September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
Progression of atrophic lesions prospectively determined by fundus autofluorescence: the natural history of the progression of atrophy secondary to Stargardt disease (ProgStar) study
Author Affiliations & Notes
  • Rupert Wolfgang Strauss
    Wilmer Eye institute, Johns Hopkins University, Baltimore, Maryland, United States
    Moorfields Eye Hospital and UCL Institute of Ophthalmology, London, United Kingdom
  • Xiangrong Kong
    Wilmer Eye institute, Johns Hopkins University, Baltimore, Maryland, United States
  • Anamika Jha
    Doheny Imaging Reading Center, Doheny Eye Institute, Los Angeles, California, United States
  • Paul S Bernstein
    Moran Eye Center, University of Utah, Salt Lake City, Utah, United States
  • Michel Michaelides
    Moorfields Eye Hospital and UCL Institute of Ophthalmology, London, United Kingdom
  • Jose Sahel
    CHNO des Quinze-Vingts, DHU Sight Restore, INSERM-DHOS CIC 1423, Sorbonne Universités, UPMC Univ Paris 06, INSERM, CNRS, Institut de la Vision, Paris, France
  • Elias I Traboulsi
    Cole Eye Institute, Cleveland Clinic, Cleveland, Ohio, United States
  • Eberhart Zrenner
    Center for Ophthalmology, Eberhards Karls University, Tübingen, Germany
  • Sheila K West
    Wilmer Eye institute, Johns Hopkins University, Baltimore, Maryland, United States
  • Srinivas R Sadda
    Doheny Imaging Reading Center, Doheny Eye Institute, Los Angeles, California, United States
    David Geffen School of Medicine, UCLA, Los Angeles, California, United States
  • Hendrik P Scholl
    Wilmer Eye institute, Johns Hopkins University, Baltimore, Maryland, United States
  • Footnotes
    Commercial Relationships   Rupert Strauss, None; Xiangrong Kong, None; Anamika Jha, None; Paul Bernstein, None; Michel Michaelides, None; Jose Sahel, Chronocam (I), Chronolife (I), Genesignal (C), GenSight Biologics (C), GenSight Biologics (I), Pixium Vision (C), Pixium Vision (I), Sanofi-Fovea (C), Vision Medicines (C); Elias Traboulsi, Retrophin (C), Sanofi (C), Sparks Therapeutics (C); Eberhart Zrenner, None; Sheila West, None; Srinivas Sadda, Allergan (F), Allergan (C), Avalanche (C), Bayer (C), Carl Zeiss Meditec (F), Genetech (F), Genetech (C), Iconic (C), Novartis (C), Optos (F), Optos (C), Regeneron (C), Stem Cells Inc (C), Thrombogenics (C); Hendrik Scholl, None
  • Footnotes
    Support  Supported by the Foundation Fighting Blindness Clinical Research Institute (FFB CRI) and a grant to FFB CRI by the U.S. Department of Defense USAMRMC TATRC, Fort Meade, Maryland (grant numbers W81-XWH-07-1-0720 and W81XWH-09-2-0189).
Investigative Ophthalmology & Visual Science September 2016, Vol.57, No Pagination Specified. doi:
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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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      © 2017 Association for Research in Vision and Ophthalmology.

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Abstract

Purpose : 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.

Methods : 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.

Results : 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.

Conclusions : 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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