为地理萎缩临床试验的终点提供依据

IF 5 2区医学 Q1 NEUROSCIENCES

Annual Review of Vision Science Pub Date : 2024-09-18 DOI:10.1146/annurev-vision-101922-045110

Eleonora M. Lad, Monika Fleckenstein, Frank G. Holz, Liangbo Shen, Lucian V. Del Priore, Rufino Silva, Giovanni Staurenghi, Nadia Waheed, Usha Chakravarthy

{"title":"为地理萎缩临床试验的终点提供依据","authors":"Eleonora M. Lad, Monika Fleckenstein, Frank G. Holz, Liangbo Shen, Lucian V. Del Priore, Rufino Silva, Giovanni Staurenghi, Nadia Waheed, Usha Chakravarthy","doi":"10.1146/annurev-vision-101922-045110","DOIUrl":null,"url":null,"abstract":"Geographic atrophy (GA), the non-neovascular advanced form of age-related macular degeneration, remains an important disease area in which treatment needs are currently unmet. Recent clinical trials using drugs that target the complement pathway have shown modest yet consistent reductions in GA expansion but without commensurate changes in measures of visual function. In this review, we summarize information from the wide range of studies describing the characteristics of GA morphology and enumerate the factors influencing the growth rates of lesions and the directionality of expansion. In addition, we review the relationship between GA growth and the various measures of vision that reflect changes in function. We consider the reasons for the discordance between the anatomical and functional endpoints in current use and discuss methods to align these key outcomes.","PeriodicalId":48658,"journal":{"name":"Annual Review of Vision Science","volume":"85 1","pages":""},"PeriodicalIF":5.0000,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Informing Endpoints for Clinical Trials of Geographic Atrophy\",\"authors\":\"Eleonora M. Lad, Monika Fleckenstein, Frank G. Holz, Liangbo Shen, Lucian V. Del Priore, Rufino Silva, Giovanni Staurenghi, Nadia Waheed, Usha Chakravarthy\",\"doi\":\"10.1146/annurev-vision-101922-045110\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Geographic atrophy (GA), the non-neovascular advanced form of age-related macular degeneration, remains an important disease area in which treatment needs are currently unmet. Recent clinical trials using drugs that target the complement pathway have shown modest yet consistent reductions in GA expansion but without commensurate changes in measures of visual function. In this review, we summarize information from the wide range of studies describing the characteristics of GA morphology and enumerate the factors influencing the growth rates of lesions and the directionality of expansion. In addition, we review the relationship between GA growth and the various measures of vision that reflect changes in function. We consider the reasons for the discordance between the anatomical and functional endpoints in current use and discuss methods to align these key outcomes.\",\"PeriodicalId\":48658,\"journal\":{\"name\":\"Annual Review of Vision Science\",\"volume\":\"85 1\",\"pages\":\"\"},\"PeriodicalIF\":5.0000,\"publicationDate\":\"2024-09-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Annual Review of Vision Science\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1146/annurev-vision-101922-045110\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"NEUROSCIENCES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Annual Review of Vision Science","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1146/annurev-vision-101922-045110","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"NEUROSCIENCES","Score":null,"Total":0}

引用次数: 0

摘要

地理萎缩（GA）是老年性黄斑变性的一种非新血管性晚期形式，目前仍是一个治疗需求尚未得到满足的重要疾病领域。最近使用靶向补体途径的药物进行的临床试验显示，GA 的扩大程度虽有轻微但却持续减少，但视觉功能却没有相应的变化。在这篇综述中，我们总结了描述 GA 形态特征的大量研究信息，并列举了影响病变生长速度和扩展方向的因素。此外，我们还回顾了GA生长与反映功能变化的各种视力测量指标之间的关系。我们考虑了目前使用的解剖终点和功能终点不一致的原因，并讨论了调整这些关键结果的方法。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Informing Endpoints for Clinical Trials of Geographic Atrophy

Geographic atrophy (GA), the non-neovascular advanced form of age-related macular degeneration, remains an important disease area in which treatment needs are currently unmet. Recent clinical trials using drugs that target the complement pathway have shown modest yet consistent reductions in GA expansion but without commensurate changes in measures of visual function. In this review, we summarize information from the wide range of studies describing the characteristics of GA morphology and enumerate the factors influencing the growth rates of lesions and the directionality of expansion. In addition, we review the relationship between GA growth and the various measures of vision that reflect changes in function. We consider the reasons for the discordance between the anatomical and functional endpoints in current use and discuss methods to align these key outcomes.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Annual Review of Vision Science Medicine-Ophthalmology

CiteScore

11.10

自引率

1.70%

发文量

期刊介绍： The Annual Review of Vision Science reviews progress in the visual sciences, a cross-cutting set of disciplines which intersect psychology, neuroscience, computer science, cell biology and genetics, and clinical medicine. The journal covers a broad range of topics and techniques, including optics, retina, central visual processing, visual perception, eye movements, visual development, vision models, computer vision, and the mechanisms of visual disease, dysfunction, and sight restoration. The study of vision is central to progress in many areas of science, and this new journal will explore and expose the connections that link it to biology, behavior, computation, engineering, and medicine.