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告知临床试验中地理萎缩的终点。

Informing Endpoints for Clinical Trials of Geographic Atrophy.

机构信息

Department of Ophthalmology, Duke University Medical Center, Durham, North Carolina, USA; email:

Department of Ophthalmology and Visual Science, John A. Moran Eye Center, University of Utah, Salt Lake City, Utah, USA; email:

出版信息

Annu Rev Vis Sci. 2024 Sep;10(1):455-476. doi: 10.1146/annurev-vision-101922-045110.

DOI:10.1146/annurev-vision-101922-045110
PMID:39292553
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.

摘要

地理萎缩(GA)是与年龄相关的黄斑变性的非新生血管性晚期形式,仍然是一个重要的疾病领域,目前尚未满足其治疗需求。最近使用靶向补体途径的药物进行的临床试验表明,GA 扩展有适度但一致的减少,但视觉功能的衡量标准没有相应变化。在这篇综述中,我们总结了广泛描述 GA 形态特征的研究信息,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之间的关系。我们考虑了当前使用的解剖学和功能终点之间不一致的原因,并讨论了使这些关键结果一致的方法。

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引用本文的文献

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Geographic atrophy: Understanding the relationship between structure and function.地图样萎缩:理解结构与功能之间的关系。
Asia Pac J Ophthalmol (Phila). 2025 May-Jun;14(3):100207. doi: 10.1016/j.apjo.2025.100207. Epub 2025 May 19.
2
Imaging of Geographic Atrophy: A Practical Approach.地图样萎缩的影像学检查:实用方法
Ophthalmol Ther. 2025 May 5. doi: 10.1007/s40123-025-01158-3.
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Relationships Between Diet and Geographic Atrophy Progression in the Age-Related Eye Diseases Studies 1 and 2.年龄相关性眼病研究1和2中膳食与地图样萎缩进展的关系
Nutrients. 2025 Feb 22;17(5):771. doi: 10.3390/nu17050771.
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Age-related macular degeneration: natural history revisited in geographic atrophy.年龄相关性黄斑变性:地图样萎缩的自然病程再探讨。
Eye (Lond). 2025 Feb;39(2):217-227. doi: 10.1038/s41433-024-03443-0. Epub 2024 Oct 29.