Natural History of Visual Dysfunction in ABCA4 Retinopathy and Its Genetic Correlates.


Journal

American journal of ophthalmology
ISSN: 1879-1891
Titre abrégé: Am J Ophthalmol
Pays: United States
ID NLM: 0370500

Informations de publication

Date de publication:
09 2023
Historique:
received: 13 02 2023
revised: 07 05 2023
accepted: 08 05 2023
pmc-release: 01 09 2024
medline: 18 8 2023
pubmed: 22 5 2023
entrez: 21 5 2023
Statut: ppublish

Résumé

To systematically assess the ability to detect change and retest reliability for a panel of visual function assessments in ABCA4 retinopathy. Prospective natural history study (NCT01736293). Patients with at least 1 documented pathogenic ABCA4 variant and a clinical phenotype consistent with ABCA4 retinopathy were recruited from a tertiary referral center. Participants underwent longitudinal, multifaceted functional testing, including measures of function at fixation (best-corrected visual acuity, low-vision Cambridge Color Test), macular function (microperimetry), and retina-wide function (full-field electroretinography [ERG]). Two- and 5-year ability to detect change was determined based on the η A total of 134 eyes from 67 participants with a mean follow-up of 3.65 years were included. In the 2-year interval, the microperimetry-derived perilesional sensitivity (η

Identifiants

pubmed: 37211138
pii: S0002-9394(23)00206-4
doi: 10.1016/j.ajo.2023.05.014
pmc: PMC10524499
mid: NIHMS1904150
pii:
doi:

Substances chimiques

ABCA4 protein, human 0
ATP-Binding Cassette Transporters 0

Banques de données

ClinicalTrials.gov
['NCT01736293']

Types de publication

Journal Article Research Support, N.I.H., Intramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

224-232

Subventions

Organisme : Intramural NIH HHS
ID : ZIA EY000534
Pays : United States

Informations de copyright

Published by Elsevier Inc.

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Auteurs

Maximilian Pfau (M)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Laryssa A Huryn (LA)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Marisa P Boyle (MP)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Catherine A Cukras (CA)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Wadih M Zein (WM)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Amy Turriff (A)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Ehsan Ullah (E)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Robert B Hufnagel (RB)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

Brett G Jeffrey (BG)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA. Electronic address: brett.jeffrey@nih.gov.

Brian P Brooks (BP)

From the National Eye Institute, National Institutes of Health, Bethesda, Maryland, USA.

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Classifications MeSH