PROGRESSION OF ABCA4-RELATED RETINOPATHY: Prognostic value of demographic, functional, genetic, and imaging parameters.
ATP-Binding Cassette Transporters
/ genetics
Adolescent
Adult
Aged
Atrophy
Demography
Disease Progression
Female
Follow-Up Studies
Humans
Male
Middle Aged
Mutation
/ genetics
Optical Imaging
Prognosis
Retinal Dystrophies
/ diagnosis
Retinal Pigment Epithelium
/ pathology
Retrospective Studies
Visual Acuity
/ physiology
Young Adult
Journal
Retina (Philadelphia, Pa.)
ISSN: 1539-2864
Titre abrégé: Retina
Pays: United States
ID NLM: 8309919
Informations de publication
Date de publication:
Dec 2020
Dec 2020
Historique:
entrez:
20
11
2020
pubmed:
21
11
2020
medline:
4
9
2021
Statut:
ppublish
Résumé
To investigate the prognostic value of demographic, functional, genetic, and imaging parameters on retinal pigment epithelium atrophy progression secondary to ABCA4-related retinopathy. Patients with retinal pigment epithelium atrophy secondary to ABCA4-related retinopathy were examined longitudinally with fundus autofluorescence imaging. Lesion area, perimeter, circularity, caliper diameters, and focality of areas with definitely decreased autofluorescence were determined. A model was used to predict the lesion enlargement rate based on baseline variables. Sample size calculations were performed to model the power in a simulated interventional study. Sixty-eight eyes of 37 patients (age range, 14-78 years) with a follow-up time of 10 to 100 months were included. The mean annual progression of retinal pigment epithelium atrophy was 0.89 mm. The number of atrophic areas, the retina-wide functional impairment, and the age-of-onset category constituted significant predictors for future retinal pigment epithelium atrophy growth, explaining 25.7% of the variability. By extension of a simulated study length and/or specific patient preselection based on these baseline characteristics, the required sample size could significantly be reduced. Trial design based on specific shape-descriptive factors and patients' baseline characteristics and the adaption of the trial duration may provide potential benefits in required cohort size and absolute number of visits.
Identifiants
pubmed: 33214501
doi: 10.1097/IAE.0000000000002747
pii: 00006982-202012000-00012
doi:
Substances chimiques
ABCA4 protein, human
0
ATP-Binding Cassette Transporters
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2343-2356Références
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