June 2017
Volume 58, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2017
Progression of Atrophic Lesions Determined by Fundus Autofluorescence: The Natural History of the Progression of Atrophy secondary to Stargardt Disease (ProgStar) Study
Author Affiliations & Notes
  • Rupert Wolfgang Strauss
    Moorfields Eye Hosp & Inst of Ophthal UCL, London, United Kingdom
    Ophthalmology, Johannes Kepler University & Medical University Graz, Linz, Austria
  • Xiangrong Kong
    DANA Center for preventive ophthalmology, Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland, United States
  • Alex Ho
    Doheny Eye Institute, Los Angeles, California, United States
  • Anamika Jha
    Doheny Eye Institute, Los Angeles, California, United States
  • Michel Michaelides
    Moorfields Eye Hosp & Inst of Ophthal UCL, London, United Kingdom
  • Artur V Cideciyan
    Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, United States
  • Jose Alain Sahel
    Institut de la Vision, Paris, France
    Ophthalmology, UPMC Medical Center, Pittsburgh, Pennsylvania, United States
  • David G Birch
    Retina Foundation of the Southwest, Dallas, Texas, United States
  • Amir H Hariri
    Doheny Eye Institute, Los Angeles, California, United States
  • Srinivas R Sadda
    Doheny Eye Institute, Los Angeles, California, United States
    UCLA David Geffen School of Medicine, Los Angeles, California, United States
  • Sheila West
    DANA Center for preventive ophthalmology, Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland, United States
  • Hendrik P Scholl
    Ophthalmology, Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland, United States
    Department of Ophthalmology, University of Basel, Basel, Switzerland
  • Footnotes
    Commercial Relationships   Rupert Strauss, None; Xiangrong Kong, None; Alex Ho, None; Anamika Jha, None; Michel Michaelides, None; Artur Cideciyan, None; Jose Sahel, Banque publique d'Investissement (F), Chronocam (I), Chronolife (I), ERC Synergy "HELMHOLTZ" (F), Foundation Fighting Blindness (F), Genesignal (C), GenSight (F), GenSight Biologics (C), GenSight Biologics (I), LabEx LIFESENSES (ANR-10-LABX-65) (F), Pixium Vision (C), Pixium Vision (I); David Birch, None; Amir Hariri, None; Srinivas Sadda, Allergan (F), Allergan (C), Carl Zeiss Meditec (F), Genentech (F), Genentech (C), Iconic (C), Novartis (C), Optos (F), Optos (C), Thrombogenics (C); Sheila West, None; Hendrik Scholl, Acucela Inc. (F), Boehringer Ingelheim Pharma GmbH & Co. KG (C), Daiichi Sankyo (C), Genentech Inc./F. Hoffmann-La Roche Ltd. (R), Gensight Biologics (C), Genzyme Corp./Sanofi (R), Gerson Lehrman Group (C), Guidepoint (C), Intellia Therapeutics (C), NightstaRx Ltd. (F), QLT, Inc (F), ReNeuron Group Plc/Ora Inc. (C), Shire (C), Vision Medicines, Inc. (C)
  • Footnotes
    Support  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 June 2017, Vol.58, 2013. doi:
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    • Get Citation

      Rupert Wolfgang Strauss, Xiangrong Kong, Alex Ho, Anamika Jha, Michel Michaelides, Artur V Cideciyan, Jose Alain Sahel, David G Birch, Amir H Hariri, Srinivas R Sadda, Sheila West, Hendrik P Scholl; Progression of Atrophic Lesions Determined by Fundus Autofluorescence: The Natural History of the Progression of Atrophy secondary to Stargardt Disease (ProgStar) Study. Invest. Ophthalmol. Vis. Sci. 2017;58(8):2013.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : The multicenter ProgStar study aims to characterize the natural history of Stargardt disease type 1 (STGD1) and develop new outcome measures for trials. The yearly rate of growth of atrophic lesions measured by fundus autofluorescence (FAF) imaging is the primary endpoint.

Methods : Participants were enrolled from nine sites in the US and Europe, and were followed every 6 months. 18-month follow-up data were used in this analysis. FAF images were sent from the nine participating sites to a central reading center 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. Based on background uniformity, homogeneous versus heterogeneous background was defined. Linear mixed effects models were used to estimate the annual rate of change in lesion areas while accounting for between-eye and within-eye correlations.

Results : 489 study eyes of 259 patients (54 % female) were enrolled in the prospective study. Mean age at baseline was 33.3 (sd 15.1) years. At baseline, DDAF was present in 307 (63%) eyes, mean lesion size 3.94 (sd 4.36) mm2; WD-QDAF in 71 (15%) eyes, mean lesion size 1.54 (sd 1.38) mm2, and PD-QDAF in 300 (62%) eyes, mean lesion size 2.16 (sd 1.91) mm2. Over 18 months, 50 eyes out of the 151 eyes (33.1 %) without any DDAF at baseline and available at month 18, developed a lesion of DDAF. Among eyes with DDAF at baseline, progression rate of DDAF was 0.75 (95%CI 0.61-0.90) mm2 per year (p<.001), and the rate of DDAF growth was significantly different by baseline AF background (p<.001): it was 0.44 (0.23-0.65) and 1.02 (0.83-1.21) mm2 per year in eyes with homogeneous versus heterogeneous background, respectively. Baseline DDAF size was also significantly associated with DDAF growth rate - the larger the baseline lesion, the faster the progression (p<.001). Combining all lesion types, the mean progression rate was 0.65 (CI 0.55-0.74) mm2 per year.

Conclusions : FAF may serve as an outcome measure of treatment trials that aim to slow/halt the progression of STGD1.

This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.

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