Journal
OPHTHALMOLOGY
Volume 123, Issue 4, Pages 817-828Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/j.ophtha.2015.12.009
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Funding
- Labex Lifesenses (Paris, France)
- Makindus (Doylestown, PA)
- Optos (Marlborough, MA)
- Carl Zeiss Meditec (Jena, Germany)
- Nidek (Padova, Italy)
- Topcon Medical Systems (Oakland, NJ)
- Scientific Technical Advisory Committee - Alcon Research Institute (Fort Worth, TX)
- Research to Prevent Blindness, Inc, New York, New York
- QLT, Inc. (Vancouver, BC, Canada)
- Office of Research and Administration
- Austrian Science Fund (Vienna, Austria) [J 3383-B23]
- National Institutes of Health, Bethesda, Maryland [EY013203]
- Foundation Fighting Blindess Career Development Award
- National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, United Kingdom
- Federal Ministry of Research of Germany
- Kerstan Foundation (Tubingen, Germany)
- Foundation Fighting Blindness Clinical Research Institute
- United States Department of Defense USAMRMC TATRC, Fort Meade, Maryland [W81-XWH-07-1-0720, W81XWH-09-2-0189]
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Purpose: To describe the design and baseline characteristics of patients enrolled into 2 natural history studies of Stargardt disease (STGD1). Design: Multicenter retrospective and prospective cohort studies. Participants: Three hundred sixty-five unique patients aged 6 years and older at baseline harboring diseasecausing variants in the ABCA4 gene and with specified ocular lesions were enrolled from 9 centers in the United States and Europe. Methods: In the retrospective study, patients contributed medical record data from at least 2 and up to 4 visits for at least 1 examination modality: fundus autofluorescence (FAF), spectral-domain (SD) optical coherence tomography (SD OCT), and/or microperimetry (MP). The total observational period was at least 2 years and up to 5 years between single visits. Demographic and visual acuity (VA) data also were obtained. In the prospective study, eligible patients were examined at baseline using a standard protocol, with 6-month follow-up visits planned for a 2-year period for serial Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected VA, SD OCT, FAF, and MP. Main Outcome Measures: Design and rationale of a multicenter study to determine the progression of STGD1 in 2 large retrospective and prospective international cohorts. Detailed baseline characteristics of both cohorts are presented, including demographics, and structural and functional retinal metrics. Results: Into the retrospective study, 251 patients (458 eyes) were enrolled; mean follow-up +/- standard deviation was 3.9 +/- 1.6 years. At baseline, 36% had no or mild VA loss, and 47% of the study eyes had areas of definitely decreased autofluorescence (DDAF) with an average lesion area of 2.5 +/- 2.9 mm(2) (range, 0.02-16.03 mm(2)). Two hundred fifty-nine patients (489 eyes) were enrolled in the prospective study. At baseline, 20% had no or mild VA loss, and 64% had areas of DDAF with an average lesion area of 4.0 +/- 4.4 mm(2) (range, 0.03-24.24 mm(2)). The mean retinal sensitivity with MP was 10.8 +/- 5.0 dB. Conclusions: The ProgStar cohorts have baseline characteristics that encompass a wide range of disease severity and are expected to provide valuable data on progression based on serial quantitative measurements derived from multiple methods, which will be critical to the design of planned clinical trials. (C) 2016 by the American Academy of Ophthalmology.
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