Vision-Related Patient-Reported Outcomes in Randomized Controlled Trials of Age-Related Macular Degeneration: A Systematic Review

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• Picked for Ophthalmology (top studies of the week, 9 August 2026): Phase III RCT, practice-changing for AMD outcomes

Abstract

TOPIC: This systematic review examined how vision-related patient-reported outcome measures (PROMs) were selected, operationalized, and reported at baseline in randomized controlled trials (RCTs) enrolling patients across the spectrum of age-related macular degeneration (AMD). CLINICAL RELEVANCE: Age-related macular degeneration is a major cause of visual disability, and visual acuity alone does not fully capture its effects on everyday functioning and quality of life. Inconsistent selection and incomplete reporting of PROMs may limit interpretation, comparison across trials, and evidence synthesis. METHODS: A systematic search of Medical Literature Analysis and Retrieval System Online, Embase, Web of Science Core Collection, and Scopus was conducted from database inception to September 27, 2023, supplemented by a targeted update for relevant 2024 and 2025 publications. Eligible studies were RCTs enrolling ≥20 participants with AMD and reporting both baseline visual acuity and baseline vision-related PROM data. Data on PROM instrument selection, scoring, baseline distributions, laterality context, and subgroup reporting were extracted and synthesized descriptively. RESULTS: Thirty-eight RCTs met the inclusion criteria, spanning early through late-stage AMD. Across included trials, 11 distinct PROM instruments were reported. The National Eye Institute Visual Function Questionnaire family predominated (78.9%), with the National Eye Institute Visual Function Questionnaire‑25 being the most commonly used version (n = 17). Baseline patient-reported functional status varied by disease stage: early and intermediate AMD cohorts reported higher scores (75.3-89.1), whereas trials enrolling neovascular AMD patients showed lower and more heterogeneous scores (59.1-77.9). Subscale and domain reporting was inconsistent, with many trials presenting only composite scores or a limited subset of subscales. Baseline PROM results were not reported by subgroup, preventing comparisons across patient populations. Reporting of visual function was incomplete with only 13% of studies reporting both better-seeing and worse-seeing eyes. CONCLUSION: The interpretive value of PROMs in AMD trials is constrained by inconsistent baseline reporting and limited reporting of both-eye and binocular acuity needed to contextualize patient-level function. Enhanced consistency in stage-appropriate PROM selection and routine reporting of baseline distributions are essential to support patient-centered evaluation and evidence synthesis in AMD research. FINANCIAL DISCLOSURES: Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

Abstract as published, via PubMed.