Long-Term Natural History of Treatment-Naïve Geographic Atrophy in Age-Related Macular Degeneration.

IF 2.6 3区医学 Q2 OPHTHALMOLOGY

Translational Vision Science & Technology Pub Date : 2025-05-01 DOI:10.1167/tvst.14.5.5

Daniel R Muth, Laurence Quérat, Abinaya P Venkataraman, Alberto Dominguez-Vicent, Goran Petrovski, Pete A Williams, Filippo Locri, Sandrine A Zweifel, Anders Kvanta

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引用次数: 0

Abstract

Purpose: To evaluate the long-term fundus autofluorescence-based growth rate (GR) of treatment-naïve patients with geographic atrophy (GA) in age-related macular degeneration.

Methods: We conducted a prospective, single-center, observational study between February 2013 and September 2024 at the Department of Clinical Neuroscience, Karolinska Institutet, and St. Erik Eye Hospital, Stockholm/Solna, Sweden. Clinical examination and fundus autofluorescence were performed in patients with GA owing to dry age-related macular degeneration. The area and the absolute and square root transformed GR were analyzed every 6 months.

Results: We examined 432 eyes and enrolled 204 eyes (111patients). The median follow-up was 21 months (minimum-maximum, 5-123). Of 73 fovea-sparing, 22 eyes converted to foveal-involving over a median of 24 months. The mean growth for the total cohort was 1.597 mm2/y and 0.264 mm/y after square root transformation. Bilateral (1.621 mm2/y; 0.267 mm/y), multifocal (1.961 mm2/y; 0.322 mm/y), and fovea-sparing (1.987 mm2/y; 0.234 mm/y) lesions showed significantly faster growth when analyzed in isolation. In a mixed statistical model that controlled for bilaterality, only fovea status remained a significant influencer on the square root transformed GR (P < 0.001).

Conclusions: In this long-term GA cohort, an influence of lesion characteristics on GRs can be observed. Fovea sparing, multifocality, and bilaterality showed faster growth, depending on the statistical model. Patients presenting with one or more of these lesion characteristics hold a high potential for benefit of future treatments because a growth slow down may be more likely to be achieved. In fovea-sparing cases, functional preservation may be possible.

Translational relevance: By analyzing the data of one of the most extensive geographic atrophy patient cohorts in the Nordics, this study establishes a dataset on the long-term treatment-naïve growth dynamics. It provides a reference for upcoming preclinical treatment developments and clinical trial end points.

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年龄相关性黄斑变性中Treatment-Naïve地理萎缩的长期自然历史。

目的：评价treatment-naïve年龄相关性黄斑变性地理性萎缩（GA）患者的长期眼底自身荧光生长率（GR）。方法：我们于2013年2月至2024年9月在瑞典斯德哥尔摩/索尔纳的卡罗林斯卡医学院临床神经科学系和圣埃里克眼科医院进行了一项前瞻性、单中心、观察性研究。对干性老年性黄斑变性引起的GA患者进行了临床检查和眼底自身荧光检查。每6个月对面积、绝对GR和平方根GR进行分析。结果：共检查432只眼，纳入204只眼（111例患者）。中位随访为21个月（最短-最长，5-123）。在73只保留中央窝的眼睛中，22只眼睛在平均24个月的时间里转变为涉及中央窝。整个队列的平均增长率为1.597 mm2/年，平方根转化后为0.264 mm/年。双侧(1.621 mm2/y；0.267 mm/y)，多焦(1.961 mm2/y；0.322 mm/y)，中央凹保留(1.987 mm2/y；0.234 mm/y)病变在单独分析时显示出明显更快的生长。在控制双侧性的混合统计模型中，只有中央凹状态对平方根变换后的GR仍然有显著影响（P < 0.001）。结论：在这个长期GA队列中，可以观察到病变特征对GRs的影响。根据统计模型的不同，中央凹保留、多焦点和双侧的增长速度更快。表现出一种或多种病变特征的患者在未来的治疗中具有很高的获益潜力，因为生长减缓可能更有可能实现。在保留中央窝的病例中，功能保留是可能的。转化相关性：通过分析北欧最广泛的地理萎缩患者队列之一的数据，本研究建立了一个关于treatment-naïve长期增长动态的数据集。它为即将到来的临床前治疗发展和临床试验终点提供了参考。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Translational Vision Science & Technology Engineering-Biomedical Engineering

CiteScore

5.70

自引率

3.30%

发文量

346

审稿时长

25 weeks

期刊介绍： Translational Vision Science & Technology (TVST), an official journal of the Association for Research in Vision and Ophthalmology (ARVO), an international organization whose purpose is to advance research worldwide into understanding the visual system and preventing, treating and curing its disorders, is an online, open access, peer-reviewed journal emphasizing multidisciplinary research that bridges the gap between basic research and clinical care. A highly qualified and diverse group of Associate Editors and Editorial Board Members is led by Editor-in-Chief Marco Zarbin, MD, PhD, FARVO. The journal covers a broad spectrum of work, including but not limited to: Applications of stem cell technology for regenerative medicine, Development of new animal models of human diseases, Tissue bioengineering, Chemical engineering to improve virus-based gene delivery, Nanotechnology for drug delivery, Design and synthesis of artificial extracellular matrices, Development of a true microsurgical operating environment, Refining data analysis algorithms to improve in vivo imaging technology, Results of Phase 1 clinical trials, Reverse translational ("bedside to bench") research. TVST seeks manuscripts from scientists and clinicians with diverse backgrounds ranging from basic chemistry to ophthalmic surgery that will advance or change the way we understand and/or treat vision-threatening diseases. TVST encourages the use of color, multimedia, hyperlinks, program code and other digital enhancements.