American Journal of Ophthalmology最新文献

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Real-World Applicability of Anti-VEGF Trial Criteria for Diabetic Macular Edema. 抗vegf试验标准对糖尿病黄斑水肿的实际适用性
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-04 DOI: 10.1016/j.ajo.2026.09.002
Raj Kundu, Nathan Goturi, Michael Green, Vasiliki Poulaki, Nicole H Siegel, Manju L Subramanian, Steven Ness
{"title":"Real-World Applicability of Anti-VEGF Trial Criteria for Diabetic Macular Edema.","authors":"Raj Kundu, Nathan Goturi, Michael Green, Vasiliki Poulaki, Nicole H Siegel, Manju L Subramanian, Steven Ness","doi":"10.1016/j.ajo.2026.09.002","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.09.002","url":null,"abstract":"<p><strong>Objective: </strong>To characterize the real-world eligibility based on systemic medical criteria of patients receiving anti-VEGF injections for DME, including potential rates of trial exclusion and associated patient demographics.</p><p><strong>Design: </strong>Cross-sectional study.</p><p><strong>Subjects: </strong>Patients who received at least one anti-VEGF injection for treatment of DME with recorded pre-injection blood pressure (BP) and hemoglobin A1c (A1c) measurements.</p><p><strong>Methods: </strong>Data was sourced from United States healthcare organizations enrolled in the TriNetX database. Trial selection criteria were sourced from ClinicalTrials.gov.</p><p><strong>Main outcome measures: </strong>The primary outcome was the overall rate of trial exclusion across all participants. Secondary outcomes comprised the rate of exclusion based on patient demographics.</p><p><strong>Results: </strong>The most inclusionary (\"liberal\") criteria allowed for participation with BP ≤180/100, A1c ≤12.0%, no history of myocardial infarction (MI) or cerebrovascular accident (CVA) within 3 months prior to first injection, and no history of end stage renal disease (ESRD). The most exclusionary (\"stringent\") criteria required BP ≤160/95, A1c ≤10.0%, no history of MI or CVA within 6 months prior to first injection, and no history of ESRD. Application of the liberal and stringent criteria excluded 25.7% and 59.1% of real-world patients, respectively. Compared to White individuals, Black individuals were significantly more likely to be excluded by the liberal criteria (Odds ratio [OR] 1.97; 95% CI, 1.57 to 2.46; P<sub>adj</sub> < .001) as well as stringent criteria (OR, 2.29; 95% CI, 1.82 to 2.87; P<sub>adj</sub> < .001). Individuals of Other race(s) were significantly more likely to be excluded by the liberal criteria (OR, 1.94; 95% CI, 1.33 to 2.84; P<sub>adj</sub> = .004).</p><p><strong>Conclusions: </strong>Systemic medical selection criteria for clinical trials of anti-VEGF therapy for DME exclude a significant portion of real-world patients, with members of racial minority groups being disproportionately affected.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148890826","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
Changing Trends in the Microbiological spectrum and clinical presentation of Postcataract Endophthalmitis over a 23-year period. 23年来白内障后眼内炎的微生物谱和临床表现的变化趋势。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-03 DOI: 10.1016/j.ajo.2026.08.042
André Baudere, Max Maurin, Corinne Dot, Benjamin Matagrin, Mathilde Gallice, Karine Palombi, Christophe Chiquet
{"title":"Changing Trends in the Microbiological spectrum and clinical presentation of Postcataract Endophthalmitis over a 23-year period.","authors":"André Baudere, Max Maurin, Corinne Dot, Benjamin Matagrin, Mathilde Gallice, Karine Palombi, Christophe Chiquet","doi":"10.1016/j.ajo.2026.08.042","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.042","url":null,"abstract":"<p><strong>Objective: </strong>To characterize temporal changes in clinical severity, bacterial spectrum, and antimicrobial susceptibility in acute postcataract endophthalmitis over 23 years.</p><p><strong>Design: </strong>Retrospective comparative cohort study.</p><p><strong>Subjects, participants, and/or controls: </strong>This study included 234 eyes of 234 patients presenting within 6 weeks after cataract surgery at 2 French tertiary referral centers. The historical cohort comprised 126 eyes managed from 2002 through 2007, and the recent cohort comprised 108 eyes managed from 2016 through 2025.</p><p><strong>Methods, intervention, or testing: </strong>Microbiological analyses included conventional cultures and 16S polymerase chain reaction on aqueous and/or vitreous samples. Antimicrobial susceptibility testing was performed on bacterial isolates. Baseline clinical severity, pathogen distribution, and resistance profiles were compared between cohorts.</p><p><strong>Main outcome measures: </strong>Pathogen distribution, cefuroxime susceptibility, methicillin resistance among staphylococci, and markers of clinical severity at presentation.</p><p><strong>Results: </strong>Bacterial identification was obtained in 85 of 126 eyes (67.5%) in the historical cohort and 63 of 108 eyes (58.3%) in the recent cohort. Coagulase-negative staphylococci decreased from 38% to 25% of isolates, whereas Enterococcus faecalis increased from 2% to 10%; Gram-negative bacteria increased from 3.1% to 7.3%. Cefuroxime resistance increased from 26.0% (13/50) to 66.1% (adjusted OR, 3.75; 95% CI, 1.59-8.82; P = .003) of tested isolates. Among staphylococci, methicillin resistance increased from 30.3% to 75.0% (adjusted OR, 6.75; 95% CI, 1.99-22.83; P = .002), and multidrug-resistant phenotypes increased from 3.0% to 37.5% (P = .003). Eyes in the recent cohort had milder clinical presentation, with visual acuity of 20/400 or better in 29.0% versus 11.2%, visible fundus in 24.1% versus 9.6%, and pars plana vitrectomy performed in 51.9% versus 63.5%. Interpretation is limited by the retrospective design and incomplete susceptibility data.</p><p><strong>Conclusions: </strong>This retrospective comparative cohort study of 234 cases across two periods spanning 23 years was associated with a marked shift in the bacterial spectrum of acute postcataract endophthalmitis, with fewer coagulase-negative staphylococci and more Enterococcus faecalis isolates, alongside increased cefuroxime and methicillin resistance. Continued microbiological surveillance is warranted to guide empiric therapy and future prophylactic strategies.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148886139","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
USH2A-Associated Retinitis Pigmentosa in Koreans: Molecular Genetics and Clinical Characteristics. 韩国人ush2a相关色素性视网膜炎:分子遗传学和临床特征。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-03 DOI: 10.1016/j.ajo.2026.08.044
Sungsoon Hwang, Sohee Jeon, Je Moon Yoon, Youn-Ji Hong, Ja-Hyun Jang, Sang Jin Kim
{"title":"USH2A-Associated Retinitis Pigmentosa in Koreans: Molecular Genetics and Clinical Characteristics.","authors":"Sungsoon Hwang, Sohee Jeon, Je Moon Yoon, Youn-Ji Hong, Ja-Hyun Jang, Sang Jin Kim","doi":"10.1016/j.ajo.2026.08.044","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.044","url":null,"abstract":"<p><strong>Purpose: </strong>To describe the molecular genetic spectrum and age-related clinical features of USH2A-associated retinitis pigmentosa in Korean patients.</p><p><strong>Design: </strong>Retrospective cohort study.</p><p><strong>Methods: </strong>A total of 182 Korean patients with biallelic pathogenic or likely pathogenic USH2A variants were included. Patients were classified as Usher syndrome (USH) or non-syndromic retinitis pigmentosa (NSRP), and stratified by the number of truncating USH2A variants (0, 1, or 2). Best-corrected visual acuity (BCVA), Goldmann visual field, ellipsoid zone (EZ) band length on optical coherence tomography, and ultra-widefield fundus and autofluorescence imaging were assessed. Age-related changes were modeled using restricted cubic splines with generalized estimating equations.</p><p><strong>Results: </strong>The cohort comprised 56 patients with USH (30.8%) and 126 with NSRP (69.2%); mean age was 44.3 ± 12.7 years. Among 364 pathogenic or likely pathogenic alleles, c.2802T>G (p.Cys934Trp) was the most common (23.6%), followed by c.8559-2A>G (12.1%). The European hotspots c.2299delG and c.2276G>T were not identified. Missense variants accounted for 53.8% of alleles. At least one c.2802T>G allele was present in 82 patients (45.1%), including 4 homozygotes. Both Usher syndrome and an increasing truncating variant burden were associated with worse age-adjusted patterns of visual acuity, visual field, and EZ band length.</p><p><strong>Conclusion: </strong>USH2A-associated retinitis pigmentosa in Korean patients follows an East Asian variant spectrum that is distinct from European populations. Approximately half of patients carried at least one c.2802T>G allele, supporting inclusion of East Asian patients in emerging exon 13-targeted therapies.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148886208","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
LYNX-1: A Phase 3 Study of 0.75% Phentolamine Ophthalmic Solution in Subjects with Dim Light Vision Disturbances. LYNX-1: 0.75%酚妥拉明眼液治疗弱光视力障碍的3期研究。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-02 DOI: 10.1016/j.ajo.2026.08.045
Jay S Pepose, Stephen Montaquila, David Wirta, Mitchell Brigell, Konstantinos Charizanis
{"title":"LYNX-1: A Phase 3 Study of 0.75% Phentolamine Ophthalmic Solution in Subjects with Dim Light Vision Disturbances.","authors":"Jay S Pepose, Stephen Montaquila, David Wirta, Mitchell Brigell, Konstantinos Charizanis","doi":"10.1016/j.ajo.2026.08.045","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.045","url":null,"abstract":"<p><strong>Objective or purpose: </strong>To evaluate the efficacy and safety of 0.75% phentolamine ophthalmic solution (POS) for the treatment of dim light disturbances (DLD) in subjects with reduced mesopic low-contrast vision, including post-keratorefractive subjects.</p><p><strong>Design: </strong>Phase 3, multicenter, double-masked, randomized, placebo-controlled trial SUBJECTS, PARTICIPANTS, AND/OR CONTROLS: 145 subjects with self-reported symptoms of DLD, mesopic pupil diameter of ≥5 mm, and 30 ETDRS letters (equivalent to 20/63 Snellen) or worse mesopic low-contrast best-corrected distance visual acuity (mLCVA) in the study eye, were randomized 1:1 to receive POS or placebo. The largest subset (25) of the enrolled subjects was status post-keratorefractive surgery.</p><p><strong>Methods, intervention, or testing: </strong>One drop of POS or placebo was self-administered in each eye once nightly for 14 days.</p><p><strong>Main outcome measures: </strong>Assessments included visual acuity testing, pupil diameter measurement, wavefront aberrometry, intraocular pressure (IOP), slit-lamp biomicroscopy, and a questionnaire assessing visual symptoms.</p><p><strong>Results: </strong>POS-treated subjects demonstrated significant improvement in mLCVA at Day 8 (13% vs. 3% placebo; p<0.05) and Day 15 (21% vs. 3% placebo; p<0.01). Subjects receiving POS reported significant reduction in overall severity of DLD (on a scale of 1-7), and photic phenomena including glare, halos and starburst (on a scale of 0-3) at Day 8 and 15 (p<0.01). There was no significant difference between arms in conjunctival hyperemia or vital signs. Results from post-hoc analysis of post-keratorefractive surgery patients also showed significant improvement in mLCVA at Day 8 and 15 and significant improvement in patient reported outcome measures, such as overall severity of DLD.</p><p><strong>Conclusions: </strong>POS significantly improved mesopic low-contrast best-corrected distance visual acuity in subjects with reduced mesopic vision and dim light disturbances of varying etiologies, including post-keratorefractive patients. Treatment-emergent adverse events were predominantly mild and transient. These findings suggest POS may warrant further clinical investigation as a potential pharmacologic approach for patients experiencing dim light disturbances associated with reduced mesopic low-contrast visual acuity and photic symptoms.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148878923","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
Null studies in ophthalmology journals: a large-scale analysis of PubMed indexed research. 眼科学期刊中的空白研究:PubMed索引研究的大规模分析。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-01 DOI: 10.1016/j.ajo.2026.08.040
Idan Hecht, Ruti Sella, Piotr Kanclerz, Vera Sorin, Raimo Tuuminen
{"title":"Null studies in ophthalmology journals: a large-scale analysis of PubMed indexed research.","authors":"Idan Hecht, Ruti Sella, Piotr Kanclerz, Vera Sorin, Raimo Tuuminen","doi":"10.1016/j.ajo.2026.08.040","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.040","url":null,"abstract":"<p><strong>Purpose: </strong>Null studies, in which the primary outcome is not statistically significant, are essential for unbiased evidence-based medicine and help reveal selective dissemination of research. We estimated the prevalence of null studies and characterized differences between null and positive studies in ophthalmology journals.</p><p><strong>Design: </strong>Retrospective analysis of original studies published from January 1, 2014, through December 31, 2025.</p><p><strong>Subjects: </strong>Original studies published in 90 PubMed-indexed journals listed in the Ophthalmology category of the 2025 Journal Citation Reports.</p><p><strong>Methods: </strong>Study characteristics were derived using rule-based processing, except classification of the main study result as null versus positive, which used a large language model pipeline (gpt-4.1) applied to abstracts. Large language model performance was evaluated against a prespecified manual validation set of 500 abstracts, achieving kappa 0.87 (prespecified threshold 0.75).</p><p><strong>Main outcome measures: </strong>Prevalence and differences in study characteristics between null and positive studies.</p><p><strong>Results: </strong>A total of 93,963 studies were included, of which 19,231 (20.5%) were null studies. The proportion of null studies declined from 25.6% in 2014 to 15.1% in 2025 (p <0.001). Positive studies were more often funded than null studies (68.4% vs 58.0%; risk difference 95% CI 9.6 to 11.2; p<0.001) and were more often prospective in design (17.5% vs 16.0%; risk difference 95% CI 0.9 to 2.0; p<0.001), whereas randomized design was similarly common between groups (6.5% vs 6.5%, p=0.867). Positive studies were also more often drug-focused (13.6% vs 11.8%), device-focused (9.3% vs 6.9%), or surgery-focused (13.4% vs 9.3%) (all p<0.001). Null studies were published in journals with lower mean impact factor (2.59±1.62 vs 2.73±1.69; mean difference 95% CI 0.11 to 0.17; p<0.001), and worse mean journal rank (40.11 ± 22.6 vs 37.80 ± 22.2; mean difference 95% CI -2.67 to -1.95; p<0.001) compared to positive studies.</p><p><strong>Conclusions: </strong>In this large-scale analysis of ophthalmology journals, one in five studies had a null primary outcome. Overall, null studies became less common over time and were more often published in lower-impact, lower-ranked journals and were more likely to be unfunded. These findings suggest systematic differences in the characteristics and dissemination of null versus positive studies in ophthalmology.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148872601","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
Central obesity in women and high overall body fat in men are associated with higher odds of dry eye disease 女性的中枢性肥胖和男性的高整体脂肪与干眼病的高发病率有关
IF 4.2 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-01 DOI: 10.1016/j.ajo.2026.08.046
Mengyao Li,Li Li,Jingting Liu,Jiayi Feng,Yan Lee,Bowen Wang,Jin Yuan
{"title":"Central obesity in women and high overall body fat in men are associated with higher odds of dry eye disease","authors":"Mengyao Li,Li Li,Jingting Liu,Jiayi Feng,Yan Lee,Bowen Wang,Jin Yuan","doi":"10.1016/j.ajo.2026.08.046","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.046","url":null,"abstract":"","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":"2 1","pages":""},"PeriodicalIF":4.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148893880","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
Hypertrophic Pachymeningitis Revealing Breast Cancer. 增生性厚性脑膜炎揭示乳腺癌。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-01 DOI: 10.1016/j.ajo.2026.08.041
Norimitsu Fujii, Ken Fukuda, Kenji Yamashiro
{"title":"Hypertrophic Pachymeningitis Revealing Breast Cancer.","authors":"Norimitsu Fujii, Ken Fukuda, Kenji Yamashiro","doi":"10.1016/j.ajo.2026.08.041","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.041","url":null,"abstract":"","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148872618","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
Cumulative Atopic Disease Burden and Keratoconus: Cross-Sectional and Longitudinal Associations in the All of Us Research Program 累积性特应性疾病负担与圆锥角膜:我们所有人研究计划的横断面和纵向关联
IF 4.2 1区 医学
American Journal of Ophthalmology Pub Date : 2026-09-01 DOI: 10.1016/j.ajo.2026.09.001
Fahad R. Butt,Thanansayan Dhivagaran,Tasha Miller,Jawad Muayad,Salem Abu Al-Burak,Fatima Abid,Wissam Nassrallah,Brendan Tao,Yusuf Ahmed,Clara C. Chan
{"title":"Cumulative Atopic Disease Burden and Keratoconus: Cross-Sectional and Longitudinal Associations in the All of Us Research Program","authors":"Fahad R. Butt,Thanansayan Dhivagaran,Tasha Miller,Jawad Muayad,Salem Abu Al-Burak,Fatima Abid,Wissam Nassrallah,Brendan Tao,Yusuf Ahmed,Clara C. Chan","doi":"10.1016/j.ajo.2026.09.001","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.09.001","url":null,"abstract":"","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":"9 1","pages":""},"PeriodicalIF":4.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148893879","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
Voretigene Neparvovec (Luxturna) for Biallelic RPE65-Associated Retinal Dystrophy: Systematic Review. Voretigene Neparvovec (Luxturna)治疗双等位基因rpe65相关视网膜营养不良:系统综述。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-08-31 DOI: 10.1016/j.ajo.2026.08.043
Abu Bakar Butt, Michele Zaman, Andrew Mihalache, Sara KamaliZonouzi, Parsa Babaei Zadeh, Yaksh R Shah, Marko M Popovic, Peter J Kertes, Brian G Ballios, Ajoy Vincent, Elise Heon, Rajeev H Muni, Radha P Kohly
{"title":"Voretigene Neparvovec (Luxturna) for Biallelic RPE65-Associated Retinal Dystrophy: Systematic Review.","authors":"Abu Bakar Butt, Michele Zaman, Andrew Mihalache, Sara KamaliZonouzi, Parsa Babaei Zadeh, Yaksh R Shah, Marko M Popovic, Peter J Kertes, Brian G Ballios, Ajoy Vincent, Elise Heon, Rajeev H Muni, Radha P Kohly","doi":"10.1016/j.ajo.2026.08.043","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.043","url":null,"abstract":"<p><strong>Topic: </strong>Biallelic RPE65-associated inherited retinal disorders (IRDs) cause progressive vision loss, impaired light sensitivity, and reduced mobility. Voretigene neparvovec-rzyl (LUXTURNA), the first FDA-approved gene therapy for this condition, is a disease-modifying option whose clinical benefits and harms warrant synthesis.</p><p><strong>Clinical relevance: </strong>Patients with biallelic RPE65-associated IRDs often have severe functional impairment despite variable BCVA changes. Understanding treatment effects on BCVA, light sensitivity, VF, and adverse events is essential for referral timing, counseling, and monitoring.</p><p><strong>Methods: </strong>Ovid MEDLINE, Embase, and the Cochrane Library were searched from January 2000 through October 2025 for randomized trials and observational studies evaluating voretigene neparvovec-rzyl in RPE65-associated IRDs. The primary outcome was BCVA; secondary outcomes included full-field stimulus threshold (FST), visual field (VF), and adverse events including chorioretinal atrophy and central retinal thickness (CRT) change. Findings were synthesized narratively for the systematic review, with random-effects meta-analyses for perifoveal atrophy incidence and CRT change where data allowed.</p><p><strong>Results: </strong>Twenty-eight studies met inclusion criteria, encompassing 376 individuals and 673 treated eyes. BCVA outcomes were variable and generally modest, with somewhat greater gains in pediatric than adult cohorts. Light sensitivity improved consistently on FST, evident by 1 month and sustained through 6 months in the largest cohort (mean change -18.24 dB); one study reported greater 12-month gains in children (13 dB) than adults (8 dB). VF improved substantially in the pivotal trial cohort (92% mean increase at 1 year, sustained to 3-4 years), with more equivocal findings in real-world pediatric cohorts. Chorioretinal atrophy and intraocular inflammation were the most frequently reported adverse events. Pooled analysis of 3 studies estimated 75% of treated eyes (95% CI, 57%-89%) developed new or progressive perifoveal atrophy; CRT changes ranged from a mean increase of 8.3 µm to a decrease of 27.5 µm. Severe complications such as retinal tears and detachment were infrequent.</p><p><strong>Conclusion: </strong>Voretigene neparvovec-rzyl is associated with improvements in light sensitivity and VF in pediatric patients treated before advanced degeneration, while BCVA gains are variable and usually modest. Chorioretinal atrophy, including perifoveal atrophy in a majority of treated eyes, is a common structural finding warranting monitoring alongside functional benefits.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148863047","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
Reply to Comment on Axial Length, Refraction, and the Language of Ophthalmology. 回复关于眼轴长度、屈光和眼科学语言的评论。
IF 4.7 1区 医学
American Journal of Ophthalmology Pub Date : 2026-08-30 DOI: 10.1016/j.ajo.2026.08.038
Ta Chen Peter Chang, Charles F Bartlett, Arif O Khan
{"title":"Reply to Comment on Axial Length, Refraction, and the Language of Ophthalmology.","authors":"Ta Chen Peter Chang, Charles F Bartlett, Arif O Khan","doi":"10.1016/j.ajo.2026.08.038","DOIUrl":"https://doi.org/10.1016/j.ajo.2026.08.038","url":null,"abstract":"","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.7,"publicationDate":"2026-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148860419","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
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