Debourdeau Eloi, Beylerian Helene, Nguyen Vuong, Barthelmes Daniel, Gillies Mark, Gabrielle Pierre Henry, Vujosevic Stela, Otoole Louise, Puzo Martin, Creuzot-Garcher Catherine, Wolff Benjamin, Daien Vincent
Department of Ophthalmology, Gui de Chauliac Hospital, 80 Avenue Augustin Fliche, 34000, Montpellier, France.
Institute for Neurosciences of Montpellier INM, Univ. Montpellier, INSERM, 34091, Montpellier, France.
Ophthalmol Ther. 2024 Sep;13(9):2343-2355. doi: 10.1007/s40123-024-00983-2. Epub 2024 Jul 8.
Anti-vascular endothelial growth factor (VEGF) is generally given using pro re nata or "treat-and-extend" (T&E) regimens for neovascular age-related macular degeneration (nAMD). Randomized clinical trials have reported that T&E is superior to Pro re nata (PRN), but results from clinical trials may not always be replicated in clinical practice. Real-world data comparing T&E and PRN regimens for nAMD are limited. The objective of this work was to report 24-month outcomes of PRN versus T&E regimens for ranibizumab and aflibercept to treat nAMD in routine clinical practice.
We conducted a retrospective analysis of data from a prospectively designed observational outcomes registry, the Fight Retinal Blindness! Project (FRB). Treatment-naïve eyes starting nAMD treatment with at least three injections using a T&E or PRN regimen were tracked by using the FRB. The primary outcome was the mean change in visual acuity (VA) measured by the number of letters read on a logarithm of the minimum angle of resolution chart at 2 years versus baseline. The secondary outcome was the number of injections at 2 years.
From January 1, 2015 to January 31, 2019, 3313 eyes from 2948 patients with nAMD were included: 1243 eyes from 1065 patients were classified as PRN and 2070 eyes from 1935 patients started a T&E regimen. At 24 months, patients on the T&E regimen experienced significantly greater mean (95% confidence interval) improvement in VA than those on PRN (+ 4.2 [3.1, 5.2] vs. + 1.3 [0.1, 2.6] letters; p < 0.001), with more injections (14.9 standard deviation(SD) 4.3) vs. 9.8(SD 4.3); p < 0.001).
Eyes treated with a T&E regimen had better VA outcomes from VEGF inhibitors than eyes treated PRN. This large real-world data assessment supports previous data from randomized clinical trials that the T&E regimen delivers better outcomes than PRN.
抗血管内皮生长因子（VEGF）通常采用按需给药或“治疗-延长”（T&E）方案用于治疗新生血管性年龄相关性黄斑变性（nAMD）。随机临床试验报告称T&E方案优于按需给药（PRN）方案，但临床试验结果在临床实践中可能并不总是能得到重复验证。比较nAMD的T&E和PRN方案的真实世界数据有限。这项研究的目的是报告在常规临床实践中，雷珠单抗和阿柏西普的PRN与T&E方案治疗nAMD的24个月结果。
我们对前瞻性设计的观察性结局登记库“抗击视网膜失明！项目”（FRB）的数据进行了回顾性分析。使用FRB追踪首次接受nAMD治疗、采用T&E或PRN方案且至少注射三次的初治眼。主要结局是在2年时与基线相比，通过在最小分辨角对数视力表上读出的字母数测量的视力（VA）的平均变化。次要结局是2年时的注射次数。
从2015年1月1日至2019年1月31日，纳入了来自2948例nAMD患者的3313只眼：来自1065例患者的1243只眼被归类为PRN组，来自1935例患者的2070只眼开始采用T&E方案。在24个月时，采用T&E方案的患者在VA方面的平均改善（95%置信区间）显著大于采用PRN方案的患者（+4.2[3.1,5.2]对+1.3[0.1,2.6]个字母；p<0.001），且注射次数更多（14.9标准差（SD）4.3次对9.8次（SD 4.3次）；p<0.001）。
与采用PRN方案治疗的眼相比，采用T&E方案治疗的眼使用VEGF抑制剂后VA结局更好。这项大型真实世界数据评估支持了先前随机临床试验的数据，即T&E方案比PRN方案能带来更好的结局。
