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Rupert Wolfgang Strauss, Xiangrong Kong, Anamika Jha, Paul S Bernstein, Michel Michaelides, Jose Sahel, Elias I Traboulsi, Eberhart Zrenner, Sheila K West, Srinivas R Sadda, Hendrik P Scholl; Progression of atrophic lesions prospectively determined by fundus autofluorescence: the natural history of the progression of atrophy secondary to Stargardt disease (ProgStar) study. Invest. Ophthalmol. Vis. Sci. 201657(12):.
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© ARVO (1962-2015); The Authors (2016-present)
The multicenter ProgStar study aims to characterize the natural history of Stargardt disease (STGD1) and to develop new outcome measures for clinical trials. The yearly rate of progression of STGD1 using the growth of atrophic lesions as measured by fundus autofluorescence (AF) imaging is the primary endpoint.
AF images from genetically confirmed STGD1 patients were sent from the nine participating sites to a central reading center (Doheny Image Reading Center, CA) and areas of definitely decreased AF (DDAF), well-demarcated questionably decreased AF (WD-QDAF), and poorly demarcated questionably decreased AF (PD-QDAF) were outlined and quantified. Background uniformity was graded as homogeneous versus heterogeneous. Linear models with generalized estimating equations were used to estimate the mean changes of lesion areas while accounting for within-eye and between-eye correlations.
489 study eyes of 259 study patients (54.4 % female) were enrolled in the prospective study and images from 444 eyes of 234 participants were graded for visits at baseline and six months follow-up. Mean age at baseline was 33.3 (sd 15.1) years. At baseline, DDAF was present in 306 (64%) eyes with a mean lesion size of 3.96 (sd 4.38) mm2; WD-QDAF in 71 (15%) eyes with a mean lesion size of 1.54 (sd 1.38) mm2; and PD-QDAF in 299 (62%) eyes with a mean lesion size of 2.17 (sd 1.92) mm2. Over six months, 14 of the 138 eyes (10.0 %) without any DDAF at baseline, developed a new DDAF lesion. Progression of DDAF in eyes which had a DDAF lesion at baseline was 0.24 (0.14-0.33) mm2 (p<0.001). There was a statistically significant difference in progression of lesion size over 6 months between eyes with homogeneous (estimated progression rate: 0.18 (CI 0.01 – 0.35) mm2) and heterogeneous (estimated progression rate: 0.49 (CI 0.30 – 0.70) mm2 ) background. Combining all lesion types, progression of areas of decreased AF was 0.33 (CI 0.27 – 0.40) mm2 over 6 months.
Mean increase of DDAF lesions in STGD1 was 0.24 mm2 over six months (suggesting a yearly progression rate of 0.48 mm2). AF may serve as a monitoring tool for interventional clinical trials in STGD1 that aim to slow down disease progression.
This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.
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