A Workshop on Measuring the Progression of Atrophy Secondary to Stargardt Disease in the ProgStar Studies: Findings and Lessons Learned

Ann-Margret Ervin^{1

2}, Rupert W Strauss^{1

3

4

5}, Mohamed I Ahmed¹, David Birch⁶, Janet Cheetham⁷, Frederick L Ferris 3rd⁸, Michael S Ip⁹, Glenn J Jaffe¹⁰, Maureen G Maguire¹¹, Etienne M Schönbach^{1

12}, SriniVas R Sadda⁹, Sheila K West¹, Hendrik P N Scholl^{1

13

14}; ProgStar Study Group

Affiliations

¹ Wilmer Eye Institute, The Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
² Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA.
³ Moorfields Eye Hospital NHS Foundation Trust, and UCL Institute of Ophthalmology, University College London, London, UK.
⁴ Department of Ophthalmology, Kepler University Clinic, Linz, Austria.
⁵ Department of Ophthalmology, Medical University Graz, Graz, Austria.
⁶ Retina Foundation of the Southwest, Dallas, TX, USA.
⁷ Foundation Fighting Blindness Clinical Research Institute, Columbia, MD, USA.
⁸ National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
⁹ Doheny Imaging Reading Center, Doheny Eye Institute, David Geffen School of Medicine at University of California Los Angeles, CA, USA.
¹⁰ Department of Ophthalmology, Duke University School of Medicine, Durham, NC, USA.
¹¹ Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
¹² Case Western Reserve University, Cleveland, OH, USA.
¹³ Department of Ophthalmology, University of Basel, Basel, Switzerland.
¹⁴ Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland.

PMID: 31019847
PMCID: PMC6469878
DOI: 10.1167/tvst.8.2.16

A Workshop on Measuring the Progression of Atrophy Secondary to Stargardt Disease in the ProgStar Studies: Findings and Lessons Learned

Ann-Margret Ervin et al. Transl Vis Sci Technol. 2019.

. 2019 Apr 12;8(2):16.

doi: 10.1167/tvst.8.2.16. eCollection 2019 Apr.

Authors

Affiliations

¹ Wilmer Eye Institute, The Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
² Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA.
³ Moorfields Eye Hospital NHS Foundation Trust, and UCL Institute of Ophthalmology, University College London, London, UK.
⁴ Department of Ophthalmology, Kepler University Clinic, Linz, Austria.
⁵ Department of Ophthalmology, Medical University Graz, Graz, Austria.
⁶ Retina Foundation of the Southwest, Dallas, TX, USA.
⁷ Foundation Fighting Blindness Clinical Research Institute, Columbia, MD, USA.
⁸ National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
⁹ Doheny Imaging Reading Center, Doheny Eye Institute, David Geffen School of Medicine at University of California Los Angeles, CA, USA.
¹⁰ Department of Ophthalmology, Duke University School of Medicine, Durham, NC, USA.
¹¹ Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
¹² Case Western Reserve University, Cleveland, OH, USA.
¹³ Department of Ophthalmology, University of Basel, Basel, Switzerland.
¹⁴ Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland.

PMID: 31019847
PMCID: PMC6469878
DOI: 10.1167/tvst.8.2.16

Abstract

The Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies were designed to measure the progression of Stargardt disease through the use of fundus autofluorescence imaging, optical coherence tomography, and microperimetry. The overarching objectives of the studies were to document the natural course of Stargardt disease and identify the most appropriate clinical outcome measures for clinical trials assessing the efficacy and safety of upcoming treatments for Stargardt disease. A workshop organized by the Foundation Fighting Blindness Clinical Research Institute was held on June 11, 2018, in Baltimore, MD, USA. Invited speakers discussed spectral-domain optical coherence tomography, fundus autofluorescence, and microperimetry methods and findings in the ProgStar prospective study. The workshop concluded with a panel discussion of optimal endpoints for measuring treatment efficacy in Stargardt disease. We summarize the workshop presentations in light of the most current literature on Stargardt disease and discuss potential clinical outcome measures and endpoints for future treatment trials.

Keywords: Stargardt; fundus autofluorescence; microperimetry; natural history; optical coherence tomography.

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Figures

**Figure 1**
EZ layer disruption. PRS outer, photoreceptor segment layer – outer.

See this image and copyright information in PMC

References

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1. Tanna P, Strauss RW, Fujinami K, Michaelides M. Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options. Br J Ophthalmol. 2017;101:25–30. - PMC - PubMed
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A Workshop on Measuring the Progression of Atrophy Secondary to Stargardt Disease in the ProgStar Studies: Findings and Lessons Learned

Affiliations

A Workshop on Measuring the Progression of Atrophy Secondary to Stargardt Disease in the ProgStar Studies: Findings and Lessons Learned

Authors

Affiliations

Abstract

Figures

References

LinkOut - more resources

Full Text Sources