Abstract
AbstractMany gene therapies are in development for treating people with inherited retinal diseases (IRD). We hypothesized that potential recipients of gene therapy would have knowledge gaps regarding treatment. We aimed to assess knowledge, attitudes, and perceptions of genetic therapies among potential recipients with IRD, using a novel instrument we designed (Attitudes to Gene Therapy-Eye (AGT-Eye)) and their associations with demographic data, self-reported visual status, and tools assessing quality of life and attitudes toward clinical trials using a community-based cross-sectional survey of Australian adults with IRD. AGT-Eye, overall quality of life EQ-5D-5L, National Eye Institute Visual Functioning Questionnaire (NEI-VFQ-25) and Patient Attitudes to Clinical Trials (PACT-22) instruments were administered. Six hundred and eighty-one people completed the study, 51.7% women of mean age 53.5 years (SD ± 15.8). Most participants (91.6%) indicated they would likely accept gene therapy if it was available to them or family members. However, only 28.3% agreed that they had good knowledge of gene therapy. Most obtained information about gene therapy from the internet (49.3%). Respondents with post-graduate degrees scored highest compared to other educational levels on methods (p < 0.001) and outcomes (p = 0.003) and were more likely to see economic value of treatment (p = 0.043). Knowledge gaps were present regarding methods and outcomes of gene therapy. This survey has shown high level of interest in the IRD community for gene therapies, and highlights areas for improved clinician and patient education.
Funder
Retina Australia, grant not numbered
Department of Health | National Health and Medical Research Council
Victorian State Government Operational Infrastructure Support Program. Grant not numbered.
Retina Australia, grant not numbered.
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference44 articles.
1. Federal Drug Administration. FDA approval brings first gene therapy to the United States. 2022. https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states.
2. Federal Drug Administration. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease.
3. Federal Drug Administration. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss.
4. Maguire AM, Russell S, Chung DC, Yu Z-F, Tillman A, Drack AV, et al. Durability of Voretigene Neparvovec for biallelic RPE65-mediated inherited retinal disease: phase 3 results at 3 and 4 years. Ophthalmology. 2021;128:1460–8.
5. Britten-Jones AC, Jin R, Gocuk SA, Cichello E, O’Hare F, Hickey DG. et al. The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: a systematic review. Genet Med.2021;S1098-3600:05365-X. https://doi.org/10.1016/j.gim.2021.10.013.
Cited by
4 articles.
订阅此论文施引文献
订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献