Christiana Dinah,Jamie Enoch,Arevik Ghulakhszian,Mandeep Sekhon,Serena Salvatore,Gabriella DeSalvo,Praveen Kumar,Sanjiv Banerjee,Devaki Nayak,Winfried Amoaku,Marianne Shiew,Olayinka Osoba,David P Crabb,Deanna J Taylor
{"title":"Patient-Reported Importance of Functional Benefit in Geographic Atrophy.","authors":"Christiana Dinah,Jamie Enoch,Arevik Ghulakhszian,Mandeep Sekhon,Serena Salvatore,Gabriella DeSalvo,Praveen Kumar,Sanjiv Banerjee,Devaki Nayak,Winfried Amoaku,Marianne Shiew,Olayinka Osoba,David P Crabb,Deanna J Taylor","doi":"10.1001/jamaophthalmol.2025.3264","DOIUrl":null,"url":null,"abstract":"Importance\r\nIntravitreal complement inhibitors injections (IVCIs) slowed progression of geographic atrophy (GA) lesions in several registration phase 3 trials although without benefit for prespecified secondary functional vision outcomes. Patient acceptability of these therapies needs further exploration.\r\n\r\nObjective\r\nTo quantify the acceptability of IVCI therapy to United Kingdom patients with GA, assuming vision outcome benefits are expected.\r\n\r\nDesign, Setting, and Participants\r\nThis cross-sectional study took place at 9 geographically dispersed UK National Health Service centers from April 2023 to April 2024 among 153 participants with treatment-naive GA in at least 1 eye.\r\n\r\nExposures\r\nGA in at least 1 eye.\r\n\r\nMain Outcomes and Measures\r\nMain outcomes were (1) acceptability of IVCI therapy based on completion of validated acceptability questionnaire. Participants were provided with a treatment information leaflet coproduced by a patients with lived experience of GA to inform them about the risks and benefits of IVCI for GA, assuming there were vision outcome benefits to this treatment and (2) response to the EuroQol 5-dimension with a vision bolt-on questionnaire to assess general health and vision-related quality of life. Spearman rank correlations and χ2 tests were used to explore associations between acceptability levels and specific ocular and sociodemographic characteristics.\r\n\r\nResults\r\nA total of 153 participants were recruited (93 [60%] women; mean [SD] age, 82 [7]), 57 (38%) of whom had bilateral foveal involvement. Median (IQR) visual acuity with habitual correction in the better-seeing eye and in eyes where neither eye was better or worse was logMAR, 0.30 (0.14-0.54; approximate Snellen equivalent, 20/40) and 0.47 (0.14-0.84; approximate Snellen equivalent, 20/63), respectively. Among the 153 participants, 81 (53%; 95% CI, 45-61) reported IVCIs were very much or extremely acceptable under the theoretical scenarios provided. The proportion finding IVCIs acceptable rose to 82% (95% CI, 76-88) when including those who rated prospective treatment as moderately acceptable. Belief in the perceived effectiveness of the treatment (ρ, 0.52; 95% CI, 0.40-0.63; P < .001) and confidence in their ability to attend the eye clinic regularly (ρ, 0.51; 95% CI, 0.38-0.62; P < .001) correlated with overall acceptability.\r\n\r\nConclusions and Relevance\r\nIVCI therapy for GA may be acceptable to most UK patients with GA under the assumption that there are vision outcome benefits to this treatment. While current treatments do not result in vision outcome benefits, perceived effectiveness by patients was associated with acceptability, emphasizing the desire to quantify vision functional benefit concomitant with anatomical slowing of progression.","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"15 1","pages":""},"PeriodicalIF":9.2000,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"JAMA ophthalmology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1001/jamaophthalmol.2025.3264","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"OPHTHALMOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Importance
Intravitreal complement inhibitors injections (IVCIs) slowed progression of geographic atrophy (GA) lesions in several registration phase 3 trials although without benefit for prespecified secondary functional vision outcomes. Patient acceptability of these therapies needs further exploration.
Objective
To quantify the acceptability of IVCI therapy to United Kingdom patients with GA, assuming vision outcome benefits are expected.
Design, Setting, and Participants
This cross-sectional study took place at 9 geographically dispersed UK National Health Service centers from April 2023 to April 2024 among 153 participants with treatment-naive GA in at least 1 eye.
Exposures
GA in at least 1 eye.
Main Outcomes and Measures
Main outcomes were (1) acceptability of IVCI therapy based on completion of validated acceptability questionnaire. Participants were provided with a treatment information leaflet coproduced by a patients with lived experience of GA to inform them about the risks and benefits of IVCI for GA, assuming there were vision outcome benefits to this treatment and (2) response to the EuroQol 5-dimension with a vision bolt-on questionnaire to assess general health and vision-related quality of life. Spearman rank correlations and χ2 tests were used to explore associations between acceptability levels and specific ocular and sociodemographic characteristics.
Results
A total of 153 participants were recruited (93 [60%] women; mean [SD] age, 82 [7]), 57 (38%) of whom had bilateral foveal involvement. Median (IQR) visual acuity with habitual correction in the better-seeing eye and in eyes where neither eye was better or worse was logMAR, 0.30 (0.14-0.54; approximate Snellen equivalent, 20/40) and 0.47 (0.14-0.84; approximate Snellen equivalent, 20/63), respectively. Among the 153 participants, 81 (53%; 95% CI, 45-61) reported IVCIs were very much or extremely acceptable under the theoretical scenarios provided. The proportion finding IVCIs acceptable rose to 82% (95% CI, 76-88) when including those who rated prospective treatment as moderately acceptable. Belief in the perceived effectiveness of the treatment (ρ, 0.52; 95% CI, 0.40-0.63; P < .001) and confidence in their ability to attend the eye clinic regularly (ρ, 0.51; 95% CI, 0.38-0.62; P < .001) correlated with overall acceptability.
Conclusions and Relevance
IVCI therapy for GA may be acceptable to most UK patients with GA under the assumption that there are vision outcome benefits to this treatment. While current treatments do not result in vision outcome benefits, perceived effectiveness by patients was associated with acceptability, emphasizing the desire to quantify vision functional benefit concomitant with anatomical slowing of progression.
期刊介绍:
JAMA Ophthalmology, with a rich history of continuous publication since 1869, stands as a distinguished international, peer-reviewed journal dedicated to ophthalmology and visual science. In 2019, the journal proudly commemorated 150 years of uninterrupted service to the field. As a member of the esteemed JAMA Network, a consortium renowned for its peer-reviewed general medical and specialty publications, JAMA Ophthalmology upholds the highest standards of excellence in disseminating cutting-edge research and insights. Join us in celebrating our legacy and advancing the frontiers of ophthalmology and visual science.