1. Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)
- Author
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Xiangrong Kong, Rupert W. Strauss, Michel Michaelides, Artur V. Cideciyan, José-Alain Sahel, Beatriz Muñoz, Sheila West, Hendrik P.N. Scholl, Yulia Wolfson, Millena Bittencourt, Syed Mahmood Shah, Mohammed Ahmed, Etienne Schonbach, Kaoru Fujinami, Elias Traboulsi, Justis Ehlers, Meghan Marino, Susan Crowe, Rachael Briggs, Angela Borer, Anne Pinter, Tami Fecko, Nikki Brugnoni, Janet S. Sunness, Carol Applegate, Leslie Russell, Anthony Moore, Andrew Webster, Sophie Connor, Victoria McCudden, Maria Pefkianaki, Jonathan Aboshiha, Gerald Liew, Graham Holder, Anthony Robson, Alexa King, Daniela Ivanova Cajas Narvaez, Katy Barnard, Catherine Grigg, Hannah Dunbar, Yetunde Obadeyi, Karine Girard-Claudon, Hilary Swann, Avani Rughani, Charles Amoah, Dominic Carrington, Kanom Bibi, Emerson Ting Co, Andrew Carter, Anne Georgiou, Selma Lewis, Saddaf Shaheen, Harpreet Shinmar, Linda Burton, Paul Bernstein, Kimberley Wegner, Briana Lauren Sawyer, Bonnie Carlstrom, Kellian Farnsworth, Cyrie Fry, Melissa Chandler, Glen Jenkins, Donnel Creel, David Birch, Yi-Zhong Wang, Luis Rodriguez, Kirsten Locke, Martin Klein, Paulina Mejia, Samuel G. Jacobson, Sharon B. Schwartz, Rodrigo Matsui, Michaela Gruzensky, Alejandro J. Roman, Eberhart Zrenner, Fadi Nasser, Gesa Astrid Hahn, Barbara Wilhelm, Tobias Peters, Benjamin Beier, Tilman Koenig, Susanne Kramer, Saddek Mohand-Said, Isabelle Audo, Caroline Laurent-Coriat, Ieva Sliesoraityte, Christina Zeitz, Fiona Boyard, Minh Ha Tran, Mathias Chapon, Céline Chaumette, Juliette Amaudruz, Victoria Ganem, Serge Sancho, Aurore Girmens, Robert Wojciechowski, Shazia Khan, David G. Emmert, Dennis Cain, Mark Herring, Jennifer Bassinger, Lisa Liberto, Ann-Margret Ervin, Beatriz Munoz, Kurt Dreger, Jennifer Jones, Srinivas Sadda, Anamika Jha, Alex Ho, Brendan Kramer, Amirhossein Hariri, Gloria Rebecca Blanquel, Ngoc Lam, Sean Pitetta, Yue Shi, Rita Tawdros, Christine Petrossian, Dennis Jenkins, Muneeswar Gupta, Yong Dong Zhou, Katherine Aguilar, Cynthia Chan, Lisa Santos, Brian Seo, Christopher Sison, Silvia Perez, Stephanie Chao, Kelly Miyasato, Julia Higgins, Zoila Luna, Anita Menchaca, Norma Gonzalez, Vicky Robledo, Karen Carig, Kirstie Baker, David Ellenbogen, James Russell, Daniel Bluemel, Alex Moreno, Royal Pham, Theo Sanford, Daisy Linares, and Mei Tran
- Subjects
0301 basic medicine ,Adult ,Male ,Pediatrics ,medicine.medical_specialty ,Visual acuity ,Adolescent ,Cross-sectional study ,Vision Disorders ,Visual Acuity ,03 medical and health sciences ,Macular Degeneration ,Young Adult ,0302 clinical medicine ,Risk Factors ,medicine ,Humans ,Stargardt Disease ,Age of Onset ,Prospective cohort study ,Child ,Generalized estimating equation ,Aged ,Retrospective Studies ,business.industry ,Retrospective cohort study ,Middle Aged ,medicine.disease ,United States ,Stargardt disease ,Europe ,Ophthalmology ,030104 developmental biology ,Cross-Sectional Studies ,030221 ophthalmology & optometry ,Physical therapy ,Disease Progression ,Linear Models ,Age of onset ,medicine.symptom ,business ,Cohort study - Abstract
To examine the association between characteristics of Stargardt disease and visual acuity (VA), to estimate the longitudinal rate of VA loss, and to identify risk factors for VA loss.Retrospective, multicenter cohort study.A total of 176 patients (332 eyes) with molecularly and clinically confirmed Stargardt disease enrolled from the United States and Europe.Standardized data report forms were used to collect retrospective data on participants' characteristics and best-corrected or presenting VA from medical charts. Linear models with generalized estimating equations were used to estimate the cross-sectional associations, and linear mixed effects models were used to estimate the longitudinal VA loss.Yearly change in VA.The median duration of observation was 3.6 years. At baseline, older age of symptom onset was associated with better VA, and a longer duration of symptoms was associated with worse VA. Longitudinal analysis estimated an average of 0.3 lines loss (P0.0001) per year overall, but the rate varied according to baseline VA: (1) eyes with baseline VA ≥20/25 (N = 53) declined at a rate of approximately 1.0 line per year; (2) eyes with VA between 20/25 and 20/70 (N = 65) declined at a rate of approximately 0.9 lines per year; (3) eyes with VA between 20/70 and 20/200 (N = 163) declined at a rate of 0.2 lines per year; and (4) eyes with VA worse than 20/200 (n = 49) improved at a rate of 0.5 lines per year. Older age of onset was associated with slower VA loss: Patients with onset age30 years showed 0.4 lines slower change of VA per year (P = 0.01) compared with patients with onset age ≤14 years.Given the overall slow rate of VA loss, VA is unlikely to be a sensitive outcome measure for treatment trials of Stargardt disease. However, given the faster decline in younger patients and those with no or mild visual impairment, VA may be a potential outcome measure for trials targeting such subgroups of patients. These observations will need to be assessed in a prospective study bearing in mind the inherent limitations of retrospective datasets.
- Published
- 2016