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Perfluorohexyloctane ophthalmic solution: a review of a prescription treatment for dry eye disease that directly targets tear evaporation 全氟己基辛烷眼药水:一种直接针对泪液蒸发的干眼症处方治疗方法综述
IF 0.7 Q4 OPHTHALMOLOGY Pub Date : 2023-11-02 DOI: 10.1080/17469899.2023.2275586
P. Karpecki, John D. Sheppard
ABSTRACT Introduction Perfluorohexyloctane (PFHO) ophthalmic solution (brand name, MIEBO) was recently approved by the United States Food and Drug Administration to treat the signs and symptoms of dry eye disease (DED). Unlike most DED treatments, PFHO addresses evaporative DED, which represents the vast majority of DED cases. PFHO may function as a surrogate for the tear film’s lipid layer, inhibiting evaporation. Areas covered This article summarizes data – found via PubMed and clinicaltrials.gov (May 30-1 September 2023) – surrounding PHFO and provides some considerations for its use. Preclinically, PFHO’s unique molecular properties facilitate formation of a layer at the tear film’s air-liquid interface and inhibit saline evaporation by 81%. These properties translate to improved clinical outcomes in DED: in phase 3 studies, 40% to 50% of PFHO-group patients had a ≥ 3-step total corneal fluorescein staining improvement at day 57, and approximately 60% of patients had ≥ 30% reduction in visual analog scale dryness scores, improvements that persisted to 52 weeks. Additionally, PFHO demonstrated a robust safety profile. Expert opinion PFHO is the first prescription eye drop that directly addresses hyperevaporation in DED. PFHO is poised to help fill the large unmet need of patients with evaporative DED, potentially improving patient outcomes and quality of life.
ABSTRACT Introduction 全氟己基辛烷(PFHO)眼科溶液(品牌名 MIEBO)最近获得美国食品和药物管理局批准,用于治疗干眼症(DED)的症状和体征。与大多数 DED 治疗方法不同,PFHO 针对的是蒸发性 DED,而蒸发性 DED 占 DED 病例的绝大多数。PFHO 可作为泪膜脂质层的替代物,抑制泪液蒸发。本文概述了通过 PubMed 和 clinicaltrials.gov(2023 年 5 月 30 日至 9 月 1 日)找到的有关 PHFO 的数据,并提供了使用 PHFO 的一些注意事项。临床前研究表明,PFHO 独特的分子特性有助于在泪膜的气液界面形成一层,并能抑制 81% 的生理盐水蒸发。这些特性改善了 DED 的临床疗效:在 3 期研究中,40%-50% 的 PFHO 组患者在第 57 天的总角膜荧光素染色改善≥ 3 级,约 60% 的患者视觉模拟量表干燥评分降低≥ 30%,这些改善持续了 52 周。此外,PFHO 还具有良好的安全性。专家观点 PFHO 是第一款直接解决 DED 过度蒸发问题的处方眼药水。PFHO 有望帮助满足蒸发性 DED 患者的大量未满足需求,从而改善患者的治疗效果和生活质量。
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引用次数: 0
Principles of ophthalmic ultrasound 眼科超声原理
Q4 OPHTHALMOLOGY Pub Date : 2023-10-31 DOI: 10.1080/17469899.2023.2277781
Ronald H. Silverman
ABSTRACTIntroduction Ultrasound imaging of the eye was introduced over 50 years ago. While the physical principles of ultrasound imaging have not changed, technology has undergone tremendous and ongoing development.Areas covered The fundamentals of ultrasound physics, biometry (A-scan), structural imaging (B-scan) and blood-flow imaging and measurement (Doppler) will be described. Emphasis will be placed on technological development and potential future advances.Expert opinion While A- and B-scan ultrasound of the eye has traditionally been performed with focused single-element transducers, the introduction of annular and linear arrays has enhanced clinical utility. Future advances, especially in multielement arrays, and point-of-care systems promise amazing new capabilities for diagnostic imaging of the eye and orbit.KEYWORDS: UltrasoundeyeDopplertransducerDisclaimerAs a service to authors and researchers we are providing this version of an accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proofs will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to these versions also. Article highlightsMedical diagnostic ultrasound developed in the aftermath of the second world war as a spinoff of Sonar technology used for underwater range finding.While ophthalmic ultrasound has largely been based on mechanically scanned, focused single-element transducer technology, virtually all other clinical specialties use linear array-based systems.Array-based systems allow control of focal depth. Linear arrays offer high scan rates and can provide Doppler to visualize and measure blood-flow.The advantages and decreasing cost of linear array systems is leading towards greater utilization for ophthalmic imaging.While the principles of ultrasound imaging are unchanged, the technology, especially in array-based systems, continues to advance.Declaration of interestR H Silverman has a financial interest in Arcscan, Inc. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.Reviewer disclosuresPeer reviewers on this manuscript have no relevant financial or other relationships to disclose.Figure 1: Left: Plots representing ultrasound waveform of a highly damped (top) and a poorly damped (bottom) transducer of the same wavelength (λ). To the right of each plot is the power spectrum corresponding to each waveform. Bandwidth (usually measured at 50% of maximum spectral power) is inversely related to pulse-length.Display full sizeFigure 2: 1970’s setup for immersion ultrasonography. The probe was pivoted by hand to sweep out a B-scan or held steady for an A-scan while the sh
本论文由NIH拨款R01 EY025215, R01 EB032082, P30 EY019007以及哥伦比亚大学眼科预防失明研究中心的无限制拨款资助。
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引用次数: 0
The long-term clinical outcomes of latanoprostene bunod 0.024% in glaucoma Treatment at a tertiary glaucoma center. 0.024%拉坦前列腺素治疗青光眼的长期临床效果
Q4 OPHTHALMOLOGY Pub Date : 2023-10-30 DOI: 10.1080/17469899.2023.2275587
Jae-Chiang Wong, Rebecca F. Neustein, Melih Ustaoglu, Eric Shiuey, Nikki Mehran, Marlene R. Moster, Reza Razeghinejad, L. Jay Katz, Jonathan Myers, Daniel Lee
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引用次数: 0
Are allergic conjunctivitis videos on YouTube a reliable source of information? YouTube上的过敏性结膜炎视频是可靠的信息来源吗?
Q4 OPHTHALMOLOGY Pub Date : 2023-10-18 DOI: 10.1080/17469899.2023.2272050
Nesime Setge Tıskaoğlu, Sevim Ayça Seyyar, Gizem Gürbostan Sosyal
ABSTRACTBackground Social media as well as YouTube are widely used to gain information on medical conditions. We aimed to assess and evaluate the quality and reliability of YouTube videos on ocular allergies and determine whether they are a trustworthy source of information.Research design and methods The first 60 videos from the search terms ‘allergic conjunctivitis’, ‘atopic conjunctivitis’, ‘allergic conjunctivitis symptoms’, and ‘allergic conjunctivitis eye drops’ were analyzed using modified DISCERN, Global Quality Score, Journal of the American Medical Association scores and Health on the Net Code criteria. The total number of views, view ratio, likes, comments, and duration were recorded and videos were evaluated as useful, non-useful, and misleading.Results The average mDISCERN score for the videos was 3.25 ± 0.76 (moderate), the average JAMA score was 2.76 ± 0.64 (intermediate), the average GQS score was 3.13 ± 0.8 (intermediate) and the average HONcode score was 7.8 ± 2.6 (intermediate). The most common video source was health professionals (61.9%).Conclusions Videos on allergic conjunctivitis while having reliable publishers are of moderate quality. Quality regulation of content uploaded on allergic conjunctivitis is needed. Health professionals should try to improve video content and provide more information on the treatment of allergic conjunctivitis.KEYWORDS: Allergic diseasesYouTubeocular allergyallergic conjunctivitismDISCERNJAMAGQS; HONcodeDisclaimerAs a service to authors and researchers we are providing this version of an accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proofs will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to these versions also. Declaration of interestsThe authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royaltiesAuthor contribution statement: All authors contributed to the conception and design of the article and interpreting the relevant literature, all authors were involved in writing the article and/or revised it for intellectual content.Reviewer disclosuresPeer reviewers on this manuscript have no relevant financial or other relationships to disclose.Figure 1: Flowchart of selection of videos included in the study.Display full sizeAdditional informationFundingThis paper was not funded.
社交媒体和YouTube被广泛用于获取医疗信息。我们的目的是评估和评估YouTube上关于眼部过敏的视频的质量和可靠性,并确定它们是否是值得信赖的信息来源。研究设计和方法对搜索词“过敏性结膜炎”、“特应性结膜炎”、“过敏性结膜炎症状”和“过敏性结膜炎滴眼液”的前60个视频进行分析,使用修改后的DISCERN、全球质量评分、美国医学协会杂志评分和健康网代码标准。视频的总观看次数、观看率、点赞、评论和持续时间被记录下来,并被评估为有用、无用和误导。结果视频的mDISCERN评分平均为3.25±0.76(中等),JAMA评分平均为2.76±0.64(中等),GQS评分平均为3.13±0.8(中等),HONcode评分平均为7.8±2.6(中等)。最常见的视频来源是卫生专业人员(61.9%)。结论有可靠的发布者的变应性结膜炎视频质量一般。需要对过敏性结膜炎上传内容进行质量规范。卫生专业人员应该努力改进视频内容,并提供更多关于过敏性结膜炎治疗的信息。关键词:过敏性疾病;眼过敏症;过敏性结膜炎;honcode免责声明作为对作者和研究人员的服务,我们提供了这个版本的接受手稿(AM)。在最终出版版本记录(VoR)之前,将对该手稿进行编辑、排版和审查。在制作和印前,可能会发现可能影响内容的错误,所有适用于期刊的法律免责声明也与这些版本有关。利益声明作者与任何组织或实体没有任何关联关系或财务参与,这些组织或实体与手稿中讨论的主题或材料有经济利益或经济冲突。这包括雇佣、咨询、酬金、股票所有权或期权、专家证词、获得或未决的赠款或专利,或版税。作者贡献声明:所有作者都对文章的概念和设计做出了贡献,并对相关文献进行了解释,所有作者都参与了文章的写作和/或对其进行了智力内容的修改。审稿人披露本文的每位审稿人没有相关的财务或其他关系需要披露。图1:研究中视频的选择流程图。附加信息本文未获得资助。
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引用次数: 0
Current concepts and recent updates in the treatment of ocular surface squamous neoplasia 眼表鳞状瘤样病变治疗的现状及最新进展
Q4 OPHTHALMOLOGY Pub Date : 2023-10-09 DOI: 10.1080/17469899.2023.2267763
Bharat Gurnani, Kirandeep Kaur
ABSTRACTIntroduction Ocular surface squamous neoplasia (OSSN) is a broad pathological entity that includes squamous neoplasm of conjunctival and corneal tissue. These various malignancies include conjunctival intraepithelial neoplasia (CIN), corneal epithelial dysplasia, squamous cell carcinoma (SCC), and mucoepidermoid carcinoma. OSSN diagnosis rests on exfoliative cytology, vital dyes, and imaging in the form of confocal microscopy and anterior segment optical coherence tomography. The medical management modalities are topical chemotherapy (Mitomycin C and 5 fluorouracil), immunotherapy (Interferon alpha 2b), and upcoming drugs, such as antivirals, anti-VEGF, and retinoic acid. Excision biopsy with no touch technique remains the gold standard for managing OSSN. However, surgery can lead to recurrence and unfavorable results in some cases.Areas Covered This article provides insights into the treatment aspects of OSSN with an overview of recent updates. The authors have discussed the current concept regarding the medical and surgical management of OSSN. The authors also dwell upon the recent updates along with the expert opinion.Expert Opinion- Although surgery remains the gold standard, topical drugs have revolutionized the management of OSSN. Patients should be closely followed up to look for any side effects. Cost, time duration, cosmesis, and side are the remaining major factors in deciding for medical versus surgical therapy.KEYWORDS: Ocular surface squamous neoplasiamitomycin C5-fluorouracilinterferon alpha 2bexcision biopsy Article highlights Ocular surface squamous neoplasia (OSSN) is a diverse ocular pathology that includes squamous neoplasm of conjunctival and corneal tissueThe important diagnostic modality includes exfoliative and impression cytology, vital dyes, high resolution anterior segment optical coherence tomography, and confocal microscopy.The medical management options of OSSN can be chemotherapeutic agents and immunotherapy.The gold standard for OSSN management is surgical excision biopsy by no touch technique and cryotherapy to the margins.The recent advances in OSSN management comprises human papilloma virus vaccine, light-activated belzupacap sarotalocan, verteporfin, cidofovir, bevacizumab, retinoic acid, and aloe vera.Declaration of interestsThe authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants, or patents received or pending, or royaltiesReviewer disclosuresPeer reviewers on this manuscript have no relevant financial or other relationships to disclose.Additional informationFundingThis paper was not funded.
摘要简介眼表鳞状瘤变(OSSN)是一个广泛的病理实体,包括结膜和角膜组织的鳞状肿瘤。这些不同的恶性肿瘤包括结膜上皮内瘤变(CIN)、角膜上皮发育不良、鳞状细胞癌(SCC)和黏液表皮样癌。OSSN的诊断依赖于剥脱性细胞学、重要染料和共聚焦显微镜和前段光学相干断层成像。医疗管理方式是局部化疗(丝裂霉素C和5氟尿嘧啶),免疫治疗(干扰素α 2b),以及即将推出的药物,如抗病毒药物,抗vegf和维甲酸。无接触切除活检技术仍然是治疗OSSN的金标准。然而,在某些情况下,手术可能导致复发和不良结果。本文提供了对OSSN治疗方面的见解,并概述了最近的更新。作者讨论了目前关于OSSN的内科和外科治疗的概念。作者还详细介绍了最近的更新以及专家的意见。专家意见-尽管手术仍然是金标准,局部药物已经彻底改变了OSSN的管理。患者应密切随访,以寻找任何副作用。费用、持续时间、外观和侧边是决定内科还是外科治疗的主要因素。摘要:眼表鳞状瘤变(OSSN)是一种包括结膜和角膜组织鳞状肿瘤在内的多种眼部病理,重要的诊断方法包括剥脱和印迹细胞学检查、活体染料检查、高分辨率前段光学相干断层扫描和共聚焦显微镜检查。OSSN的医疗管理选择可以是化疗药物和免疫治疗。治疗OSSN的金标准是手术切除活检无接触技术和冷冻治疗边缘。OSSN管理的最新进展包括人乳头瘤病毒疫苗、光激活belzupacap sarotalocan、维替波芬、西多福韦、贝伐单抗、维甲酸和芦荟。利益声明作者与任何组织或实体没有任何关联关系或财务参与,这些组织或实体与手稿中讨论的主题或材料有经济利益或经济冲突。这包括就业,咨询,酬金,股票所有权或期权,专家证词,赠款,或已收到或正在申请的专利,或版税。审稿人披露本手稿的每位审稿人没有相关的财务或其他关系要披露。本文未获得资助。
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引用次数: 0
The impact of lacrimal gland biopsies in current clinical practice 泪腺活检在当前临床实践中的影响
Q4 OPHTHALMOLOGY Pub Date : 2023-10-07 DOI: 10.1080/17469899.2023.2268285
Steffani Krista Someda, Yasuhiro Takahashi
KEYWORDS: dacryoadenitisidiopathic orbital inflammationimmunoglobulin G4-related diseaselacrimal gland biopsyorbital lymphoma
关键词:泪腺炎;特发性眼眶炎;免疫球蛋白g4相关疾病
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引用次数: 0
Deep anterior lamellar keratoplasty for advanced keratoconus: an updated review of indications, techniques and outcomes 深度前板层角膜移植术治疗晚期圆锥角膜:适应症、技术和结果的最新综述
Q4 OPHTHALMOLOGY Pub Date : 2023-10-06 DOI: 10.1080/17469899.2023.2268292
Sepehr Feizi, Niloufar Bineshfar, Helia Ashourizadeh, Mahan Shafie
ABSTRACTIntroduction The management of keratoconus has been changed significantly due to the advancement of technology in the field of contact lens materials and designs and introduction of new surgical approaches that aim to avoid or postpone corneal transplantation until the advanced stage of the disease. Deep anterior lamellar keratoplasty (DALK) has the advantage of preserving the patient’s own endothelium and eliminating endothelial graft rejection. Since the recent trend is to perform corneal transplantation mainly for severe keratoconus that are not amenable to other less invasive approaches, this review aims to provide an update on the outcomes of DALK in eyes with the advanced stage of keratoconus.Areas covered A comprehensive literature review of studies in English was conducted using the following keywords: “deep anterior lamellar keratoplasty” or “anterior lamellar keratoplasty” or “DALK” and “keratoconus” and “severe” or “advanced.”Expert commentary DALK significantly improves vision and refraction in advanced keratoconus and provides favorable graft survival. Femtosecond laser seems to improve the technique of the DALK procedure, and future developments could improve the reproducibility of DALK even further. However, current data is inconclusive for the benefit of femtosecond laser-assisted DALK for advanced keratoconus.KEYWORDS: Keratoconusadvanced stagecorneal transplantationdeep anterior lamellar keratoplastyDALKDisclaimerAs a service to authors and researchers we are providing this version of an accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proofs will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to these versions also. Article highlightsAs surgeons gain more experience and modify their techniques, DALK has emerged as an acceptable alternative to PK for advanced keratoconus when other less invasive approaches have failed to restore vision.Compared to PK, DALK has a superior safety profile as it eliminates the risk of endothelial graft rejection and preserves globe integrity against trauma.Despite improved safety, the visual and refractive outcomes after DALK can be inferior to those achieved after PK in advanced keratoconus eyes.Femtosecond laser has been used for the DALK procedure to increase the rate of successful big-bubble formation, decrease the rate of intraoperative Descemet membrane perforation, and provide better visual and refractive outcomes.Femtosecond laser-assisted DALK in the present form shows limited benefit, therefore, surgical design and parameters still need to be optimized.A medical economics study is required to assess the cost-effectiveness of laser-assisted DALK techniques in this particular subgroup of patients.AbbreviationsAS-OCT=Anterior segment optical coherence tomographyCDVA
由于隐形眼镜材料领域的技术进步,设计和引入新的手术方法,旨在避免或推迟角膜移植,直到疾病的晚期,圆锥角膜的治疗已经发生了重大变化。深前板层角膜移植术(DALK)具有保留患者自身内皮和消除内皮移植排斥反应的优点。由于最近的趋势是角膜移植主要用于严重圆锥角膜,而这些严重圆锥角膜无法采用其他侵入性较小的方法,因此本综述旨在提供晚期圆锥角膜DALK的最新结果。使用以下关键词:“深前板层角膜移植术”或“前板层角膜移植术”或“DALK”和“圆锥角膜”和“严重”或“进展”,对英文研究进行了全面的文献综述。专家评论DALK可显著改善晚期圆锥角膜患者的视力和屈光,并提供良好的移植物存活率。飞秒激光似乎改进了DALK过程的技术,未来的发展可以进一步提高DALK的可重复性。然而,目前的数据还不确定飞秒激光辅助的DALK对晚期圆锥角膜的益处。【关键词】角膜圆锥;晚期角膜移植;深前板层角膜成形术(dalk)免责声明:作为对作者和研究人员的服务,我们提供此版本的已接受稿件(AM)。在最终出版版本记录(VoR)之前,将对该手稿进行编辑、排版和审查。在制作和印前,可能会发现可能影响内容的错误,所有适用于期刊的法律免责声明也与这些版本有关。随着外科医生获得更多的经验和改进他们的技术,当其他侵入性较小的方法无法恢复视力时,DALK已成为晚期圆锥角膜患者PK的可接受替代方法。与PK相比,DALK具有更高的安全性,因为它消除了内皮移植排斥的风险,并保持了创伤的全球完整性。尽管安全性有所提高,但在晚期圆锥角膜患者中,DALK术后的视力和屈光效果可能不如PK术后。飞秒激光已被用于DALK手术,以提高大泡形成的成功率,降低术中网膜穿孔的发生率,并提供更好的视力和屈光效果。目前形式的飞秒激光辅助DALK的效益有限,因此,手术设计和参数仍需优化。需要一项医学经济学研究来评估激光辅助DALK技术在这一特定亚组患者中的成本效益。缩写as - oct =前段光学相干断层扫描cdva =矫正距离视力d =DiopterDALK=深前板层角膜成形术mm=毫米- μm=微孔soct =光学相干断层扫描ypk =穿透性角膜成形术udva =未矫正距离视力利益声明作者与任何组织或实体没有任何关联或经济参与与本文所讨论的主题或材料有经济利益或经济冲突手稿。这包括雇佣、咨询、酬金、股票所有权或期权、专家证词、获得或未决的赠款或专利,或特许权使用费。审稿人披露本文的每位审稿人没有相关的财务或其他关系需要披露。图1:描述筛选过程的文献检索流程图。附加信息本文未获得资助。
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引用次数: 0
Advances towards personalized therapies for Stargardt disease Stargardt病个性化治疗的进展
Q4 OPHTHALMOLOGY Pub Date : 2023-10-06 DOI: 10.1080/17469899.2023.2268289
Di Huang, Sam McLenachan, Fred K. Chen
ABSTRACTIntroduction ABCA4-associated Stargardt disease (STGD1) leads to bilateral central vision loss and is responsible for 12% of inherited retinal disease-related blindness.The lack of approved treatments highlights the urgent need for effective therapies.Areas covered This review explores personalized treatments for STGD1, focusing on therapeutic alternative splicing, genome editing, and translational read-through technologies. Literature searches as of July 2023 were undertaken via PubMed.Expert opinion Significant progress has been made in sequencing technology revealing the complexities of theABCA4 locus. Comprehensive functional assays now enable the determination of pathogenicity for ABCA4variants of uncertain significance. These breakthroughs facilitate the application of gene expression modulation technologies, ushering in a new era of personalized therapeutics. By targeting the ABCA4 gene and manipulating its expression, tailored treatments can address ABCA4-associated STGD1, offering enhanced efficacy and precise interventions based on the individual’s genetic profile. These advancements provide hope to those affected by STGD1, with improved treatment options and the potential to prevent vision loss. The convergence of genetic analysis breakthroughs and gene expression modulation technologies revolutionizes our understanding and treatment of inherited disorders, unlocking a promising frontier in personalized therapeutics. This transformative approach to STGD1 holds promise for similar breakthroughs in other inherited conditions.KEYWORDS: ABCA4inherited retinal diseasejuvenile macular dystrophypersonalized therapeuticsantisense oligonucleotidestherapeutic alternative splicinggene editingCRISPRbase editorprime editingDisclaimerAs a service to authors and researchers we are providing this version of an accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proofs will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to these versions also. Article highlightsAutosomal recessive Stargardt disease (STGD1) is a common monogenic inherited retinal dystrophy that leads to bilateral central vision loss and is responsible for 12% of IRD-related blindness.The spectrum of retinopathy associated with STGD1 includes childhood-onset cone-rod dystrophy, late-onset STGD1 with foveal sparing, and rapid-onset chorioretinopathy phenotype.STGD1 is caused by bi-allelic variants in the ATP-binding cassette transporter subfamily A4 gene (ABCA4) gene, which encodes the ABCA4 protein responsible for importing specific retinoid compounds in photoreceptor cells.Variable accumulation of cytotoxic lipofuscin-related fluorophores, such as A2E, is determined by combined deleterious effect of the biallelic ABCA4 variants and contributes to phenotypic varia
abca4相关的Stargardt病(STGD1)导致双侧中央视力丧失,是12%的遗传性视网膜疾病相关失明的原因。由于缺乏经批准的治疗方法,因此迫切需要有效的治疗方法。本综述探讨了STGD1的个性化治疗,重点是治疗替代剪接、基因组编辑和翻译读取技术。截至2023年7月,通过PubMed进行文献检索。测序技术取得了重大进展,揭示了abca4基因座的复杂性。综合功能分析现在能够确定不确定意义的abca4变异的致病性。这些突破促进了基因表达调节技术的应用,开创了个性化治疗的新时代。通过靶向ABCA4基因并操纵其表达,量身定制的治疗可以针对ABCA4相关的STGD1,提供基于个体遗传谱的增强疗效和精确干预。这些进步为STGD1患者带来了希望,改善了治疗选择,并有可能预防视力丧失。基因分析的突破和基因表达调节技术的融合彻底改变了我们对遗传性疾病的理解和治疗,为个性化治疗开辟了一个充满希望的前沿。这种针对STGD1的变革性方法有望在其他遗传疾病中取得类似突破。关键词:abca4遗传性视网膜疾病青少年黄斑营养不良个性化治疗三义寡核苷酸治疗替代剪接基因编辑crispr碱基编辑引物编辑免责声明作为对作者和研究人员的服务,我们提供此版本的接受稿件(AM)。在最终出版版本记录(VoR)之前,将对该手稿进行编辑、排版和审查。在制作和印前,可能会发现可能影响内容的错误,所有适用于期刊的法律免责声明也与这些版本有关。常染色体隐性Stargardt病(STGD1)是一种常见的单基因遗传性视网膜营养不良,可导致双侧中央视力丧失,并导致12%的ird相关失明。与STGD1相关的视网膜病变包括儿童期发病的锥杆营养不良、伴中央凹保留的迟发性STGD1和快速发病的脉络膜视网膜病变表型。STGD1是由atp结合盒转运蛋白亚家族A4基因(ABCA4)基因的双等位变异引起的,该基因编码ABCA4蛋白,负责在光感受器细胞中输入特定的类视黄醇化合物。细胞毒性脂褐素相关荧光团(如A2E)的可变积累是由双等位基因ABCA4变异的综合有害作用决定的,并有助于表型变异。反义寡核苷酸(AONs)技术被用于修饰基因表达,通过靶向特定序列和纠正由ABCA4变异引起的剪接缺陷,特别是在解决由深内含子变异引起的假外显子包含和由近外显子变异引起的外显子延伸/跳变。碱基编辑和引物编辑有望解决ABCA4基因中的错义变异,并可能潜在地靶向与STGD1相关的大量ABCA4变异。翻译读透诱导药物(TRIDs)正在被探索作为无义变异引起的STGD1的治疗策略,无义变异约占遗传疾病的12%。利益声明书Chen和S. McLenachan分别被列为国际专利WO/2020/237294(于2020年5月25日提交)的共同发明人和共同贡献者,该专利用于治疗视网膜色素变性的反义寡核苷酸药物。F.K. Chen是PYC Therapeutics, Apellis和Janssen的顾问。除了披露的内容外,作者与任何组织或实体没有其他相关关系或财务参与,这些组织或实体与稿件中讨论的主题或材料有经济利益或经济冲突。审稿人披露本文的一位审稿人披露,他们是诺华公司和杨森公司的顾问,与本文无关。本文的同行审稿人没有其他相关的财务关系或其他需要披露的信息。本文由第七频道telthon信托基金资助;未来健康研究及创新基金;澳大利亚黄斑疾病基金会;澳大利亚国家卫生与医学研究委员会,资助:GNT1116360, GNT1188694, GNT1054712, RF1142962;视网膜澳大利亚;以及麦卡斯克慈善基金会。
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引用次数: 0
What is on the horizon for dry age-related macular degeneration drug treatment? 干性老年性黄斑变性药物治疗前景如何?
Q4 OPHTHALMOLOGY Pub Date : 2023-10-04 DOI: 10.1080/17469899.2023.2267179
Federico Rissotto, Riccardo Sacconi, Aurelio Apuzzo, Gloria Oldoni, Emanuele Fusi, Andrea Servillo, Francesco Bandello, Giuseppe Querques
ABSTRACTIntroduction Age-related Macular Degeneration (AMD) is one of the leading causes of irreversible visual acuity reduction among the elderly population. Dry AMD, the most frequent AMD form, is characterized by photoreceptor loss, Retinal Pigmented Epithelium (RPE) dysfunction and retinal degeneration. Although in the last years several clinical trials have been carried out and many therapeutic molecules have been examined to treat dry AMD, currently its treatment remains unsatisfactory.Areas covered In this review we report and analyze the most important molecules and treatment that have been studied and carried out till nowadays, and the future therapies that may be a hope in this complex and serious disease. We have provided a narrative review after having analyzed the literature and we have selected more than 120 papers to deliver this article.Expert opinion Although in 2023 has been carried out the first FDA approved drugs, there is still a long way to go in developing effective therapies for dry-AMD. In the future, a better understanding of its pathogenesis may lead to the development of targeted or genetic therapies that not only have an anatomical effect on the retina but also have a functional impact.KEYWORDS: Age related macular degenerationGeographic atrophydrusencomplement inhibitorsneuroprotectiongenetic therapyintravitreal injectionDisclaimerAs a service to authors and researchers we are providing this version of an accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proofs will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to these versions also. Article highlightsIn this review we have summarized the most important molecules and trials that have been carried out till now in the attempt to treat dry-AMD.AREDS nutritional supplementation remains the standard of care for dry-AMD.In 2023 FDA has approved the first proven effect therapies for dry-AMD: Pegcetacoplan, and Avacincaptad Pegol.In the future, a genetic therapy focused on preventing and blocking the development of dry-AMD could be the most effective treatment, but at the present time, its development is at the embryonic stage.Declaration of InterestsR Sacconi has the following disclosures: Allergan Inc, Bayer Shering-Pharma, Medivis, Novartis, Zeiss.G Querques has the following disclosures: Alimera Sciences, Allergan Inc, Amgen, Bayer Shering-Pharma, Heidelberg, KBH, LEH Pharma, Lumithera, Novartis, Sandoz, Sifi, Sooft-Fidea, Zeiss.F Bandello has the following disclosures: Alcon (consultant), Alimera Sciences (consultant), Allergan Inc (consultant), Farmila-Thea (consultant), Bayer Shering-Pharma (consultant), Bausch and Lomb Genentech (consultant), Hoffmann-La-Roche (consultant), Novagali Pharma (consultant), Novartis (consultant), Sanofi-Aventis (consu
摘要简介年龄相关性黄斑变性(Age-related Macular Degeneration, AMD)是导致老年人视力不可逆性下降的主要原因之一。干性AMD是最常见的AMD形式,其特征是光感受器丧失,视网膜色素上皮(RPE)功能障碍和视网膜变性。尽管在过去的几年中进行了一些临床试验,并研究了许多治疗性分子来治疗干性黄斑变性,但目前其治疗仍不令人满意。在这篇综述中,我们报告和分析了迄今为止已经研究和开展的最重要的分子和治疗方法,以及未来治疗这种复杂严重疾病的可能希望。我们在分析文献后提供了一篇叙述性的综述,我们选择了120多篇论文来发表这篇文章。虽然在2023年已经进行了第一个FDA批准的药物,但在开发有效的治疗干性amd的方法上还有很长的路要走。在未来,对其发病机制的更好理解可能会导致靶向或基因治疗的发展,这些治疗不仅对视网膜有解剖作用,而且对功能也有影响。关键词:年龄相关性黄斑变性;地理萎缩症;补体抑制剂;神经保护;在最终出版版本记录(VoR)之前,将对该手稿进行编辑、排版和审查。在制作和印前,可能会发现可能影响内容的错误,所有适用于期刊的法律免责声明也与这些版本有关。本文综述了迄今为止治疗干性amd的重要分子和试验。AREDS营养补充仍然是干性amd护理的标准。2023年,FDA批准了首个经证实有效的治疗干性amd的药物:Pegcetacoplan和Avacincaptad Pegol。在未来,一种专注于预防和阻断干性amd发展的基因治疗可能是最有效的治疗方法,但目前,它的发展处于胚胎阶段。Sacconi有以下披露:Allergan Inc ., Bayer sher- pharma, Medivis, Novartis, Zeiss。G Querques有以下披露:Alimera Sciences, Allergan Inc ., Amgen, Bayer sher- Pharma,海德堡,KBH, LEH Pharma, Lumithera,诺华,山德士,Sifi, soft - fidea,蔡司。F Bandello有以下披露:Alcon(顾问)、Alimera Sciences(顾问)、Allergan Inc(顾问)、Farmila-Thea(顾问)、Bayer sherling -Pharma(顾问)、Bausch and lomgenentech(顾问)、Hoffmann-La-Roche(顾问)、Novagali Pharma(顾问)、Novartis(顾问)、Sanofi-Aventis(顾问)、Thrombogenics(顾问)、Zeiss(顾问)。除了披露的内容外,作者与任何组织或实体没有其他相关关系或财务参与,这些组织或实体与稿件中讨论的主题或材料有经济利益或经济冲突。审稿人披露本文的每位审稿人没有相关的财务或其他关系需要披露。图1:通过多彩图和OCT显示的中度AMD,黄色箭头表示一组结节,蓝色箭头表示多彩图和OCT同时显示的结节。图2:该图像显示了典型的遗传算法模式,由不同的滤波器指出。图3:通过红外、多色和OCT图像显示的中央凹保留遗传算法显示完整尺寸图4:图像显示主要补体抑制剂药物及其补体级联相应靶标显示完整尺寸附加信息
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引用次数: 0
Minimizing the risk of endophthalmitis following cataract surgery: Current antibiotic approaches 最小化白内障手术后眼内炎的风险:目前的抗生素方法
Q4 OPHTHALMOLOGY Pub Date : 2023-09-28 DOI: 10.1080/17469899.2023.2264508
Nimesh C. Patel, Avinash Pathengay, Mudit Tyagi, Rajeev Reddy Pappuru, Vivek Pravin Dave
ABSTRACTIntroduction Cataract surgery is the most commonly performed intraocular surgery all over the world. Endophthalmitis is a sight-threatening complication subsequent to intraocular procedures, open-globe injuries, metastatic systemic infections, and systemic inflammatory diseases. The incidence of post-cataract surgery endophthalmitis is 0.012% to 1.3%.Areas covered Patients’ ocular surface flora, surgical instruments, and surgical complications like posterior capsule rupture with vitreous loss and anterior vitrectomy. The most common bacteria causing post-cataract endophthalmitis in the Western world are gram-positive coagulase-negative Staphylococci, followed by Streptococci and Pseudomonas aeruginosa. Povidone-iodine (PI) is the only topical prophylactic antiseptic known to reduce endophthalmitis perioperatively with a three to five times reduction rate within one minute of irrigation. The European Society of Cataract & Refractive Surgeons (ESCRS) study recommendations are also discussed.Expert opinion There are no randomized controlled trials of PI with endophthalmitis rate as the primary end point. Based on retrospective data, 5% PI applied to conjunctiva prior to surgery reduced endophthalmitis rates four-fold. Intracameral injection of either vancomycin, cefazoline, cefuroxime, or moxifloxacin has a prophylactic effect. We recommend using preoperative 5% povidone-iodine for 30 seconds in the cul-de-sac and intracameral moxifloxacin or cefuroxime as effective prophylaxis against post-operative endophthalmitis.KEYWORDS: Endophthalmitisprophylaxisintracameral antibioticsintracameral moxifloxacinpovidone-iodineDisclaimerAs a service to authors and researchers we are providing this version of an accepted manuscript (AM). Copyediting, typesetting, and review of the resulting proofs will be undertaken on this manuscript before final publication of the Version of Record (VoR). During production and pre-press, errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal relate to these versions also. Declaration of interestThe authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.Reviewer disclosuresPeer reviewers on this manuscript have no relevant financial or other relationships to disclose.Table 2 Identified Risk factors and associationDownload CSVDisplay TableAdditional informationFundingThis paper was supported by funds from the Hyderabad Eye Research Foundation.
摘要白内障手术是世界范围内最常见的眼内手术。眼内炎是眼内手术、眼球开放性损伤、转移性全身性感染和全身性炎症性疾病之后的一种威胁视力的并发症。白内障术后眼内炎的发生率为0.012% ~ 1.3%。区域包括患者的眼表菌群,手术器械,手术并发症,如后囊膜破裂伴玻璃体丢失和前玻璃体切除术。在西方世界,引起白内障后眼内炎最常见的细菌是革兰氏阳性凝固酶阴性葡萄球菌,其次是链球菌和铜绿假单胞菌。聚维酮碘(PI)是唯一已知的局部预防性防腐剂,可减少围手术期眼内炎,冲洗后1分钟内减少3至5倍。欧洲白内障和屈光外科医师协会(ESCRS)的研究建议也进行了讨论。目前尚无以眼内炎发生率为主要终点的PI随机对照试验。根据回顾性数据,手术前结膜应用5% PI可将眼内炎发生率降低4倍。肠腔内注射万古霉素、头孢唑啉、头孢呋辛或莫西沙星均有预防作用。我们建议术前使用5%聚维酮碘30秒,眼内注射莫西沙星或头孢呋辛作为预防术后眼内炎的有效药物。免责声明作为对作者和研究人员的服务,我们提供此版本的已接受稿件(AM)。在最终出版版本记录(VoR)之前,将对该手稿进行编辑、排版和审查。在制作和印前,可能会发现可能影响内容的错误,所有适用于期刊的法律免责声明也与这些版本有关。利益声明作者与任何组织或实体没有相关的从属关系或财务参与,这些组织或实体与手稿中讨论的主题或材料有经济利益或经济冲突。这包括雇佣、咨询、酬金、股票所有权或期权、专家证词、获得或未决的赠款或专利,或特许权使用费。审稿人披露本文的每位审稿人没有相关的财务或其他关系需要披露。表2确定的危险因素及其关联下载csv显示表附加信息资金支持本文由海德拉巴眼科研究基金会资助。
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Expert Review of Ophthalmology
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