JAMA ophthalmology最新文献

筛选
英文 中文
Axial Growth and Myopia Progression After Discontinuing Soft Multifocal Contact Lens Wear. 停止佩戴软性多焦点隐形眼镜后眼轴生长和近视进展。
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.5885
David A Berntsen,Anita Ticak,Danielle J Orr,Amber Gaume Giannoni,Loraine T Sinnott,Donald O Mutti,Lisa A Jones-Jordan,Jeffrey J Walline,
{"title":"Axial Growth and Myopia Progression After Discontinuing Soft Multifocal Contact Lens Wear.","authors":"David A Berntsen,Anita Ticak,Danielle J Orr,Amber Gaume Giannoni,Loraine T Sinnott,Donald O Mutti,Lisa A Jones-Jordan,Jeffrey J Walline,","doi":"10.1001/jamaophthalmol.2024.5885","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5885","url":null,"abstract":"ImportanceFor myopia control to be beneficial, it would be important that the benefit of treatment (slowed eye growth) is not lost because of faster than normal growth (rebound) after discontinuing treatment.ObjectiveTo determine whether there is a loss of treatment effect (rebound) after discontinuing soft multifocal contact lenses in children with myopia.Design, Setting, and ParticipantsThe Bifocal Lenses in Nearsighted Kids 2 (BLINK2) cohort study involved children with myopia (aged 11-17 years at BLINK2 baseline) who completed the BLINK Study randomized clinical trial. Enrollment was from September 2019 through January 2021; follow-up was completed in January 2024. In the BLINK2 Study, all children wore high-add (+2.50 diopter [D]) multifocal soft contact lenses for 2 years and single-vision soft contact lenses during the third year to determine if rebound occurred.ExposureHigh-add multifocal soft contact lenses and single-vision soft contact lenses.Main Outcomes and MeasuresEye length (optical biometry) and refractive error (cycloplegic autorefraction) were measured annually.ResultsOf 248 participants enrolled in BLINK2, 235 completed the study. The median age at the baseline visit was 15 years (range, 11-17 years); 146 participants (59%) were female, and 102 (41%) were male. At baseline for BLINK2, mean (SD) axial length and spherical equivalent refractive error were 25.2 (0.9) mm and -3.40 (1.40) D, respectively. After participants switched from multifocal to single-vision contact lenses, axial elongation increased by 0.03 mm per year (95% CI, 0.01 to 0.05) regardless of their original BLINK treatment assignment (P = .81). There was also an increase in myopia progression after switching to single-vision lenses of -0.17 D per year (95% CI, -0.22 to -0.12) that did not depend on the original BLINK treatment assignment (P = .57). There continued to be a difference in axial length and refractive error throughout BLINK2 based on the BLINK Study treatment assignment with the original high-add group having shorter eyes and less myopia than the original medium-add (+1.50 D) and single-vision groups.Conclusions and RelevanceThe BLINK2 Study found no evidence of a loss of treatment effect after discontinuing multifocal contact lenses in older teenagers. These data suggest eye growth and myopia progression returned to faster but age-expected rates and support continuing multifocal lenses until cessation of elongation and progression.","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"30 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142988586","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Representation Quotients to Examine Diversity in Ophthalmology Residency Applicants and Matriculants 代表性商数以检验眼科住院医师申请人和毕业生的多样性
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.5863
Saba S. Paracha, Shani A. Williams, Alizeh Shamshad, Elijah M. Persad-Paisley, Michael E. Migliori
{"title":"Representation Quotients to Examine Diversity in Ophthalmology Residency Applicants and Matriculants","authors":"Saba S. Paracha, Shani A. Williams, Alizeh Shamshad, Elijah M. Persad-Paisley, Michael E. Migliori","doi":"10.1001/jamaophthalmol.2024.5863","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5863","url":null,"abstract":"ImportanceA diverse ophthalmic workforce is key to equitable care, and identifying areas of underrepresentation is important in tackling vision care disparities.ObjectiveTo evaluate the diversity of applicants and matriculants in ophthalmology residency programs relative to medical school graduates and analyze the intersection of race and gender within this pathway.Design, Setting, and ParticipantsThis retrospective cross-sectional study conducted from 2022 to 2024 used representation quotients (RQs) using reports from the Association of University Professors of Ophthalmology and San Francisco Match, along with demographic data from the Association of American Medical Colleges from 2008 to 2021. RQs measure equity by dividing a racial, ethnic, or gender group’s proportion in a specific population by its proportion in a larger reference population. Individuals who graduated from US medical schools from 2008 to 2021 or who applied to or matriculated into US ophthalmology residency programs from 2015 to 2021 and from 2008 to 2021, respectively, were eligible for inclusion.ExposureApplying to or matriculating into ophthalmology residency.Main Outcomes and MeasuresThe primary outcome was median RQs and trends among racial, ethnic, and gender groups for ophthalmology applicants (RQ<jats:sub>app</jats:sub>) and matriculants (RQ<jats:sub>mat</jats:sub>), with RQ differences evaluated using Mann-Whitney <jats:italic>U</jats:italic> tests.ResultsAmong total applicants (n = 5304) and matriculants (n = 6139), 36% and 42% of applicants and matriculants, respectively, were female (applicants: 28% Asian, 4% Black, 7% Hispanic, and 47% White; matriculants: 31% Asian, 3% Black, 5% Hispanic, and 57% White). Self-identified Black individuals had the lowest median (IQR) RQs (RQ<jats:sub>app</jats:sub>, 0.604 [0.437-0.771]; RQ<jats:sub>mat</jats:sub>, 0.469 [0.341-0.597]). Regression analysis revealed increased representation for male applicants (slope, 0.036; 95% CI, 0.015-0.057; <jats:italic>P</jats:italic> = .007) and matriculants (slope, 0.009; 95% CI, 0.002-0.016; <jats:italic>P</jats:italic> = .02), but decreased representation for female applicants (slope, –0.031; 95% CI, –0.010 to –0.102; <jats:italic>P</jats:italic> = .01) and matriculants (slope, –0.009; 95% CI, –0.016 to –0.002; <jats:italic>P</jats:italic> = .02). Black (RQ<jats:sub>app</jats:sub>, 0.604 vs RQ<jats:sub>mat</jats:sub>, 0.469; <jats:italic>P</jats:italic> = .047) and Hispanic (RQ<jats:sub>app</jats:sub>, 1.46 vs RQ<jats:sub>mat</jats:sub>, 1.04; <jats:italic>P</jats:italic> = .03) groups experienced a decrease between applicant and matriculants.Conclusions and RelevanceThis cross-sectional retrospective study found persistent underrepresentation of Black and female individuals in ophthalmology residency programs, with increases in Black and Hispanic applicants failing to translate into higher matriculation rates. Barriers that female or Black medical students face when applying or matricula","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"8 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142986886","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Retinal Vasculitis Associated With Castleman Disease 与Castleman病相关的视网膜血管炎
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.5152
Yujing Qian, Meifen Zhang
{"title":"Retinal Vasculitis Associated With Castleman Disease","authors":"Yujing Qian, Meifen Zhang","doi":"10.1001/jamaophthalmol.2024.5152","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5152","url":null,"abstract":"This case report describes a diagnosis of retinal vasculitis associated with Castleman disease in a man who presented with 6 months of progressive floaters bilaterally and no change in visual acuity.","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"205 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142986917","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Temperature Excursion of Intravitreal Anti-VEGF Agents During Home Storage 玻璃体内抗vegf药物在家庭储存过程中的温度偏移
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.5884
Inès Ben Ghezala, Laure-Anne Steinberg, Maxime Sibert, Aline Lazzarotti, Aude Ambresin, Pierre-Henry Gabrielle, Catherine Creuzot-Garcher
{"title":"Temperature Excursion of Intravitreal Anti-VEGF Agents During Home Storage","authors":"Inès Ben Ghezala, Laure-Anne Steinberg, Maxime Sibert, Aline Lazzarotti, Aude Ambresin, Pierre-Henry Gabrielle, Catherine Creuzot-Garcher","doi":"10.1001/jamaophthalmol.2024.5884","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5884","url":null,"abstract":"ImportanceSome patients worldwide are asked to acquire an anti–vascular endothelial growth factor (anti-VEGF) agent from a pharmacy, store it, and then bring it to a physician for intravitreal injection (IVT). Anti-VEGF agents must be stored in the refrigerator to avoid bacterial contamination or denaturation. Some cases of severe intraocular inflammation have been reported following IVT of more recently approved anti-VEGF agents, which might be explained by thermal instability.ObjectiveTo investigate whether patients followed the storage temperature guidelines for intravitreal anti-VEGF agents in daily clinical practice.Design, Setting, and ParticipantsThis quality improvement study, performed between May 27 and June 7, 2024, at the Ophthalmology Department of Dijon University Hospital in Dijon, France, included all consecutive patients with new or renewed prescriptions for intravitreal anti-VEGF agents for any macular disease.Main Outcomes and MeasuresAll participants were given a time-temperature indicator that changed color according to brief (2-12 hours), moderate (12-48 hours), and prolonged (≥48 hours) exposure over 8 °C. The indicator was activated at the time of the anti-VEGF agent delivery to the participant in community pharmacies.ResultsDuring the study period, 50 participants were prescribed intravitreal anti-VEGF agents and were given a time-temperature indicator. A total of 38 participants (24 [63.2%] female; median age, 79.3 [IQR, 74.3-86.3] years) returned for their IVT with an analyzable indicator. Of the 38 analyzable indicators, all showed temperature excursions above the threshold of 8 °C, including 26 (68.4%) for 12 to 48 hours and 11 (28.9%) for 48 hours or longer. Following anti-VEGF agent IVT, no participant experienced any ocular adverse effect that might be associated with severe intraocular inflammation.Conclusions and RelevanceIn this quality improvement study, temperature excursions of anti-VEGF agents above 8 °C for 48 hours or longer were recorded for 28.9% of participants during home storage. While it is unknown how these 38 participants relate to other patients worldwide who are required to obtain their own anti-VEGF agents, these findings suggest that patients need education about the importance of rigorous maintenance of cold storage of their anti-VEGF agents.","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"43 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142986887","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Temperature Excursion of Patient-Purchased Anti-VEGF Agents-A Cause for Concern? 患者购买的抗vegf药物的体温偏移——值得关注的原因?
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.6080
Jason Fan,Harry W Flynn,Nicolas A Yannuzzi
{"title":"Temperature Excursion of Patient-Purchased Anti-VEGF Agents-A Cause for Concern?","authors":"Jason Fan,Harry W Flynn,Nicolas A Yannuzzi","doi":"10.1001/jamaophthalmol.2024.6080","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.6080","url":null,"abstract":"","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"56 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142988590","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Fever and Floaters in a 56-Year-Old Woman 56岁妇女发烧及飞蚊症
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.5862
Kim Firn, Justin Youn, David Sierpina
{"title":"Fever and Floaters in a 56-Year-Old Woman","authors":"Kim Firn, Justin Youn, David Sierpina","doi":"10.1001/jamaophthalmol.2024.5862","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5862","url":null,"abstract":"A 56-year-old woman presents with fever, headache, nausea, and vomiting. Computed tomography scan of the head shows multiple coarse, dystrophic calcifications, and she reports floaters with normal vision. What would you do next?","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"37 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142986912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Myopia Control-Are We Getting Any Closer? 近视控制——我们离目标越来越近了吗?
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.6083
Lori Ann F Kehler,David K Wallace
{"title":"Myopia Control-Are We Getting Any Closer?","authors":"Lori Ann F Kehler,David K Wallace","doi":"10.1001/jamaophthalmol.2024.6083","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.6083","url":null,"abstract":"","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"25 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142988587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bilateral Mumps-Associated Retinitis in an Unvaccinated Male. 未接种疫苗男性双侧腮腺炎相关视网膜炎病例。
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-16 DOI: 10.1001/jamaophthalmol.2024.4885
Radhika Sriram,Padmamalini Mahendradas,Rohit Shetty
{"title":"Bilateral Mumps-Associated Retinitis in an Unvaccinated Male.","authors":"Radhika Sriram,Padmamalini Mahendradas,Rohit Shetty","doi":"10.1001/jamaophthalmol.2024.4885","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.4885","url":null,"abstract":"","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"23 1","pages":"e244885"},"PeriodicalIF":8.1,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142988588","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Efficacy and Safety of Different Atropine Regimens for the Treatment of Myopia in Children 不同阿托品方案治疗儿童近视的疗效和安全性
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-09 DOI: 10.1001/jamaophthalmol.2024.5703
James Loughman, Gareth Lingham, Ernest Kyei Nkansah, Emmanuel Kobia-Acquah, Daniel Ian Flitcroft
{"title":"Efficacy and Safety of Different Atropine Regimens for the Treatment of Myopia in Children","authors":"James Loughman, Gareth Lingham, Ernest Kyei Nkansah, Emmanuel Kobia-Acquah, Daniel Ian Flitcroft","doi":"10.1001/jamaophthalmol.2024.5703","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5703","url":null,"abstract":"ImportanceAdditional data are required regarding atropine treatment regimens for control of myopia progression.ObjectiveTo investigate the efficacy and safety of different atropine regimens for myopia in children.Design, Setting, and ParticipantsThis was a secondary analysis of the 3-year results of the 24-Month Myopia Outcome Study of Atropine in Children (MOSAIC) trial, called the MOSAIC2 trial. The MOSAIC trial was an investigator-led, double-masked, randomized clinical trial of different atropine concentrations and regimens. The MOSAIC2 study took place at the Centre for Eye Research Ireland, in Dublin, Ireland, and included children and adolescents with myopia from the MOSAIC trial. Data analysis was conducted from November 2023 to February 2024.InterventionsParticipants were randomly assigned to the following cohorts: group 1, nightly placebo for 2 years then 0.05% atropine eye drops for 1 year and group 2, nightly 0.01% atropine eye drops for 2 years then rerandomization to placebo nightly, tapering placebo, or tapering of 0.01% atropine eye drops for 1 year.Main Outcomes and MeasuresObserved changes in cycloplegic spherical equivalent refraction and axial length from month 24, or baseline, to month 36.ResultsA total of 199 children with myopia (mean [SD] age, 13.9 [2.4] years; 121 female [60.8%]) of the 250 children and adolescents from the MOSAIC trial were included in the MOSAIC2 trial analysis. Of 83 participants assigned to group 1, 66 (79.5%) reconsented to year 3, and 61 (73.5%) completed the trial. Of 167 participants assigned to group 2, 133 (79.6%) continued to year 3, and 121 (72.5%) completed the trial (0.01% atropine, then nightly placebo: n = 31 and n = 29 [93.5%]; 0.01% atropine, then tapering placebo: n = 29 and n = 25 [86.2%]; 0.01% atropine then tapering 0.01% atropine: n = 73 and n = 67 [91.8%], respectively). Compared with the group taking placebo then 0.05% atropine, the combined atropine then placebo groups had more spherical equivalent progression (adjusted difference, −0.13 diopters [D]; 95% CI, −0.22 to −0.04 D; <jats:italic>P</jats:italic> = .01) and axial elongation (adjusted difference, 0.06 mm; 95% CI, 0.02-0.09 mm; <jats:italic>P</jats:italic> = .008), and the group taking 0.01% atropine then tapering 0.01% atropine had more axial elongation (adjusted difference, 0.04 mm; 95% CI, 0.009-0.07 mm; <jats:italic>P</jats:italic> = .04). In the group taking placebo then 0.05% atropine, 15% (n = 10) and 8% (n = 5) reported blurred near vision and photophobia, respectively, during year 3, compared with 3% (n = 2) and 0%, respectively, in the group taking 0.01% atropine then tapering 0.01% atropine, and no reports in both placebo groups.Conclusions and RelevanceDespite more adverse events, participants using 0.05% atropine during year 3 had no differences in treatment completion rates and exhibited 0.13-D less myopia progression and 0.06-mm less axial elongation, compared with participants using placebo, supporting cons","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"15 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142939872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy Bietti晶体营养不良基因治疗的安全性和视力结果
IF 8.1 1区 医学
JAMA ophthalmology Pub Date : 2025-01-09 DOI: 10.1001/jamaophthalmol.2024.5619
Xiuju Chen, Xiao Liu, Shihe Cui, Gang Wang, Yiting Liu, Guang Qu, Lixin Jiang, Yong Liu, Xiaoxin Li
{"title":"Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy","authors":"Xiuju Chen, Xiao Liu, Shihe Cui, Gang Wang, Yiting Liu, Guang Qu, Lixin Jiang, Yong Liu, Xiaoxin Li","doi":"10.1001/jamaophthalmol.2024.5619","DOIUrl":"https://doi.org/10.1001/jamaophthalmol.2024.5619","url":null,"abstract":"ImportanceBietti crystalline dystrophy (BCD) is a severe genetic retinopathy caused by variants in the <jats:italic>CYP4V2</jats:italic> gene. Currently, there is no approved treatment for BCD.ObjectiveTo evaluate safety and vision outcomes following gene therapy with adeno-associated virus (AAV) encoding CYP4V2 (rAAV-hCYP4V2, NGGT001 [Next Generation Gene Therapeutics]).Design, Setting, and ParticipantsThis open-label, dose-escalation nonrandomized clinical trial was conducted from February 2023 to May 2024 at 2 study sites in China. Patients with genetically confirmed biallelic disease-linked <jats:italic>CYP4V2</jats:italic> variants received subretinal injections of rAAV2-hCYP4V2 at 1 of 2 dosage levels and were followed up for 12 months.InterventionA single unilateral injection of 1.5 × 10<jats:sup>11</jats:sup> or 3.0 × 10<jats:sup>11</jats:sup> total vector genomes of recombinant AAV-hCYP4V2 in the worse eye, based on visual acuity letter score.Main Outcomes and MeasuresThe primary outcome was safety, assessed by clinical examination of ocular inflammation and evaluated by routine clinical chemistry and immunogenicity testing. Secondary outcomes were changes in visual function from baseline in best-corrected visual acuity (BCVA), microperimetry, and contrast sensitivity 12 months after treatment.ResultsAmong 12 patients with BCD (6 patients per dose group), mean (SD) patient age was 40.5 (7.1) years, and 5 patients (42%) were female. No severe adverse events related to the treatment were observed. However, mild intraocular inflammation was noted in 1 participant. The median (IQR) baseline BCVA letter score for the study eye was 34 (10-53), equivalent to 20/200 Snellen, while the nonstudy eye had a median (IQR) BCVA of 60 (40-67), equivalent to approximately 20/63 Snellen. At 12 months, the study eye improved by a mean (SD) letter score of 13.9 (13.1) compared with 6.3 (7.4) in the nonstudy eye. The 12-month median (IQR) BCVA for the study eye was 53 (37-64) (equivalent to approximately 20/80 Snellen) and 62 (42-70) (approximately 20/50 Snellen) for the nonstudy eye.Conclusions and RelevanceThis open-label, exploratory nonrandomized clinical trial identified no serious safety concerns related to gene therapy over 12 months’ follow-up among patients with BCD. While improvement in BCVA was noted, the magnitude was within test-retest values typically noted in eyes with very low levels of visual acuity, and BCVA improvement in both the study and nonstudy eyes could be related to a learning effect, with greater improvement in the study eye possibly related to study eyes’ being the worse-seeing eye.Trial RegistrationClinicalTrials.gov Identifier: <jats:ext-link xmlns:xlink=\"http://www.w3.org/1999/xlink\" ext-link-type=\"uri\" xlink:href=\"https://www.clinicaltrials.gov/study/NCT06302608?cond=NCT06302608&amp;amp;amp;rank=1\">NCT06302608</jats:ext-link>","PeriodicalId":14518,"journal":{"name":"JAMA ophthalmology","volume":"20 1","pages":""},"PeriodicalIF":8.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142939873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信