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SHR Neuro Cancer Cardio Lipid Metab Microb

Strauss, RW; Muñoz, B; Ho, A; Jha, A; Michaelides, M; Mohand-Said, S; Cideciyan, AV; Birch, D; Hariri, AH; Nittala, MG; Sadda, S; Scholl, HPN; ProgStar Study Group.
Incidence of Atrophic Lesions in Stargardt Disease in the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: Report No. 5.
JAMA Ophthalmol. 2017; 135(7):687-695 Doi: 10.1001/jamaophthalmol.2017.1121 [OPEN ACCESS]
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Leading authors Med Uni Graz: Strauß Rupert
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Abstract:: Outcome measures that are sensitive to disease progression are needed as clinical end points for future treatment trials in Stargardt disease. To examine the incidence of atrophic lesions of the retinal pigment epithelium in patients with Stargardt disease as determined by fundus autofluorescence imaging. In this retrospective multicenter cohort study, 217 patients 6 years and older at baseline at tertiary referral centers in Europe, the United States, and the United Kingdom who were harboring disease-causing variants in the adenosine triphosphate (ATP)-binding cassette subfamily A member 4 (ABCA4) gene and who met the following criteria were enrolled: (1) at least 1 well-demarcated area of atrophy with a minimum diameter of 300 µm, with the total area of all atrophic lesions being less than or equal to 12 mm2 in at least 1 eye at the most recent visit, and (2) fundus autofluorescence images for at least 2 visits with a minimum of 6 months between at least 2 visits. Data were collected between August 22, 2013, and December 12, 2014. Data analysis was performed from March 15, 2015, through January 31, 2017. Images were evaluated by staff at a central reading center. Areas of definitely decreased autofluorescence (DDAF) and questionably decreased autofluorescence (QDAF) were outlined and quantified. Lesion-free survival rates were estimated using Kaplan-Meier survival curves. Incidence of atrophic lesions as determined by fundus autofluorescence. The 217 patients (mean [SD] age, 21.8 [13.3] years; 127 female [57.5%]; 148 white [68.2%]) contributed 390 eyes for which the mean (SD) follow-up time was 3.9 (1.6) years (range, 0.7-12.1 years). Among eyes without DDAF at first visit, the median time to develop a DDAF lesion was 4.9 years (95% CI, 4.3-5.6 years). Among eyes without QDAF, the median time to develop a QDAF lesion was 6.3 years (95% CI, 5.6-9.7 years). Eyes with a lesion of DDAF at the first visit were less likely to develop a QDAF lesion compared with eyes without a lesion of DDAF (hazard ratio, 0.19; 95% CI, 0.05-0.70; P = .01). An estimated 50% of the eyes without DDAF at first visit will develop the lesion in less than 5 years, suggesting that incidence of DDAF could serve as an outcome measure for treatment trials.
Find related publications in this database (using NLM MeSH Indexing): Disease Progression -; Electroretinography -; Europe - epidemiology; Female -; Fluorescein Angiography -; Follow-Up Studies -; Forecasting -; Fundus Oculi -; Humans -; Incidence -; Macular Degeneration - complications; Macular Degeneration - congenital; Macular Degeneration - diagnosis; Macular Degeneration - epidemiology; Macular Degeneration - etiology; Male -; Ophthalmoscopy -; Retinal Pigment Epithelium - pathology; Retrospective Studies -; Tomography, Optical Coherence -; United States - epidemiology; Visual Acuity -; Young Adult -