Xiangrong Kong1, Rupert W Strauss2, Artur V Cideciyan3, Michel Michaelides4, José-Alain Sahel5, Beatriz Munoz6, Mohamed Ahmed6, Ann M Ervin7, Sheila K West6, Janet K Cheetham8, Hendrik P N Scholl9. 1. School of Public Health and Health Sciences, University of Massachusetts-Amherst, Amherst, Massachusetts; Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland; Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland. 2. Department of Ophthalmology, Johannes Kepler University Linz, Linz, Austria; Department of Ophthalmology, Moorfields Eye Hospital, London, United Kingdom; UCL Institute of Ophthalmology, University College London, London, United Kingdom; Department of Ophthalmology, University of Basel, Basel, Switzerland. 3. Scheie Eye Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania. 4. Department of Ophthalmology, Moorfields Eye Hospital, London, United Kingdom; UCL Institute of Ophthalmology, University College London, London, United Kingdom. 5. Brain Institute, University of Pittsburgh, Pittsburgh, Pennsylvania. 6. Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland. 7. Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland; Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland. 8. Foundation Fighting Blindness, Columbia, Maryland. 9. Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland; Department of Ophthalmology, University of Basel, Basel, Switzerland. Electronic address: Hendrik.Scholl@usb.ch.
Abstract
PURPOSE: To estimate the yearly rate of change of best-corrected visual acuity (BCVA) and the risk of loss 1 line or more over 1 year and to identify risk factors for BCVA loss in patients with Stargardt disease (STGD1). DESIGN: Multicenter, prospective cohort study. PARTICIPANTS: Two hundred fifty-nine patients (489 eyes) with molecularly confirmed STGD1 enrolled at 9 centers in the United States and Europe. METHODS: Participants were followed up every 6 months, and data at the baseline and 6- and 12-month visits were analyzed. Best-corrected visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Standardized reporting forms were used to collect participants' characteristics and clinical observations. Linear mixed effects models were used to estimate the rate of BCVA loss. Linear models with generalized estimating equations were used to identify risk factors for BCVA loss of 1 line or more over 1 year. MAIN OUTCOME MEASURES: Change in BCVA over 1 year. RESULTS: Cross-sectional analysis at baseline showed that earlier symptom onset and longer duration since onset was associated with worse BCVA. Longitudinal analysis showed no overall significant change of BCVA within 12 months, but the rate of BCVA change was significantly different by baseline BCVA (P < 0.001). The BCVA of eyes with baseline BCVA of 20/25 or better declined at a rate of 2.8 ETDRS letters per year (P = 0.10), eyes with baseline BCVA between 20/25 and 20/70 declined at a rate of 2.3 ETDRS letters per year (P = 0.002), eyes with baseline BCVA between 20/70 and 20/200 declined at a rate of 0.8 ETDRS letters per year (P = 0.08), and eyes with baseline BCVA worse than 20/200 showed a significant improvement of 2.3 ETDRS letters per year (P < 0.001). Overall, 12.9% of eyes lost 1 line or more, and the risk of such BCVA loss was different by baseline BCVA level (P = 0.016). Smoking and vitamin A use was not associated significantly with baseline BCVA, nor with rate of BCVA loss over 1 year. CONCLUSIONS: Change in BCVA in STGD1 patients over a 12-month period was small, but varied depending on baseline BCVA. Given the slow change during 1 year, BCVA is unlikely to be a sensitive outcome measure for STGD1 treatment trials with 1 year's duration.
PURPOSE: To estimate the yearly rate of change of best-corrected visual acuity (BCVA) and the risk of loss 1 line or more over 1 year and to identify risk factors for BCVA loss in patients with Stargardt disease (STGD1). DESIGN: Multicenter, prospective cohort study. PARTICIPANTS: Two hundred fifty-nine patients (489 eyes) with molecularly confirmed STGD1 enrolled at 9 centers in the United States and Europe. METHODS:Participants were followed up every 6 months, and data at the baseline and 6- and 12-month visits were analyzed. Best-corrected visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Standardized reporting forms were used to collect participants' characteristics and clinical observations. Linear mixed effects models were used to estimate the rate of BCVA loss. Linear models with generalized estimating equations were used to identify risk factors for BCVA loss of 1 line or more over 1 year. MAIN OUTCOME MEASURES: Change in BCVA over 1 year. RESULTS: Cross-sectional analysis at baseline showed that earlier symptom onset and longer duration since onset was associated with worse BCVA. Longitudinal analysis showed no overall significant change of BCVA within 12 months, but the rate of BCVA change was significantly different by baseline BCVA (P < 0.001). The BCVA of eyes with baseline BCVA of 20/25 or better declined at a rate of 2.8 ETDRS letters per year (P = 0.10), eyes with baseline BCVA between 20/25 and 20/70 declined at a rate of 2.3 ETDRS letters per year (P = 0.002), eyes with baseline BCVA between 20/70 and 20/200 declined at a rate of 0.8 ETDRS letters per year (P = 0.08), and eyes with baseline BCVA worse than 20/200 showed a significant improvement of 2.3 ETDRS letters per year (P < 0.001). Overall, 12.9% of eyes lost 1 line or more, and the risk of such BCVA loss was different by baseline BCVA level (P = 0.016). Smoking and vitamin A use was not associated significantly with baseline BCVA, nor with rate of BCVA loss over 1 year. CONCLUSIONS: Change in BCVA in STGD1patients over a 12-month period was small, but varied depending on baseline BCVA. Given the slow change during 1 year, BCVA is unlikely to be a sensitive outcome measure for STGD1 treatment trials with 1 year's duration.
Authors: Maria Fernanda Abalem; Benjamin Otte; Chris Andrews; Katherine A Joltikov; Kari Branham; Abigail T Fahim; Dana Schlegel; Cynthia X Qian; John R Heckenlively; Thiran Jayasundera Journal: Am J Ophthalmol Date: 2017-10-14 Impact factor: 5.258
Authors: Maria A Parker; Laura R Erker; Isabelle Audo; Dongseok Choi; Saddek Mohand-Said; Kastytis Sestakauskas; Patrick Benoit; Terence Appelqvist; Melissa Krahmer; Caroline Ségaut-Prévost; Brandon J Lujan; Ambar Faridi; Elvira N Chegarnov; Peter N Steinkamp; Cristy Ku; Mariana Matioli da Palma; Pierre-Olivier Barale; Sarah Ayelo-Scheer; Andreas Lauer; Tim Stout; David J Wilson; Richard G Weleber; Mark E Pennesi; José Alain Sahel; Paul Yang Journal: Am J Ophthalmol Date: 2022-03-04 Impact factor: 5.488
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; Kaoru Fujinami; Rupert W Strauss; Beatriz Munoz; Sheila K West; Artur V Cideciyan; Michel Michaelides; Mohamed Ahmed; Ann-Margret Ervin; Etienne Schönbach; Janet K Cheetham; Hendrik P N Scholl Journal: JAMA Ophthalmol Date: 2018-08-01 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
Authors: Jin Kyun Oh; Sarah R Levi; Joonpyo Kim; Jose Ronaldo Lima de Carvalho; Joseph Ryu; Janet R Sparrow; Stephen H Tsang Journal: Am J Ophthalmol Date: 2020-05-20 Impact factor: 5.488
Authors: Jacque L Duncan; Eric A Pierce; Amy M Laster; Stephen P Daiger; David G Birch; John D Ash; Alessandro Iannaccone; John G Flannery; José A Sahel; Donald J Zack; Marco A Zarbin Journal: Transl Vis Sci Technol Date: 2018-07-18 Impact factor: 3.283