Parental perspectives on gene therapy for children with haemophilia: The Exigency study.
Haemophilia
child
decision-making
gene therapy
informed consent
parents
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
Jan 2021
Jan 2021
Historique:
received:
07
08
2020
revised:
15
10
2020
accepted:
15
10
2020
pubmed:
21
11
2020
medline:
25
9
2021
entrez:
20
11
2020
Statut:
ppublish
Résumé
Gene therapy is used in life-limiting conditions of childhood. While not a current therapeutic option for children with haemophilia, it may be considered in the future especially for those where access to treatment is limited. To assess the attitudes and opinions of parents of children with haemophilia about gene therapy as a potential future treatment, by understanding their awareness about gene therapy and what they need to know now and in the future; gauging levels of interest in gene therapy for their children; and exploring perceived current motivations and barriers. A mixed methods study with an online questionnaire and in-depth qualitative interviews in focus groups which were analysed using thematic analysis. One hundred and fifty-eight participants commenced the online survey; 63 were fully completed (39%). 60 had heard of gene therapy but few (17/60 [28.3%]) felt they had a good understanding. 38/60 (63.3%) respondents did not know that gene therapy is not available for children. However, most held positive views: 53/60 (88.3%) saying they would consider it for their child. In the interviews, participants (N = 10, all mothers) discussed their awareness and understanding of gene therapy and opinions about it for children, including how this should be communicated to the child and parents. A coherent, community-wide strategy for communicating information and news about gene therapy should now be provided for children and families living with haemophilia. This should come primarily from trusted haemophilia nursing teams, who can give tailored, age-appropriate, factual advice.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
120-128Informations de copyright
© 2020 John Wiley & Sons Ltd.
Références
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