在USH2A相关视网膜变性（RUSH2A）研究中，连续4年的视力、全视野刺激阈值和视网膜电图检查
Visual Acuity, Full-field Stimulus Thresholds, and Electroretinography for 4 Years in The Rate of Progression of USH2A-related Retinal Degeneration (RUSH2A) Study.
作者信息
Birch David G, Cheng Peiyao, Maguire Maureen G, Duncan Jacque L, Ayala Allison R, Cheetham Janet K, Doucet Nicole R, Durham Todd A, Fahim Abigail T, Ferris Frederick L, Huckfeldt Rachel M, Melia Michele, Michaelides Michel, Pennesi Mark E, Sahel José-Alain, Stingl Katarina, Vincent Ajoy, Weng Christina Y
机构信息
Retina Foundation of the Southwest, Dallas, Texas.
Jaeb Center for Health Research, Tampa, Florida.
出版信息
Ophthalmol Sci. 2024 Nov 8;5(2):100648. doi: 10.1016/j.xops.2024.100648. eCollection 2025 Mar-Apr.
PURPOSE
To describe progression of best-corrected visual acuity (BCVA), full-field stimulus thresholds (FST), and electroretinography (ERG) over 4 years in the -related Retinal Degeneration study and to assess their suitability as clinical trial endpoints.
DESIGN
Prospective natural history study.
PARTICIPANTS
Participants (n = 105) with biallelic disease-causing sequence variants in USH2A and BCVA letter scores of ≥54 were included.
METHODS
BCVA, FST, fundus-guided microperimetry, static perimetry, and spectral domain OCT were performed annually and ERG at baseline and 4 years only. Mixed effects models were used to estimate annual rates of change with 95% confidence intervals. Associations of change from baseline to 4 years between BCVA, FST, ERG, and other metrics were assessed with Spearman correlation coefficients (r).
MAIN OUTCOME MEASURES
Best-corrected visual acuity, FST, and ERG.
RESULTS
The annual rate of decline in BCVA was 0.83 (95% confidence interval: 0.65-1.02) letters/year. For FST, the change was 0.09 (0.07-0.11) log cd.s/m/year for white threshold, 0.10 (0.08-0.12) log cd.s/m/year for blue threshold, and 0.05 (0.04-0.06) log cd.s/m/year for red threshold. Changes were 22.6 (17.4-28.2)%/year for white threshold, 26.0 (20.3-32.1)%/year for blue threshold, and 12.3 (8.7-16.0)%/year for red threshold. The high percentage of eyes with undetectable ERGs at baseline limited assessment of change.
CONCLUSIONS
Best-corrected visual acuity was not a sensitive measure of progression over 4 years. Full-field stimulus threshold was a more sensitive measure; however, additional information on the clinical relevance of changes in FST is needed before this test can be adopted as an endpoint for clinical trials.
FINANCIAL DISCLOSURES
Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.
目的
描述在与视网膜色素变性相关的研究中，最佳矫正视力（BCVA）、全视野刺激阈值（FST）和视网膜电图（ERG）在4年中的变化情况，并评估它们作为临床试验终点的适用性。
设计
前瞻性自然史研究。
参与者
纳入了105名双等位基因致病序列变异位于USH2A基因且BCVA字母评分≥54的参与者。
方法
每年进行BCVA、FST、眼底引导微视野检查、静态视野检查和光谱域光学相干断层扫描（OCT），仅在基线和4年时进行ERG检查。使用混合效应模型估计年变化率及95%置信区间。用Spearman相关系数（r）评估BCVA、FST、ERG与其他指标从基线到4年的变化之间的关联。
主要观察指标
最佳矫正视力、FST和ERG。
结果
BCVA的年下降率为0.83（95%置信区间：0.65 - 1.02）个字母/年。对于FST，白色阈值的变化为0.09（0.07 - 0.11）log cd.s/m²/年，蓝色阈值为0.10（0.08 - 0.12）log cd.s/m²/年，红色阈值为0.05（0.04 - 0.06）log cd.s/m²/年。白色阈值变化率为22.6（17.4 - 28.2）%/年，蓝色阈值为26.0（20.3 - 32.1）%/年，红色阈值为12.3（8.7 - 16.0）%/年。基线时ERG无法检测的眼睛比例较高，限制了对变化的评估。
结论
最佳矫正视力不是4年进展的敏感指标。全视野刺激阈值是更敏感的指标；然而，在该测试可被用作临床试验终点之前，还需要关于FST变化的临床相关性的更多信息。
财务披露
在本文末尾的脚注和披露中可能会找到专有或商业披露信息。
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