Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania.
Ophthalmol Retina. 2023 Jul;7(7):564-572. doi: 10.1016/j.oret.2023.02.008. Epub 2023 Feb 19.
To predict 2-year visual acuity (VA) responses to anti-VEGF therapy, using early morphologic and functional responses in patients with neovascular age-related macular degeneration (nAMD).
Cohort within a randomized clinical trial.
A total of 1185 participants with untreated active nAMD and best-corrected visual acuity (BCVA) 20/25 to 20/320 at baseline.
Secondary analysis of data from participants randomized to either ranibizumab or bevacizumab and to 1 of 3 dosing regimens. Associations of 2-year BCVA responses with baseline morphologic and functional characteristics and their change from baseline at 3 months were assessed, using univariable and multivariable linear regression models for BCVA change and logistic regression models for ≥ 3-line BCVA gain from baseline. The performance of predictions for 2-year BCVA outcomes using these characteristics was assessed using R for BCVA change and area under the receiver operating characteristic curve (AUC) for ≥ 3-line BCVA gain.
Best-corrected visual acuity change and ≥ 3-line gain from baseline at year 2.
In multivariable analyses that included previously reported significant baseline predictors (baseline BCVA, baseline macular atrophy, baseline retinal pigment epithelium elevation [RPEE], and maximum width and early BCVA change from baseline at 3 months), new RPEE occurrence at 3 months was significantly associated with more BCVA gain at 2 years (10.2 letters vs. 3.5 letters for RPEE resolved, P < 0.001), and none of the other morphologic responses at 3 months were significantly associated with BCVA responses at 2 years. These significant predictors moderately predicted 2-year BCVA gain with an R = 0.36. Baseline BCVA and ≥ 3-line BCVA gain at 3 months predicted 2-year ≥ 3-line gain with AUC 0.83 (95% confidence interval, 0.81-0.86).
Most structural responses on OCT at 3 months were not independently predictive of the 2-year BCVA responses, which were associated with baseline factors and the 3-month BCVA response to anti-VEGF therapy. A combination of baseline predictors, early BCVA, and morphologic responses at 3 months only moderately predicted the long-term BCVA responses. Future research is needed to better understand the factors contributing to the variation in long-term vision outcomes with anti-VEGF therapy.
FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found after the references.
利用新生血管性年龄相关性黄斑变性（nAMD）患者早期形态学和功能反应，预测抗血管内皮生长因子（VEGF）治疗 2 年的视力（VA）反应。
随机临床试验中的队列研究。
共纳入 1185 名未经治疗的活动性 nAMD 患者，基线时最佳矫正视力（BCVA）为 20/25 至 20/320。
对随机分配至雷珠单抗或贝伐单抗治疗，并接受 3 种不同剂量方案之一的患者数据进行二次分析。采用单变量和多变量线性回归模型评估 2 年 BCVA 变化与基线形态学和功能特征及其与 3 个月时基线的变化之间的相关性，采用逻辑回归模型评估基线时 ≥ 3 行 BCVA 增益的相关性。使用 R 评估这些特征对 2 年 BCVA 结果的预测性能，采用受试者工作特征曲线下面积（AUC）评估 ≥ 3 行 BCVA 增益。
2 年时的 BCVA 变化和与基线相比的 ≥ 3 行增益。
在包含先前报道的重要基线预测因素（基线 BCVA、基线黄斑萎缩、基线视网膜色素上皮抬高[RPEE]和最大宽度以及 3 个月时的早期 BCVA 变化）的多变量分析中，3 个月时新出现的 RPEE 与 2 年时更多的 BCVA 增益显著相关（RPEE 消退者为 10.2 个字母，RPEE 未消退者为 3.5 个字母，P<0.001），而 3 个月时的其他形态学反应均与 2 年时的 BCVA 反应无显著相关性。这些重要的预测因素对 2 年 BCVA 增益的预测能力适中，R=0.36。基线 BCVA 和 3 个月时的 ≥ 3 行 BCVA 增益可预测 2 年时的 ≥ 3 行 BCVA 增益，AUC 为 0.83（95%置信区间，0.81-0.86）。
3 个月时的大多数 OCT 结构反应与 2 年 BCVA 反应均无独立相关性，而 2 年 BCVA 反应与基线因素和抗 VEGF 治疗后 3 个月的 BCVA 反应相关。基线预测因素、早期 BCVA 和 3 个月时的形态学反应相结合，只能适度预测长期 BCVA 反应。需要进一步研究以更好地了解与抗 VEGF 治疗长期视力结果相关的因素。
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