显性视神经萎缩的纵向视觉生物标志物：系统回顾和荟萃分析。

IF 4.9 2区医学 Q1 OPHTHALMOLOGY

Clinical and Experimental Ophthalmology Pub Date : 2025-05-07 DOI:10.1111/ceo.14543

Christopher A Ovens, John R Grigg, Clare L Fraser

{"title":"显性视神经萎缩的纵向视觉生物标志物：系统回顾和荟萃分析。","authors":"Christopher A Ovens, John R Grigg, Clare L Fraser","doi":"10.1111/ceo.14543","DOIUrl":null,"url":null,"abstract":"Background: Dominant Optic Atrophy (DOA) causes slowly progressive visual decline usually beginning in childhood. As new therapies come to clinical trial, the choice of biomarkers to be used as clinical trial endpoints has become a critical question to be addressed.Methods: We undertook a systematic review and meta-analysis of studies reporting longitudinal data of any biomarker in DOA patients.Results: In total, seven studies were included in the systematic review, and four presented paired results compatible with meta-analysis. Visual acuity was the only biomarker found with reported longitudinal data. Of the included studies in the meta-analysis, the rate of yearly visual acuity decline (0.022 LogMAR/year., 95% CI: -0.008 to 0.052) was not significantly different from zero (Z = 1.4, p = 0.155).Conclusion: Quantifying this slow rate of visual decline has implications for future study design and suggests that further natural history studies examining alternative biomarkers in DOA are warranted.","PeriodicalId":55253,"journal":{"name":"Clinical and Experimental Ophthalmology","volume":" ","pages":""},"PeriodicalIF":4.9000,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Longitudinal Visual Biomarkers in Dominant Optic Atrophy: A Systematic Review and Meta-Analysis.\",\"authors\":\"Christopher A Ovens, John R Grigg, Clare L Fraser\",\"doi\":\"10.1111/ceo.14543\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Background: Dominant Optic Atrophy (DOA) causes slowly progressive visual decline usually beginning in childhood. As new therapies come to clinical trial, the choice of biomarkers to be used as clinical trial endpoints has become a critical question to be addressed.Methods: We undertook a systematic review and meta-analysis of studies reporting longitudinal data of any biomarker in DOA patients.Results: In total, seven studies were included in the systematic review, and four presented paired results compatible with meta-analysis. Visual acuity was the only biomarker found with reported longitudinal data. Of the included studies in the meta-analysis, the rate of yearly visual acuity decline (0.022 LogMAR/year., 95% CI: -0.008 to 0.052) was not significantly different from zero (Z = 1.4, p = 0.155).Conclusion: Quantifying this slow rate of visual decline has implications for future study design and suggests that further natural history studies examining alternative biomarkers in DOA are warranted.\",\"PeriodicalId\":55253,\"journal\":{\"name\":\"Clinical and Experimental Ophthalmology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":4.9000,\"publicationDate\":\"2025-05-07\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Clinical and Experimental Ophthalmology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1111/ceo.14543\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"OPHTHALMOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Clinical and Experimental Ophthalmology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/ceo.14543","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"OPHTHALMOLOGY","Score":null,"Total":0}

引用次数: 0

摘要

背景：显性视萎缩（DOA）引起缓慢进行性视力下降，通常开始于儿童时期。随着新疗法进入临床试验阶段，选择生物标志物作为临床试验终点已成为一个亟待解决的关键问题。方法：我们对DOA患者中任何生物标志物的纵向数据进行了系统回顾和荟萃分析。结果：系统评价共纳入7项研究，其中4项研究的配对结果与meta分析相一致。视力是唯一与报告的纵向数据发现的生物标志物。在meta分析纳入的研究中，年视力下降率为0.022 LogMAR/年。（95% CI: -0.008 ~ 0.052）与零无显著差异（Z = 1.4, p = 0.155）。结论：量化这种缓慢的视力下降速度对未来的研究设计具有重要意义，并表明进一步的自然历史研究检查DOA的替代生物标志物是有必要的。

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

Longitudinal Visual Biomarkers in Dominant Optic Atrophy: A Systematic Review and Meta-Analysis.

Background: Dominant Optic Atrophy (DOA) causes slowly progressive visual decline usually beginning in childhood. As new therapies come to clinical trial, the choice of biomarkers to be used as clinical trial endpoints has become a critical question to be addressed.

Methods: We undertook a systematic review and meta-analysis of studies reporting longitudinal data of any biomarker in DOA patients.

Results: In total, seven studies were included in the systematic review, and four presented paired results compatible with meta-analysis. Visual acuity was the only biomarker found with reported longitudinal data. Of the included studies in the meta-analysis, the rate of yearly visual acuity decline (0.022 LogMAR/year., 95% CI: -0.008 to 0.052) was not significantly different from zero (Z = 1.4, p = 0.155).

Conclusion: Quantifying this slow rate of visual decline has implications for future study design and suggests that further natural history studies examining alternative biomarkers in DOA are warranted.

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

Clinical and Experimental Ophthalmology 医学-眼科学

CiteScore

7.60

自引率

12.50%

发文量

150

审稿时长

4-8 weeks

期刊介绍： Clinical & Experimental Ophthalmology is the official journal of The Royal Australian and New Zealand College of Ophthalmologists. The journal publishes peer-reviewed original research and reviews dealing with all aspects of clinical practice and research which are international in scope and application. CEO recognises the importance of collaborative research and welcomes papers that have a direct influence on ophthalmic practice but are not unique to ophthalmology.