Rupert W Strauss1,2,3,4, Xiangrong Kong1,5, Millena G Bittencourt1, Alexander Ho6, Anamika Jha6, Etienne M Schönbach1, Mohamed I Ahmed1, Beatriz Muñoz1, Ann-Margret Ervin1, Michel Michaelides2, David G Birch7, José-Alain Sahel8, Janet S Sunness9, Eberhart Zrenner10, Saghar Bagheri1,11, Michael Ip6, SriniVas Sadda6, Sheila West1, Hendrik P N Scholl12,13,14. 1. Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland, USA. 2. Moorfields Eye Hospital NHS Foundation Trust, and UCL Institute of Ophthalmology, University College London, London, United Kingdom. 3. Department of Ophthalmology, Kepler University Clinic and Kepler University Linz, Linz, Austria. 4. Department of Ophthalmology, Medical University Graz, Graz, Austria. 5. Department of Biostatistics and Epidemiology, University of Massachusetts-Amherst, Amherst, Massachusetts, USA. 6. Doheny Eye Institute, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, California, USA. 7. Retina Foundation of the Southwest, Dallas, Texas, USA. 8. Sorbonne Universités, University Pierre et Marie Curie (UPMC) Université de Paris 06, Institut National de la Santé et de la Recherche Médicale (INSERM), Centre National de la Recherche Scientifique (CNRS), Institut de la Vision, Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts, Paris, France. 9. Hoover Low Vision Rehabilitation Services, Greater Baltimore Medical Center and University of Maryland School of Medicine, Baltimore, Maryland, USA. 10. Center for Ophthalmology, Eberhard-Karls University Hospital, Tübingen, Germany. 11. Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, Massachusetts, USA. 12. Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland, USAHendrik.Scholl@usb.ch. 13. Department of Ophthalmology, University of Basel, Basel, SwitzerlandHendrik.Scholl@usb.ch. 14. Institute of Molecular and Clinical Ophthalmology Basel, Basel, SwitzerlandHendrik.Scholl@usb.ch.
Abstract
PURPOSE: To describe the study design and characteristics at first visit of participants in the longitudinal Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) study. METHODS: Scotopic microperimetry (sMP) was performed in one designated study eye in a subset of participants with molecularly proven ABCA4-associated Stargardt disease (STGD1) enrolled in a multicenter natural history study (ProgStar). Study visits were every 6 months over a period ranging from 6 to 24 months, and also included fundus autofluorescence (FAF). RESULTS: SMART enrolled 118 participants (118 eyes). At the first visit of SMART, the mean sensitivity in mesopic microperimetry was 11.48 (±5.05; range 0.00-19.88) dB and in sMP 11.25 (±5.26; 0-19.25) dB. For FAF, all eyes had a lesion of decreased autofluorescence (mean lesion size 3.62 [±3.48; 0.10-21.46] mm2), and a total of 76 eyes (65.5%) had a lesion of definitely decreased autofluorescence with a mean lesion size of 3.46 (±3.60; 0.21-21.46) mm2. CONCLUSIONS: Rod function is impaired in STGD1 and can be assessed by sMP. Testing rod function may serve as a potential outcome measure for future clinical treatment trials. This is evaluated in the SMART study.
PURPOSE: To describe the study design and characteristics at first visit of participants in the longitudinal Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) study. METHODS: Scotopic microperimetry (sMP) was performed in one designated study eye in a subset of participants with molecularly proven ABCA4-associated Stargardt disease (STGD1) enrolled in a multicenter natural history study (ProgStar). Study visits were every 6 months over a period ranging from 6 to 24 months, and also included fundus autofluorescence (FAF). RESULTS: SMART enrolled 118 participants (118 eyes). At the first visit of SMART, the mean sensitivity in mesopic microperimetry was 11.48 (±5.05; range 0.00-19.88) dB and in sMP 11.25 (±5.26; 0-19.25) dB. For FAF, all eyes had a lesion of decreased autofluorescence (mean lesion size 3.62 [±3.48; 0.10-21.46] mm2), and a total of 76 eyes (65.5%) had a lesion of definitely decreased autofluorescence with a mean lesion size of 3.46 (±3.60; 0.21-21.46) mm2. CONCLUSIONS:Rod function is impaired in STGD1 and can be assessed by sMP. Testing rod function may serve as a potential outcome measure for future clinical treatment trials. This is evaluated in the SMART study.
Authors: Talal Alabduljalil; Rachel C Patel; Abdullah A Alqahtani; Simon S Gao; Michael J Gale; Miao Zhang; Yali Jia; David Huang; Pei-Wen Chiang; Rui Chen; Jun Wang; Richard G Weleber; Mark E Pennesi; Paul Yang Journal: Am J Ophthalmol Date: 2019-02-14 Impact factor: 5.258
Authors: Etienne M Schönbach; Rupert W Strauss; Beatriz Muñoz; Yulia Wolfson; Mohamed A Ibrahim; David G Birch; Eberhart Zrenner; Janet S Sunness; Michael S Ip; SriniVas R Sadda; Sheila K West; Hendrik P N Scholl Journal: JAMA Ophthalmol Date: 2020-07-01 Impact factor: 7.389
Authors: Xiangrong Kong; Mohamed Ibrahim-Ahmed; Millena G Bittencourt; Rupert W Strauss; David G Birch; Artur V Cideciyan; Ann-Margaret Ervin; Alexander Ho; Janet S Sunness; Isabelle S Audo; Michel Michaelides; Eberhart Zrenner; SriniVas Sadda; Michael S Ip; Sheila West; Hendrik P N Scholl Journal: Am J Ophthalmol Date: 2021-10-23 Impact factor: 5.258