American Journal of Ophthalmology最新文献

{"title":"The role of Intravitreal chemotherapy as an adjunctive treatment for retinoblastoma: a systematic review and single-arm meta-analysis.","authors":"Andres Bravo-Gonzalez, Pablo Domínguez-Ruiz, María González, Sara Hira, Claudia Avilés-Covarrubias, Carlos Eduardo de Menezes E Souza-Filho, Mariana Tosato Zinher, Carol L Shields","doi":"10.1016/j.ajo.2024.12.012","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.12.012","url":null,"abstract":"<p><strong>Topic: </strong>evaluation of clinical outcomes of patients with retinoblastoma treated with intravitreal chemotherapy (IvitC).</p><p><strong>Design: </strong>Systematic review and single-arm meta-analysis CLINICAL RELEVANCE: Clinical outcomes with IVitC vary across reports according to patient characteristics and concomitant treatment modalities, mainly intravenous chemotherapy (IVC) and intra-arterial chemotherapy (IAC). There are currently no large clinical trials or meta-analyses focusing on the topic.</p><p><strong>Methods: </strong>A systematic search was conducted in MEDLINE, EMBASE and Cochrane. All articles reporting use of IVitC for RB and safety or efficacy outcomes were included regardless of publication date. Studies with fewer than 10 eyes were excluded. Enucleation rates (ER) were calculated using proportions and 95% confidence intervals (CIs). The analysis was performed using the Random Effects model in R Studio.</p><p><strong>Results: </strong>25 studies comprising 1082 eyes met inclusion criteria. Melphalan was exclusively used in 687 eyes (63.49%), 104 eyes received topotecan exclusively (9.61%), and the remaining 291 (26.90%) used a combination. General ER was 24.70% (95% CI 19.20-31.18%). Subgroup analysis showed an ER of 27.76% (95% CI 19.05-38.55%) for melphalan, 14.23% (95% CI 5.61-21.66%) for topotecan, and 23.82% (95% CI 11.95-41.87%) for combination therapy (p<0.05). It also revealed an ER of 21.54% (95% CI 15.57-29.01%) for studies that implemented IAC+IVitC versus 35.50% (95% CI 20.73-53.66%) for those who used IVC+IVitC (p<0.05). Pigmentary retinopathy rate was 36.56% (95% CI 24.61-50.44%) in subjects treated with melphalan and 2.42% (95% CI 0.70-8.01%) for those receiving topotecan (p<0.05). Other adverse events were cataract (17.76%) followed by vitreous hemorrhage (12.10%) and retinal detachment (5.62%). All studies, except one, were determined to have a serious risk of bias.</p><p><strong>Conclusion: </strong>IVitC represents an effective strategy for retinoblastoma, especially when administered after IAC; however, melphalan retinal toxicity still poses a challenge. Results with topotecan are promising but scarce. Comparing both drugs is needed to define the best treatment strategy. This study is limited by the lack of large, randomized studies on this subject.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142862934","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}

{"title":"Global Trends and Projections for Avoidable Visual Impairment among Working-Age Individuals: A Population-Based Study.: Avoidable Visual Impairment in Working-Age.","authors":"Ying Zhang, Jianqi Chen, Lirong Liao, Xiaohu Ding","doi":"10.1016/j.ajo.2024.12.003","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.12.003","url":null,"abstract":"<p><strong>Purpose: </strong>Visual impairment (VI) in working-age individuals significantly impacts public health and the economy. However, the avoidable causes, defined as cataract and refraction disorders, have not been extensively investigated. This study aims to quantify global trends, inequalities, and projections for avoidable VI among this demographic.</p><p><strong>Design: </strong>Retrospective, observational, population-based trend study.</p><p><strong>Methods: </strong>We derived data on avoidable VI prevalence and population size data in working age from the Global Burden of Disease (GBD) 2021 study. We employed the joinpoint regression analysis to assess trends from 1990 to 2021 by age, sex, sociodemographic index (SDI), type, and severity at the global, regional, and national levels. Cross-country inequalities were evaluated using the slope index of inequality and the health inequality concentration index. Subsequently, we performed Bayesian age-period-cohort modeling to estimate the avoidable BVL burden in working age by 2040.</p><p><strong>Results: </strong>The prevalence of avoidable VI in working age decreased overall from 1990 to 2021, driven primarily by reductions in refraction disorders, with an average annual percentage change (AAPC) of -0.15 (95% confidence interval [CI], -0.18 to -0.12; P<0.001). We observed significant declines in severe vision loss and blindness, while moderate vision loss remained stable. Females exhibited a heavier burden of avoidable VI, but showed slower improvement compared to males. Socioeconomic disparities persisted, with lower socioeconomic index (SDI) regions bearing a disproportionate burden, whereas the high SDI region showed an unfavorable increasing trend. From 1990 to 2021, the inequality slope index increased from 574.45 (95% CI, 914.95 to 233.95) to 652.27 (95% CI, 932.95 to 371.58), while the health inequality concentration index improved from -0.21 (95% CI, -0.26 to -0.17) to -0.17 (95% CI, -0.2 to -0.14). We project a continued decline in the global prevalence of avoidable working-age VI, but a 23.4% increase in avoidable VI cases by 2040, reaching approximately 146 million.</p><p><strong>Conclusions: </strong>Despite overall declines in avoidable VI among working-age individuals, significant improvement opportunities and disparities persist. The anticipated increase in avoidable VI cases necessitates integrated eye health strategies, substantial investment in eye care services to enhance accessibility and affordability, and fair employment policies for the visually impaired.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142821730","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}

{"title":"Pentosan Polysulfate Maculopathy: Final Outcomes from a 4-Year Prospective Study of Disease Progression after Drug Cessation.","authors":"Brian P Hall, Sakshi Shiromani, Emily H Jung, Riley J Lyons, Judith Tribe, Nieraj Jain","doi":"10.1016/j.ajo.2024.12.004","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.12.004","url":null,"abstract":"<p><strong>Purpose: </strong>To report the long-term disease course of pentosan polysulfate (PPS) maculopathy following drug cessation.</p><p><strong>Design: </strong>Single-institution, prospective case series.</p><p><strong>Methods: </strong>23 eyes of 12 participants seen at the Emory Eye Center with a diagnosis of PPS maculopathy were included in our study. Participants were enrolled between December 1, 2018, and December 1, 2019, and data were collected annually for four years.</p><p><strong>Main outcomes and measures: </strong>Changes in visual function and structure were the primary outcomes measured. Visual function was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA), ETDRS low-luminance visual acuity (LLVA), Minnesota low-vision reading (MNREAD) performance, contrast sensitivity, mesopic and scotopic microperimetry, and dark adaptometry. Patient reported outcomes were assessed with the National Eye Institute Visual Function Questionnaire (NEI-VFQ-39) and Low Luminance Questionnaire (LLQ). Structural outcomes included the presence of complete retinal pigment epithelium and outer retinal atrophy (cRORA), atrophic lesion size (in mm²), macular central subfield thickness (CST), and subfoveal choroidal thickness (SFCT).</p><p><strong>Results: </strong>Of the 12 participants, 11 (91.7%) were female, with a median age at enrollment of 58 years. The median ETDRS BCVA letter score at baseline was 83, with a median change of -5 letters over 4 years (P = 0.005). The median 4-year change in mesopic microperimetry average threshold and percent reduced threshold was -5.4 dB (P = 0.003) and 48.6% (P = 0.004), respectively. MNREAD performance (assessed at 2 and 4 years) declined across all measures, with a median maximum reading speed change of -21 words per minute (P = 0.007). NEI-VFQ-39 and LLQ composite scores significantly decreased over 4 years. At baseline, 9 eyes (39%) had macular cRORA. By the study's end, 5 of the remaining eyes (35.7%) developed new-onset cRORA. The median linearized growth rate of atrophic lesions was 0.23 mm/year. The median 4-year change of CST and SFCT was -7.0 μm (P = 0.055) and -22.0 μm (P = 0.610), respectively.</p><p><strong>Conclusion: </strong>This prospective study demonstrates continued progression of broad-ranging functional and structural deficits in PPS maculopathy long after drug cessation. These findings should inform PPS prescribing patterns, patient counseling, and disease monitoring strategies.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816878","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}

{"title":"Effect of prophylactic topical antibiotics on rates of infectious complications after strabismus surgery: a large database study.","authors":"Natalia M Binczyk, Euna B Koo, Scott R Lambert","doi":"10.1016/j.ajo.2024.12.005","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.12.005","url":null,"abstract":"<p><strong>Objective: </strong>Not all strabismus surgeons use prophylactic topical antibiotics postoperatively because they may be poorly tolerated, costly, and may increase antimicrobial resistance. The purpose of the study is to assess the effect of postoperative topical antibiotics on the rate of surgical site infections following strabismus surgery.</p><p><strong>Design: </strong>Retrospective clinical cohort study.</p><p><strong>Participants: </strong>Patients who underwent strabismus surgery in the past 20 years. Patients who were or were not prescribed postoperative topical antibiotics comprised the study and control groups, respectively.</p><p><strong>Methods: </strong>This study was conducted using data from TriNetX, an international electronic health-record registry containing data for over 130 million patients. Strabismus surgery patients were identified using CPT codes. The prescription of eye topical antibiotics and the frequency of surgical site infections were identified by RxNorm and ICD-10 codes, respectively. Analysis was conducted using the measures of association feature of TriNetX. Patient demographics such as age, sex, and race were recorded based on their presence in the electronic records system.</p><p><strong>Main outcome measure: </strong>Development of endophthalmitis, orbital and preseptal cellulitis.</p><p><strong>Results: </strong>There were 84,052 patients who underwent strabismus surgery. 38,484 (46%) patients received one or more postoperative topical antibiotics. About 70% of the patients were White and 10% Black. There was a slight female preponderance (51%). Endophthalmitis developed in 16 patients (0.019%), orbital cellulitis in 24 patients (0.029%) and preseptal cellulitis in 55 patients (0.065%). There was no difference in the rates of endophthalmitis, orbital, and preseptal cellulitis between the groups (p values 0.855, 0.684, 0.925 respectively).</p><p><strong>Conclusions: </strong>The prescription of prophylactic topical antibiotics did not decrease rates of surgical site infections following strabismus surgery. Administering postoperative topical antibiotics can be distressing to children, costly, and may contribute to antimicrobial resistance. Given no clear advantage and potential disadvantages, the use of postoperative topical antibiotics following strabismus surgery may not be beneficial.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816822","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}

{"title":"Subretinal Gene Therapy for Treatment of Retinal and Choroidal Vascular Diseases.","authors":"Arshad M Khanani, Aamir A Aziz, Zoha A Khanani, Hannah Khan, Ohidul Mojumder, Humza Sulahria, Ibrahim Khanani, Huma Khan, Greggory M Gahn, Kapil Mishra","doi":"10.1016/j.ajo.2024.12.002","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.12.002","url":null,"abstract":"<p><strong>Objective: </strong>This review article discusses investigational subretinal gene therapies for retinal vascular diseases, including AVA-101, an adeno-associated viral (AAV) 2 vector expressing soluble vascular endothelial growth factor (VEGF) receptor 1, ABBV-RGX-314, an AAV8 vector expressing an anti-VEGF-A antibody fragment, and EXG102-031, an AAV8 vector expressing a recombinant protein that blocks VEGF family members and angiopoietin 2.</p><p><strong>Design: </strong>Review article CONCLUSION: Subretinal injection is a commonly used delivery route for investigational gene therapy agents which is theorized to provide relative immune privilege, thereby reducing the risk of inflammation, while providing high transgene expression in photoreceptors and retinal pigment epithelium. Subretinal injection of AVA-101 demonstrated safety and tolerability in Phase I and IIa trials, but failed to maintain visual acuity and control exudation. In contrast, subretinal injection of some doses of ABBV-RGX-314 have shown evidence of controlling exudation and the maintaining vision, as well as safety and tolerability, leading to two ongoing pivotal trials comparing subretinal delivery of two different doses of ABBV-RGX-314 versus intravitreal injections of 0.5mg ranibizumab or 2mg aflibercept. These preliminary results are an encouraging and welcome development in the search for efficacious, long-duration treatments for retinal vascular diseases.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142799128","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}

{"title":"Clinical Classification and Management Outcomes of Anterior Segment Dominated Persistent Fetal Vasculature: the CCPMOH report.","authors":"Hui Chen, Wan Chen, Fengqi Zhou, Chaokun Luo, Zhuoling Lin, Xiaoyan Li, Yongbin Lin, Haotian Lin, Weirong Chen","doi":"10.1016/j.ajo.2024.11.024","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.11.024","url":null,"abstract":"<p><strong>Purpose: </strong>To describe clinical features, management, and outcomes of anterior segment dominated persistent fetal vasculature (aPFV) .</p><p><strong>Design: </strong>A secondary analysis of aPFV data from a longitudinal cohort study.</p><p><strong>Methods: </strong>The age and gender of each participant upon initial presentation, along with biological parameters, best-corrected visual acuity (BCVA), and biomicroscopic assessments were collected. The aPFV eyes were categorized into 3 groups (type I:pupillary-iris-anterior lens plane, type II:retrolental-anterior hyaloid membrane plane and type III:combined) according to the location of the vascular abnormalities and further classified into mild (a) and severe (b) according to the degree of severity. The surgical techniques used in this investigation were reported. Visual outcomes, ocular hypertension, postoperative adverse events, and additional surgical interventions were recorded at each follow-up visit.</p><p><strong>Results: </strong>A total of 470 individuals (619 eyes) were enrolled (ages 36.7±45.4 months, range 1 to 394 months), divided into 129 eyes (20.8%) Type 1, 420 eyes (67.9%) Type 2, and 70 eyes (11.3%) Type 3. Cataracts were identified in 505 eyes (81.6 %): for type I, the common forms were nuclear and membranous opacity (both 12/50 eyes, 24%); type II: subcapsular opacity (214/399 eyes, 53.6%) and type III: complete cortex (17/56 eyes, 30.4%). Posterior capsular tear was mostly present in type II a. Type III had the shallowest anterior chamber depth and the largest corneal astigmatism. Six hundred patients (96.7%) had surgery. After surgery, 119 eyes experienced adverse events: intravitreal hemorrhages occurred in 0.2% of eyes, visual axis obscuration occurred in 9.4% of eyes, and ocular hypertension which required eye drop medication occurred in 19.2% of eyes. In 47 patients (7.6%), a second surgery procedure was done. In 456 eyes (73.7%), visual acuity had improved at the last follow-up. The worst BCVA was associated with types IIb and IIIb.</p><p><strong>Conclusions: </strong>The magnitude of aPFV can be usefully characterized by a classification protocol focusing on the location and disease severity. Here, we propose a surgical and medical management algorithm for aPFV to achieve favorable outcomes with limited intraoperative and postoperative complications.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142794353","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}

{"title":"Visual Field Progression in the Ocular Hypertension Treatment Study.","authors":"Mae O Gordon, Dale K Heuer, Eve J Higginbotham, Richard K Parrish, Lei Liu, James D Brandt, Julia B Huecker, J Phillip Miller, Chamila Perera, Chris Xie, John L Keltner, Chris A Johnson, Stuart K Gardiner, Jeffrey M Liebmann, Michael A Kass","doi":"10.1016/j.ajo.2024.11.017","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.11.017","url":null,"abstract":"<p><strong>Purpose: </strong>To determine the rate of visual field (VF) loss before and after the diagnosis of primary open angle glaucoma (POAG) in the Ocular Hypertension Treatment Study (OHTS).</p><p><strong>Design: </strong>Pre-specified analyses of data collected prospectively in a clinical trial with extended follow-up.</p><p><strong>Setting and participants: </strong>Participants who developed POAG during OHTS 1 and 2 (February 1994 to December 2008) constitute an inception cohort. Follow-up data were collected in OHTS 3 (January 2016 to April 2019). Analyses were performed from July 2021 to August 2022.</p><p><strong>Intervention: </strong>In OHTS 1 and 2, visual field (VF) tests were performed every 6 months and stereoscopic optic disc photographs were taken every 12 months. These tests were repeated in OHTS 3.</p><p><strong>Main outcomes and measures: </strong>Slopes of mean deviation (MD) were calculated by linear regression for all eyes in OHTS 1 and 2: eyes that did not develop POAG, eyes that developed optic disc POAG only, and eyes that developed VF POAG with/without optic disc POAG. Mean pre- and post-POAG slopes were calculated for eyes with a minimum of 5 VFs for each period.</p><p><strong>Results: </strong>Mean age at diagnosis of POAG was 66.4 ± 9.5 SD years (n= 282 participants), (56%) were male, 61% were White non-Hispanic and 32% were Black not Hispanic by self-report. The post-POAG slope was -0.40 ± 0.64 SD dB/year for all POAG eyes (n=280 eyes), -0.19 ± 0.4 SD dB/yr. for optic disc POAG only eyes (n=112 eyes), and -0.54 ± 0.7 SD dB/yr. for VF POAG eyes with or without optic disc POAG (n=168 eyes). Among the VF POAG eyes, 69 (41%) had post-POAG MD slopes worse than or equal to -0.5 dB/year, 35 (21%) had slopes worse than or equal to -1.0 dB/year, and 9 (5.4%) had slopes worse than or equal to -2.0 dB/year.</p><p><strong>Conclusions and relevance: </strong>Some participants in OHTS had rapid rates of VF loss in one or both eyes despite being followed in a clinical study. This emphasizes that ocular hypertensive patients require careful follow-up, especially those at high risk of developing POAG to ensure early diagnosis and appropriate treatment of POAG.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142793986","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}

{"title":"Suprachoroidal Triamcinolone Acetonide for Noninfectious Uveitis: Real-World Impact on Clinical Outcomes.","authors":"Ketaki Panse, Abraham Hang, Jonathan Ruiz, Sapna Gangaputra, Sili Fan, Jeffrey Fine, Parisa Emami-Naeini, Glenn Yiu, Kareem Moussa","doi":"10.1016/j.ajo.2024.11.022","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.11.022","url":null,"abstract":"<p><strong>Purpose: </strong>To evaluate clinically relevant outcomes following suprachoroidal triamcinolone acetonide (TA) injection in eyes with noninfectious uveitis.</p><p><strong>Design: </strong>Retrospective interventional case series.</p><p><strong>Methods: </strong>61 eyes (51 patients) who underwent suprachoroidal TA injection between January 1, 2022 and July 1, 2024 were included from a single institution. Main outcome measures included change in central subfield thickness (CST) and best-corrected visual acuity (BCVA) at 6, 12, and 24 weeks in eyes with macular edema and improvement in fluorescein angiography (FA) score at 6 weeks in eyes who underwent FA.</p><p><strong>Results: </strong>52 eyes underwent suprachoroidal TA injection for uveitic macular edema. Mean CST improved from 437.61 microns at time of injection to 348.17 microns 6 weeks after (mean difference 89.44 microns, p<0.0001, n = 47). Additional treatment for macular edema was required for 17% (8/48 eyes), 48% (14/29 eyes), and 41% (9/22 eyes) of eyes that returned for follow-up at 6, 12, and 24 weeks, respectively. Of the 17 eyes that returned for follow-up at all visits (6, 12, and 24 weeks), 41.2% (7/17 eyes) did not require any additional treatment during the 24 week follow-up period. 10 eyes underwent FA at time of injection and 6 weeks later. The mean FA score improved from 11.74 to 9.14 (mean difference 2.60, p=0.047). No serious adverse events occurred.</p><p><strong>Conclusions: </strong>Suprachoroidal TA resulted in significant improvement in outcome measures, including central subfield thickness in eyes with macular edema, and leakage on fluorescein angiography, with a favorable side effect profile in real-world clinical practice.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142790934","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}

{"title":"Combining paracentral acute middle maculopathy and peripapillary fluid as biomarkers in anterior ischemic optic neuropathy: OCT biomarkers in anterior ischemic optic neuropathy.","authors":"Oliver Niels Klefter, Michael Stormly Hansen, Lea Lykkebirk, Yousif Subhi, Jane Maestri Brittain, Mads Radmer Jensen, Uffe Møller Døhn, Viktoria Fana, Anne Katrine Wiencke, Steffen Heegaard, Lene Terslev, Steffen Hamann","doi":"10.1016/j.ajo.2024.12.001","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.12.001","url":null,"abstract":"<p><strong>Purpose: </strong>To determine if paracentral acute middle maculopathy (PAMM) and peripapillary intraretinal and subretinal fluid (IRF/SRF) could help distinguish between arteritic anterior ischemic optic neuropathy (A-AION) and non-arteritic AION (NA-AION) at an early stage.</p><p><strong>Design: </strong>Nested prospective cross-sectional diagnostic accuracy study.</p><p><strong>Methods: </strong>This study used single-center optical coherence tomography (OCT) data from 8 patients with A-AION and 24 patients with NA-AION from two prospective cross-sectional studies with consecutive sampling (ClinicalTrials.gov: NCT05248906 and NCT05305079). The diagnosis of A-AION was based on expert interpretation of biochemical markers of inflammation, temporal artery biopsy and positron emission tomography/computed tomography. The diagnosis of NA-AION was made in cases without suspicion or clinical evidence of A-AION and with confirmed neuroophthalmological expert diagnosis. For this substudy patients were also required to have an OCT scan in relation to the diagnosis of AION. Macular OCT scans were graded by two independent, masked graders for the presence of PAMM and for IRF/SRF. The extension of IRF/SRF was assessed using an Early Treatment Diabetic Retinopathy Study (ETDRS) grid.</p><p><strong>Results: </strong>PAMM was found in 50% of patients with A-AION and in 0% of patients with NA-AION (P = 0.0019). In the setting of AION, the sensitivity of PAMM for the diagnosis of A-AION was 50% (95%CI: 16 to 84%) while the specificity was 100% (95%CI: 86 to 100%). Conversely, peripapillary IRF/SRF with extension into the ETDRS grid was observed in 83% of patients with NA-AION but in 0% of patients with A-AION (P =0.000047). The sensitivity of central macula-involving IRF/SRF for the diagnosis of NA-AION was 83% (95%CI: 63 to 95%), while the specificity was 100% (95%CI: 63 to 100%). Combining the two biomarkers, 75% of patients with AION could be classified based on OCT alone.</p><p><strong>Conclusion: </strong>PAMM appears to be a biomarker of A-AION while extensive peripapillary fluid appears to be a biomarker of NA-AION. Combining OCT biomarkers might allow for early classification of AION and warrants further prospective studies.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142790892","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}

{"title":"Subretinal Gene Therapy Drug AGTC-501 for XLRP Phase 1/2 Multicenter Study (HORIZON): 24-Month Safety and Efficacy Results: Subretinal Gene Therapy AGTC-501 for XLRP Ph 1/2 24M Results.","authors":"Paul Yang, David Birch, Andreas Lauer, Robert Sisk, Rajiv Anand, Mark E Pennesi, Alessandro Iannaccone, Antonio Yaghy, Abraham Scaria, JungAh Jung, Darin Curtiss, Nadia K Waheed","doi":"10.1016/j.ajo.2024.11.021","DOIUrl":"https://doi.org/10.1016/j.ajo.2024.11.021","url":null,"abstract":"<p><strong>Purpose: </strong>To evaluate the safety and efficacy of subretinal gene therapy using AGTC-501 (rAAV2tYF-GRK1-RPGR) in male participants with X-linked retinitis pigmentosa (XLRP).</p><p><strong>Design: </strong>Phase 1/2, open-label, dose-escalation study.</p><p><strong>Methods: </strong>Setting: Four centers in the United States. Patient or Study Population: Twenty-nine males with XLRP and confirmed pathogenic RPGR variants. Mean age was 31.6 years (range 15-55).</p><p><strong>Intervention: </strong>Subretinal injection of AGTC-501 at doses ranging from 2.48 × 10¹⁰ to 1.99 × 10¹² vg/eye administered in one eye per participant. Subretinal injection sites initially targeted the peripheral retina and then transitioned to the macula with successive cohorts.</p><p><strong>Main outcome measures: </strong>Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), laboratory parameters, and immunological responses. Efficacy was evaluated by mesopic microperimetry mean sensitivity.</p><p><strong>Results: </strong>All 29 participants experienced ≥1 TEAE. Eleven (38%) experienced ≥1 grade 3 TEAE. Six (21%) experienced ≥1 ocular SAE related to AGTC-501, including retinal detachment (n=4), subcapsular cataract (n=1), and glaucoma (n=1). Two (6.9%) experienced non-ocular treatment-emergent SAEs. Immunological findings did not indicate safety concerns. Three of 4 participants at the highest dose exhibited concerning retinal pigment epithelial changes. Half the participants at the highest tolerated dose (6.8 × 10¹¹vg/eye) maintained ≥7 dB improvement in ≥5 loci at 24 months.</p><p><strong>Conclusions: </strong>Subretinal AGTC-501 was generally well-tolerated. Despite all participants experiencing at least one TEAE, most of these events were mild in nature, exhibited complete resolution, and were associated with the subretinal injection procedure itself rather than the study agent. The highest dose exhibited an unfavorable risk-benefit profile due to the development of RPE changes. Although this group had the highest improvement in retinal sensitivity, our team has decided not to continue this dose in future clinical trials. Preliminary efficacy was observed at the maximum tolerated dose. Further studies are warranted to assess long-term safety and efficacy of AGTC-501 for XLRP treatment.</p>","PeriodicalId":7568,"journal":{"name":"American Journal of Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.1,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142790681","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}