Pegcetacoplan治疗的视力和生活质量：来自OAKS和DERBY试验的事后分析。

IF 4.2 1区医学 Q1 OPHTHALMOLOGY

American Journal of Ophthalmology Pub Date : 2025-05-29 DOI:10.1016/j.ajo.2025.05.023

Allen Chiang , Matthew Davis , Warren Stevens , Sunir Garg , Tom Sheidow , Daniel L. Jones , Michele Intorcia , Alex McKeown , Ursula M. Schmidt-Erfurth , Sujata P. Sarda , Caroline R. Baumal

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引用次数: 0

摘要

目的：根据地理萎缩（GA）病灶距中央凹中心的距离，评价佩西可平对视功能和患者生活质量的影响。设计：来自OAKS和DERBY的事后分析，两项全球性、24个月、多中心、随机、双盲、假对照的3期研究，评估pegcetacoplan治疗GA在年龄相关性黄斑变性（AMD）中的疗效。参与者：纳入了888例GA研究患者，这些患者收集了进行事后分析所需的所有数据。方法、干预或测试：分别在基线和每4个月和6个月对pegcetacplan治疗眼和假（观察）眼的最佳矫正视力（BCVA）和25项美国国家眼科研究所视力功能问卷（NEI VFQ-25）进行评估，直到第24个月完成。根据光学相干断层扫描得出的GA病变位置≥250µm （n=192）对眼睛进行分层或主要结局测量：根据距离（≥250）评估24个月内BCVA和NEI VFQ-25的基线变化。与假手术相比，经pegcetacoplan治疗的GA病变边缘距中央凹≥250 μm的眼睛在24个月时的视力（平均+5.6 [SE 3.2] [P=.0785]）和生活质量（平均+4.0 [SE 2.4] [P=.0905]）较基线有方向性较慢的下降。结论：对于距中央凹中心≥250 μm的GA病变患者，Pegcetacoplan治疗后，视力和生活质量的变化（BCVA，平均值为-1.6 [SE 1.1] [P=.1522]）和生活质量（NEI VFQ-25, -2.3 [1.1] [P=.0284]）有潜在意义。对GA病变患者进行培西可平治疗后视力和生活质量的变化

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Visual Acuity and Quality of Life Outcomes With Pegcetacoplan Treatment: A Post Hoc Analysis From the OAKS and DERBY Trials

OBJECTIVE

To evaluate the effect of pegcetacoplan on visual function and patient quality of life (QoL) measures based on distance of geographic atrophy (GA) lesions from the center of the fovea.

DESIGN

Post hoc analysis from OAKS and DERBY, two global, 24-month, multicenter, randomized, double-masked, sham-controlled phase 3 studies evaluating pegcetacoplan treatment for GA in age-related macular degeneration (AMD).

PARTICIPANTS

888 study patients with GA for whom all data necessary for the post hoc analysis to be performed had been collected were included.

METHODS

Best-corrected visual acuity (BCVA) and 25-item National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25) were assessed at baseline and every 4 and 6 months, respectively, to completion at month 24 in both pegcetacoplan-treated eyes and sham (observed) eyes. Eyes were stratified based on optical coherence tomography‒derived GA lesion location ≥250 µm (n = 192) or <250 µm (n = 696) from the foveal center.

MAIN OUTCOME MEASURES

Change from baseline in BCVA and NEI VFQ-25 was evaluated over 24 months in GA lesions based on distance (≥250 or <250 µm) from the foveal center. Adjustment was conducted via propensity score weighting, where model specification and baseline covariate selection were done a priori based on clinical rationale.

RESULTS

Pegcetacoplan-treated eyes with GA lesion margins ≥250 µm from the foveal center demonstrated directionally slower decline in visual acuity (mean +5.6 [SE 3.2] [P = .0785]) and QoL (mean +4.0 [SE 2.4] [P = .0905]) from baseline at 24 months compared with sham. The change in visual acuity (BCVA, mean −1.6 [SE 1.1] [P = .1522]) and QoL (NEI VFQ-25, −2.3 [1.1] [P = .0284]) in pegcetacoplan-treated eyes with GA lesion margins <250 µm from the foveal center were within the limits of variability compared with sham.

CONCLUSIONS

Pegcetacoplan treatment demonstrated a potentially meaningful slower decline in visual acuity and QoL for patients with GA lesions ≥250 µm from the foveal center. The change in visual acuity and QoL seen with pegcetacoplan treatment for patients with GA lesions <250 µm from the foveal center were within the limits of variability.

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来源期刊

American Journal of Ophthalmology 医学-眼科学

CiteScore

9.20

自引率

7.10%

发文量

406

审稿时长

36 days

期刊介绍： The American Journal of Ophthalmology is a peer-reviewed, scientific publication that welcomes the submission of original, previously unpublished manuscripts directed to ophthalmologists and visual science specialists describing clinical investigations, clinical observations, and clinically relevant laboratory investigations. Published monthly since 1884, the full text of the American Journal of Ophthalmology and supplementary material are also presented online at www.AJO.com and on ScienceDirect. The American Journal of Ophthalmology publishes Full-Length Articles, Perspectives, Editorials, Correspondences, Books Reports and Announcements. Brief Reports and Case Reports are no longer published. We recommend submitting Brief Reports and Case Reports to our companion publication, the American Journal of Ophthalmology Case Reports. Manuscripts are accepted with the understanding that they have not been and will not be published elsewhere substantially in any format, and that there are no ethical problems with the content or data collection. Authors may be requested to produce the data upon which the manuscript is based and to answer expeditiously any questions about the manuscript or its authors.