Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia.

Autor: Mack HG; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia. hmack@eyesurgery.com.au.; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia. hmack@eyesurgery.com.au., Britten-Jones AC; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia.; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia.; Department of Optometry and Vision Sciences, University of Melbourne, Melbourne, VIC, Australia., McGuinness MB; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia.; Centre for Epidemiology and Biostatistics, Melbourne School of Population and Global Health, University of Melbourne, Melbourne, VIC, Australia., Chen FK; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia.; Centre for Ophthalmology and Visual Sciences (incorporating Lions Eye Institute), The University of Western Australia, Perth, WA, Australia.; Royal Perth Hospital and Perth Children's Hospital, Perth, WA, Australia., Grigg JR; Save Sight Institute, Faculty of Medicine and Health, The University of Sydney, Sydney, NSW, Australia.; Eye Genetics Research Unit, Sydney Children's Hospitals Network, Save Sight Institute, Children's Medical Research Institute, University of Sydney, Sydney, NSW, Australia., Jamieson RV; Eye Genetics Research Unit, Sydney Children's Hospitals Network, Save Sight Institute, Children's Medical Research Institute, University of Sydney, Sydney, NSW, Australia., Edwards TL; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia.; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia., De Roach J; Centre for Ophthalmology and Visual Sciences (incorporating Lions Eye Institute), The University of Western Australia, Perth, WA, Australia.; The Australian Inherited Retinal Disease Registry and DNA Bank, Perth, WA, Australia., O'Hare F; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia.; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia.; Department of Optometry and Vision Sciences, University of Melbourne, Melbourne, VIC, Australia., Martin KR; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia.; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia., Ayton LN; Ophthalmology, Department of Surgery, University of Melbourne, Melbourne, VIC, Australia.; Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, Melbourne, VIC, Australia.; Department of Optometry and Vision Sciences, University of Melbourne, Melbourne, VIC, Australia.
Jazyk: angličtina
Zdroj: Gene therapy [Gene Ther] 2023 Apr; Vol. 30 (3-4), pp. 336-346. Date of Electronic Publication: 2022 Oct 02.
DOI: 10.1038/s41434-022-00364-z
Abstrakt: Many 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.
(© 2022. The Author(s).)
Databáze: MEDLINE
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