Unknown

Dataset Information

0

Visual Acuity Change Over 24 Months and Its Association With Foveal Phenotype and Genotype in Individuals With Stargardt Disease: ProgStar Study Report No. 10.


ABSTRACT: Importance:Limited data from prospective studies are available to understand the natural history of ABCA4-related Stargardt disease (STGD1). Such data are important for determining appropriate outcome measures for future STGD1 trials. Objective:To estimate the rate of loss of best-corrected visual acuity (BCVA) during 2 years and to estimate the associations of BCVA loss with foveal phenotype and genotype in patients with STGD1. Design, Setting, and Participants:This multicenter prospective cohort study included 259 participants (489 study eyes) with molecularly confirmed STGD1 who were 6 years or older. The participants were enrolled at 9 centers in the United States and Europe and were followed up every 6 months for 2 years. Exposures:Baseline BCVA and presence and type of foveal lesion (determined via fundus autofluorescence images) and genotype (classified into 4 groups based on the number and pathogenicity of ABCA4 mutations). Main Outcomes and Measures:Rate of BCVA change per year. Results:The mean (SD) age was 33 (15) years. Of 259 the participants, 141 (54%) were female, and 222 (85%) were white. The overall rate of BCVA loss was 0.55 (95% CI, 0.20-0.90) letters per year during the 2 years. Eyes with baseline BCVA worse than 20/200 showed an improvement of 0.65 (95% CI, 0.1-1.2) letters per year. At baseline, the mean BCVA for eyes without foveal lesion was 20/32, and their BCVA change rate over time was 0.1 (95% CI, -1.2 to 1.35) letters per year (P?=?.89). Eyes with a foveal lesion but having BCVA of 20/70 or better at baseline lost BCVA at a rate of 3 (95% CI, 1.5-4.4) letters per year (P?

SUBMITTER: Kong X 

PROVIDER: S-EPMC6142940 | biostudies-literature | 2018 Aug

REPOSITORIES: biostudies-literature

altmetric image

Publications

Visual Acuity Change Over 24 Months and Its Association With Foveal Phenotype and Genotype in Individuals With Stargardt Disease: ProgStar Study Report No. 10.

Kong Xiangrong X   Fujinami Kaoru K   Strauss Rupert W RW   Munoz Beatriz B   West Sheila K SK   Cideciyan Artur V AV   Michaelides Michel M   Ahmed Mohamed M   Ervin Ann-Margret AM   Schönbach Etienne E   Cheetham Janet K JK   Scholl Hendrik P N HPN  

JAMA ophthalmology 20180801 8


<h4>Importance</h4>Limited data from prospective studies are available to understand the natural history of ABCA4-related Stargardt disease (STGD1). Such data are important for determining appropriate outcome measures for future STGD1 trials.<h4>Objective</h4>To estimate the rate of loss of best-corrected visual acuity (BCVA) during 2 years and to estimate the associations of BCVA loss with foveal phenotype and genotype in patients with STGD1.<h4>Design, setting, and participants</h4>This multic  ...[more]

Similar Datasets

| S-EPMC7256863 | biostudies-literature
| S-EPMC7083180 | biostudies-literature
| S-EPMC10916884 | biostudies-literature
| S-EPMC6780607 | biostudies-literature
| S-EPMC5473977 | biostudies-literature
| S-EPMC6681653 | biostudies-literature
| S-EPMC9634033 | biostudies-literature
| S-EPMC7282970 | biostudies-literature
| S-EPMC7563662 | biostudies-literature
| S-EPMC6657704 | biostudies-literature