Evidence map›Paper›PMID 42096221›Full record

ArticleInvestigative ophthalmology & visual science2026

Pilot Study of Patient-Reported Outcomes in Stargardt Disease: Correlation With Functional and Structural Findings.

Johnson Hoang, Iden Amiri, Lauren Dimalanta, Alessandro Iannaccone, Alice Yang Zhang

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Article in Investigative ophthalmology & visual science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

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2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

5 authors.

Johnson HoangDepartment of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States.
Iden AmiriDepartment of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States.
Lauren DimalantaDepartment of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States.
Alessandro IannacconeDepartment of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States.
Alice Yang ZhangDepartment of Ophthalmology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Purpose: The purpose of this study was to assess the impact of Stargardt disease (STGD) on activities of daily living and mental health, correlating patient-reported outcome (PRO) measures from the Michigan Retinal Degeneration (MRDQ) and Vision-Related Anxiety Questionnaires (MVAQs) with clinical data. Methods: Patients with STGD with biallelic pathogenic ABCA4 mutations were recruited from the University of North Carolina (UNC) at Chapel Hill and Foundation Fighting Blindness (FFB) databases. Participants completed MRDQ and MVAQ testing. Best-corrected visual acuity (BCVA), electroretinogram (ERG), and fundus autofluorescence (FAF) were retrospectively analyzed for UNC patients. Results: Fifty participants (mean age = 48.8 ± 18.5) were included. Median θ for MRDQ: central vision (0.138), color vision (0.143), contrast sensitivity (0.122), and photosensitivity (0.185). Median MVAQ θ scores for cone- and rod-related anxiety were 0.005 and -0.055, respectively. The following additional analyses were performed using the UNC subset of participants (n = 12): BCVA correlated with contrast sensitivity (P = 0.027). Rod ERG response correlated with scotopic vision (P = 0.044). Central FAF lesion extent correlated with central vision (P = 0.039) and contrast sensitivity (P = 0.032). Rod-related anxiety correlated with rod ERG response (P = 0.048), color vision (P < 0.001), contrast sensitivity (P < 0.001), photopic peripheral vision (P < 0.001), cone-related anxiety (P < 0.001), photosensitivity (P = 0.014), mesopic peripheral vision (P < 0.001), and scotopic vision (P < 0.001). Cone-related anxiety correlated with color vision (P = 0.026), contrast sensitivity (P = 0.013), photopic peripheral vision (P < 0.001), mesopic peripheral vision (P < 0.001), and scotopic vision (P < 0.001). Conclusions: In patients with STGD, structural and functional measures correlated with reduced functional vision and increased vision-related anxiety per PRO criteria. These findings provide insight into patient-perceived disease burden and may help inform future STGD interventions.

Indexed as

Macular DegenerationPatient Reported Outcome MeasuresStargardt DiseaseActivities of Daily LivingAdultAgedATP-Binding Cassette TransportersColor VisionContrast SensitivityElectroretinographyFemaleFluorescein AngiographyHumansMaleMiddle AgedMutationABCA4 protein, humanATP-Binding Cassette Transporters

Identifiers

PMID42096221
PMCPMC13170730

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.