Etienne M Schönbach1,2, Rupert W Strauss1,3,4,5, Beatriz Muñoz1, Yulia Wolfson1, Mohamed A Ibrahim1,6, David G Birch7, Eberhart Zrenner8, Janet S Sunness9, Michael S Ip10,11, SriniVas R Sadda10,11, Sheila K West1, Hendrik P N Scholl1,12,13. 1. Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland. 2. University Hospitals Eye Institute, Case Western Reserve University, Cleveland, Ohio. 3. Moorfields Eye Hospital, London, United Kingdom. 4. Department of Ophthalmology, Johannes Kepler University, Linz, Austria. 5. Department of Ophthalmology, Medical University, Graz, Austria. 6. Byers Eye Institute, Stanford University School of Medicine, Palo Alto, California. 7. Retina Foundation of the Southwest, Dallas, Texas. 8. Center for Ophthalmology, Eberhard Karls Universität, Tübingen, Germany. 9. Hoover Low Vision Rehabilitation Services, Greater Baltimore Medical Center, Baltimore, Maryland. 10. Doheny Eye Institute, Los Angeles, California. 11. UCLA (University of California, Los Angeles) David Geffen School of Medicine. 12. Department of Ophthalmology, University of Basel, Switzerland. 13. Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland.
Abstract
Importance: Functional end points for clinical trials investigating the efficacy of emerging treatments for Stargardt disease type 1 (STGD1) are needed. Objective: To assess the yearly rate of change of macular function in patients with STGD1 using microperimetry. Design, Setting, and Participants: This multicenter prospective cohort study was conducted in an international selection of tertiary referral centers from October 21, 2013, to February 15, 2017. The study included participants with ABCA4-related STGD1 who were enrolled in the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study at baseline. Data were analyzed from February 16, 2017, to December 1, 2019. Exposure: ABCA4-related STGD1 with a minimum lesion size on fundus autofluorescence and a minimum visual acuity. Main Outcomes and Measures: Changes in overall macular sensitivity (MS), deep scotoma count, number of points that tested normal, and location-specific sensitivity changes. Results: Among the 359 eyes from 200 patients (87 [43.5%] men; mean [SD] age, 33.3 [15.2] years) who underwent microperimetry examination graded at baseline and month 12, the mean (SD) yearly change in MS was -0.68 (2.04) dB (95% CI, -0.89 to -0.47 dB; P < .001), and deep scotoma points increased by a mean (SD) of 1.56 (5.74) points per year. The points with sensitivity of 12 dB or higher decreased in sensitivity by a mean (SD) of -3.01 (9.84) dB (95% CI, -4.03 to -1.99 dB; P < .001). The mean (SD) yearly change in MS was not significantly different between the eyes with a grading of good or fair pattern placement at both visits (-0.67 [2.1] dB) and the eyes with a poor pattern placement during at least 1 visit (-0.64 [2.2] dB) (P = .91). Conclusions and Relevance: This study showed that MS and the number of deep scotoma points had measurably changed after follow-up of approximately 1 year. Microperimetry may serve as a useful functional outcome parameter for clinical trials aimed at slowing the progression of STGD1.
Importance: Functional end points for clinical trials investigating the efficacy of emerging treatments for Stargardt disease type 1 (STGD1) are needed. Objective: To assess the yearly rate of change of macular function in patients with STGD1 using microperimetry. Design, Setting, and Participants: This multicenter prospective cohort study was conducted in an international selection of tertiary referral centers from October 21, 2013, to February 15, 2017. The study included participants with ABCA4-related STGD1 who were enrolled in the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study at baseline. Data were analyzed from February 16, 2017, to December 1, 2019. Exposure: ABCA4-related STGD1 with a minimum lesion size on fundus autofluorescence and a minimum visual acuity. Main Outcomes and Measures: Changes in overall macular sensitivity (MS), deep scotoma count, number of points that tested normal, and location-specific sensitivity changes. Results: Among the 359 eyes from 200 patients (87 [43.5%] men; mean [SD] age, 33.3 [15.2] years) who underwent microperimetry examination graded at baseline and month 12, the mean (SD) yearly change in MS was -0.68 (2.04) dB (95% CI, -0.89 to -0.47 dB; P < .001), and deep scotoma points increased by a mean (SD) of 1.56 (5.74) points per year. The points with sensitivity of 12 dB or higher decreased in sensitivity by a mean (SD) of -3.01 (9.84) dB (95% CI, -4.03 to -1.99 dB; P < .001). The mean (SD) yearly change in MS was not significantly different between the eyes with a grading of good or fair pattern placement at both visits (-0.67 [2.1] dB) and the eyes with a poor pattern placement during at least 1 visit (-0.64 [2.2] dB) (P = .91). Conclusions and Relevance: This study showed that MS and the number of deep scotoma points had measurably changed after follow-up of approximately 1 year. Microperimetry may serve as a useful functional outcome parameter for clinical trials aimed at slowing the progression of STGD1.
Authors: Hendrik P N Scholl; Rupert W Strauss; Mandeep S Singh; Deniz Dalkara; Botond Roska; Serge Picaud; José-Alain Sahel Journal: Sci Transl Med Date: 2016-12-07 Impact factor: 17.956
Authors: Artur V Cideciyan; Malgorzata Swider; Tomas S Aleman; Willam J Feuer; Sharon B Schwartz; Robert C Russell; Janet D Steinberg; Edwin M Stone; Samuel G Jacobson Journal: Invest Ophthalmol Vis Sci Date: 2012-02-21 Impact factor: 4.799
Authors: Xiangrong Kong; Rupert W Strauss; Artur V Cideciyan; Michel Michaelides; José-Alain Sahel; Beatriz Munoz; Mohamed Ahmed; Ann M Ervin; Sheila K West; Janet K Cheetham; Hendrik P N Scholl Journal: Ophthalmology Date: 2017-05-23 Impact factor: 12.079
Authors: Xiangrong Kong; Rupert W Strauss; Michel Michaelides; Artur V Cideciyan; José-Alain Sahel; Beatriz Muñoz; Sheila West; Hendrik P N Scholl Journal: Ophthalmology Date: 2016-07-02 Impact factor: 12.079
Authors: Etienne M Schönbach; Rupert W Strauss; Xiangrong Kong; Beatriz Muñoz; Mohamed A Ibrahim; Janet S Sunness; David G Birch; Gesa-Astrid Hahn; Fadi Nasser; Eberhart Zrenner; SriniVas R Sadda; Sheila K West; Hendrik P N Scholl Journal: Am J Ophthalmol Date: 2018-06-08 Impact factor: 5.258
Authors: Kaoru Fujinami; Rupert W Strauss; John Pei-Wen Chiang; Isabelle S Audo; Paul S Bernstein; David G Birch; Samantha M Bomotti; Artur V Cideciyan; Ann-Margret Ervin; Meghan J Marino; José-Alain Sahel; Saddek Mohand-Said; Janet S Sunness; Elias I Traboulsi; Sheila West; Robert Wojciechowski; Eberhart Zrenner; Michel Michaelides; Hendrik P N Scholl Journal: Br J Ophthalmol Date: 2018-06-20 Impact factor: 4.638
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
Authors: Todd A Durham; Jacque L Duncan; Allison R Ayala; David G Birch; Janet K Cheetham; Frederick L Ferris; Carel B Hoyng; Mark E Pennesi; José-Alain Sahel Journal: Transl Vis Sci Technol Date: 2021-04-01 Impact factor: 3.048
Authors: Patty P A Dhooge; Esmee H Runhart; Stanley Lambertus; Nathalie M Bax; Johannes M M Groenewoud; B Jeroen Klevering; Carel B Hoyng Journal: Transl Vis Sci Technol Date: 2021-03-01 Impact factor: 3.283