Conclusion The results suggest that strategies need to be put into place to enable PwH fully to understand the process of gene therapy, and thereby make an informed choice as to whether it is a treatment they might wish for themselves. These include early and ongoing education, increased provision of psychosocial support and ongoing qualitative research.
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ISSN:1750-1172
1750-1172
DOI:10.1186/s13023-022-02256-2