Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)

Xiangrong Kong, Rupert W. Strauss, Beatriz Muñoz, Sheila West, Hendrik P N Scholl, Xiangrong Kong, Rupert W. Strauss, Michel Michaelides, Rupert W. Strauss, Artur V. Cideciyan, José Alain Sahel, Hendrik P N Scholl, Rupert W. Strauss, Yulia Wolfson, Millena Bittencourt, Syed Mahmood Shah, Mohammed Ahmed, Etienne Schonbach, Kaoru Fujinami, Justis Ehlers & 137 others Meghan Marino, Susan Crowe, Rachael Briggs, Angela Borer, Anne Pinter, Tami Fecko, Nikki Brugnoni, Janet S. Sunness, Carol Applegate, Leslie Russell, Michel Michaelides, 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, Artur V. Cideciyan, 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, José Alain Sahel, 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, Hendrik P N Scholl, Rupert W. Strauss, Yulia Wolfson, Syed Mahmood Shah, Mohammed Ahmed, Etienne Schonbach, Robert Wojciechowski, Shazia Khan, David G. Emmert, Dennis Cain, Mark Herring, Jennifer Bassinger, Lisa Liberto, Sheila West, Ann Margret Ervin, Beatriz Munoz, Xiangrong Kong, Kurt Dreger, Jennifer Jones, 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, Mei Tran

Research output: Contribution to journalArticle

Abstract

Purpose 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. Design Retrospective, multicenter cohort study. Participants A total of 176 patients (332 eyes) with molecularly and clinically confirmed Stargardt disease enrolled from the United States and Europe. Methods 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. Main Outcome Measures Yearly change in VA. Results 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 (P < 0.0001) 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 age >30 years showed 0.4 lines slower change of VA per year (P = 0.01) compared with patients with onset age ≤14 years. Conclusions 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.

LanguageEnglish (US)
Pages1887-1897
Number of pages11
JournalOphthalmology
Volume123
Issue number9
DOIs
StatePublished - Sep 1 2016

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Visual Acuity
Age of Onset
Stargardt disease 1
Outcome Assessment (Health Care)
Vision Disorders
Multicenter Studies
Linear Models

ASJC Scopus subject areas

  • Medicine(all)
  • Ophthalmology

Cite this

Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2). / Kong, Xiangrong; Strauss, Rupert W.; Muñoz, Beatriz; West, Sheila; Scholl, Hendrik P N; Kong, Xiangrong; Strauss, Rupert W.; Michaelides, Michel; Strauss, Rupert W.; Cideciyan, Artur V.; Sahel, José Alain; Scholl, Hendrik P N; Strauss, Rupert W.; Wolfson, Yulia; Bittencourt, Millena; Shah, Syed Mahmood; Ahmed, Mohammed; Schonbach, Etienne; Fujinami, Kaoru; Ehlers, Justis; Marino, Meghan; Crowe, Susan; Briggs, Rachael; Borer, Angela; Pinter, Anne; Fecko, Tami; Brugnoni, Nikki; Sunness, Janet S.; Applegate, Carol; Russell, Leslie; Michaelides, Michel; Moore, Anthony; Webster, Andrew; Connor, Sophie; McCudden, Victoria; Pefkianaki, Maria; Aboshiha, Jonathan; Liew, Gerald; Holder, Graham; Robson, Anthony; King, Alexa; Cajas Narvaez, Daniela Ivanova; Barnard, Katy; Grigg, Catherine; Dunbar, Hannah; Obadeyi, Yetunde; Girard-Claudon, Karine; Swann, Hilary; Rughani, Avani; Amoah, Charles; Carrington, Dominic; Bibi, Kanom; Co, Emerson Ting; Carter, Andrew; Georgiou, Anne; Lewis, Selma; Shaheen, Saddaf; Shinmar, Harpreet; Burton, Linda; Bernstein, Paul; Wegner, Kimberley; Sawyer, Briana Lauren; Carlstrom, Bonnie; Farnsworth, Kellian; Fry, Cyrie; Chandler, Melissa; Jenkins, Glen; Creel, Donnel; Birch, David; Wang, Yi Zhong; Rodriguez, Luis; Locke, Kirsten; Klein, Martin; Mejia, Paulina; Cideciyan, Artur V.; Jacobson, Samuel G.; Schwartz, Sharon B.; Matsui, Rodrigo; Gruzensky, Michaela; Roman, Alejandro J.; Zrenner, Eberhart; Nasser, Fadi; Hahn, Gesa Astrid; Wilhelm, Barbara; Peters, Tobias; Beier, Benjamin; Koenig, Tilman; Kramer, Susanne; Sahel, José Alain; Mohand-Said, Saddek; Audo, Isabelle; Laurent-Coriat, Caroline; Sliesoraityte, Ieva; Zeitz, Christina; Boyard, Fiona; Tran, Minh Ha; Chapon, Mathias; Chaumette, Céline; Amaudruz, Juliette; Ganem, Victoria; Sancho, Serge; Girmens, Aurore; Scholl, Hendrik P N; Strauss, Rupert W.; Wolfson, Yulia; Shah, Syed Mahmood; Ahmed, Mohammed; Schonbach, Etienne; Wojciechowski, Robert; Khan, Shazia; Emmert, David G.; Cain, Dennis; Herring, Mark; Bassinger, Jennifer; Liberto, Lisa; West, Sheila; Ervin, Ann Margret; Munoz, Beatriz; Kong, Xiangrong; Dreger, Kurt; Jones, Jennifer; Jha, Anamika; Ho, Alex; Kramer, Brendan; Hariri, Amirhossein; Blanquel, Gloria Rebecca; Lam, Ngoc; Pitetta, Sean; Shi, Yue; Tawdros, Rita; Petrossian, Christine; Jenkins, Dennis; Gupta, Muneeswar; Zhou, Yong Dong; Aguilar, Katherine; Chan, Cynthia; Santos, Lisa; Seo, Brian; Sison, Christopher; Perez, Silvia; Chao, Stephanie; Miyasato, Kelly; Higgins, Julia; Luna, Zoila; Menchaca, Anita; Gonzalez, Norma; Robledo, Vicky; Carig, Karen; Baker, Kirstie; Ellenbogen, David; Russell, James; Bluemel, Daniel; Moreno, Alex; Pham, Royal; Sanford, Theo; Linares, Daisy; Tran, Mei.

In: Ophthalmology, Vol. 123, No. 9, 01.09.2016, p. 1887-1897.

Research output: Contribution to journalArticle

Kong, X, Strauss, RW, Muñoz, B, West, S, Scholl, HPN, Kong, X, Strauss, RW, Michaelides, M, Strauss, RW, Cideciyan, AV, Sahel, JA, Scholl, HPN, Strauss, RW, Wolfson, Y, Bittencourt, M, Shah, SM, Ahmed, M, Schonbach, E, Fujinami, K, Ehlers, J, Marino, M, Crowe, S, Briggs, R, Borer, A, Pinter, A, Fecko, T, Brugnoni, N, Sunness, JS, Applegate, C, Russell, L, Michaelides, M, Moore, A, Webster, A, Connor, S, McCudden, V, Pefkianaki, M, Aboshiha, J, Liew, G, Holder, G, Robson, A, King, A, Cajas Narvaez, DI, Barnard, K, Grigg, C, Dunbar, H, Obadeyi, Y, Girard-Claudon, K, Swann, H, Rughani, A, Amoah, C, Carrington, D, Bibi, K, Co, ET, Carter, A, Georgiou, A, Lewis, S, Shaheen, S, Shinmar, H, Burton, L, Bernstein, P, Wegner, K, Sawyer, BL, Carlstrom, B, Farnsworth, K, Fry, C, Chandler, M, Jenkins, G, Creel, D, Birch, D, Wang, YZ, Rodriguez, L, Locke, K, Klein, M, Mejia, P, Cideciyan, AV, Jacobson, SG, Schwartz, SB, Matsui, R, Gruzensky, M, Roman, AJ, Zrenner, E, Nasser, F, Hahn, GA, Wilhelm, B, Peters, T, Beier, B, Koenig, T, Kramer, S, Sahel, JA, Mohand-Said, S, Audo, I, Laurent-Coriat, C, Sliesoraityte, I, Zeitz, C, Boyard, F, Tran, MH, Chapon, M, Chaumette, C, Amaudruz, J, Ganem, V, Sancho, S, Girmens, A, Scholl, HPN, Strauss, RW, Wolfson, Y, Shah, SM, Ahmed, M, Schonbach, E, Wojciechowski, R, Khan, S, Emmert, DG, Cain, D, Herring, M, Bassinger, J, Liberto, L, West, S, Ervin, AM, Munoz, B, Kong, X, Dreger, K, Jones, J, Jha, A, Ho, A, Kramer, B, Hariri, A, Blanquel, GR, Lam, N, Pitetta, S, Shi, Y, Tawdros, R, Petrossian, C, Jenkins, D, Gupta, M, Zhou, YD, Aguilar, K, Chan, C, Santos, L, Seo, B, Sison, C, Perez, S, Chao, S, Miyasato, K, Higgins, J, Luna, Z, Menchaca, A, Gonzalez, N, Robledo, V, Carig, K, Baker, K, Ellenbogen, D, Russell, J, Bluemel, D, Moreno, A, Pham, R, Sanford, T, Linares, D & Tran, M 2016, 'Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)' Ophthalmology, vol. 123, no. 9, pp. 1887-1897. DOI: 10.1016/j.ophtha.2016.05.027
Kong, Xiangrong ; Strauss, Rupert W. ; Muñoz, Beatriz ; West, Sheila ; Scholl, Hendrik P N ; Kong, Xiangrong ; Strauss, Rupert W. ; Michaelides, Michel ; Strauss, Rupert W. ; Cideciyan, Artur V. ; Sahel, José Alain ; Scholl, Hendrik P N ; Strauss, Rupert W. ; Wolfson, Yulia ; Bittencourt, Millena ; Shah, Syed Mahmood ; Ahmed, Mohammed ; Schonbach, Etienne ; Fujinami, Kaoru ; Ehlers, Justis ; Marino, Meghan ; Crowe, Susan ; Briggs, Rachael ; Borer, Angela ; Pinter, Anne ; Fecko, Tami ; Brugnoni, Nikki ; Sunness, Janet S. ; Applegate, Carol ; Russell, Leslie ; Michaelides, Michel ; Moore, Anthony ; Webster, Andrew ; Connor, Sophie ; McCudden, Victoria ; Pefkianaki, Maria ; Aboshiha, Jonathan ; Liew, Gerald ; Holder, Graham ; Robson, Anthony ; King, Alexa ; Cajas Narvaez, Daniela Ivanova ; Barnard, Katy ; Grigg, Catherine ; Dunbar, Hannah ; Obadeyi, Yetunde ; Girard-Claudon, Karine ; Swann, Hilary ; Rughani, Avani ; Amoah, Charles ; Carrington, Dominic ; Bibi, Kanom ; Co, Emerson Ting ; Carter, Andrew ; Georgiou, Anne ; Lewis, Selma ; Shaheen, Saddaf ; Shinmar, Harpreet ; Burton, Linda ; Bernstein, Paul ; Wegner, Kimberley ; Sawyer, Briana Lauren ; Carlstrom, Bonnie ; Farnsworth, Kellian ; Fry, Cyrie ; Chandler, Melissa ; Jenkins, Glen ; Creel, Donnel ; Birch, David ; Wang, Yi Zhong ; Rodriguez, Luis ; Locke, Kirsten ; Klein, Martin ; Mejia, Paulina ; Cideciyan, Artur V. ; Jacobson, Samuel G. ; Schwartz, Sharon B. ; Matsui, Rodrigo ; Gruzensky, Michaela ; Roman, Alejandro J. ; Zrenner, Eberhart ; Nasser, Fadi ; Hahn, Gesa Astrid ; Wilhelm, Barbara ; Peters, Tobias ; Beier, Benjamin ; Koenig, Tilman ; Kramer, Susanne ; Sahel, José Alain ; Mohand-Said, Saddek ; Audo, Isabelle ; Laurent-Coriat, Caroline ; Sliesoraityte, Ieva ; Zeitz, Christina ; Boyard, Fiona ; Tran, Minh Ha ; Chapon, Mathias ; Chaumette, Céline ; Amaudruz, Juliette ; Ganem, Victoria ; Sancho, Serge ; Girmens, Aurore ; Scholl, Hendrik P N ; Strauss, Rupert W. ; Wolfson, Yulia ; Shah, Syed Mahmood ; Ahmed, Mohammed ; Schonbach, Etienne ; Wojciechowski, Robert ; Khan, Shazia ; Emmert, David G. ; Cain, Dennis ; Herring, Mark ; Bassinger, Jennifer ; Liberto, Lisa ; West, Sheila ; Ervin, Ann Margret ; Munoz, Beatriz ; Kong, Xiangrong ; Dreger, Kurt ; Jones, Jennifer ; Jha, Anamika ; Ho, Alex ; Kramer, Brendan ; Hariri, Amirhossein ; Blanquel, Gloria Rebecca ; Lam, Ngoc ; Pitetta, Sean ; Shi, Yue ; Tawdros, Rita ; Petrossian, Christine ; Jenkins, Dennis ; Gupta, Muneeswar ; Zhou, Yong Dong ; Aguilar, Katherine ; Chan, Cynthia ; Santos, Lisa ; Seo, Brian ; Sison, Christopher ; Perez, Silvia ; Chao, Stephanie ; Miyasato, Kelly ; Higgins, Julia ; Luna, Zoila ; Menchaca, Anita ; Gonzalez, Norma ; Robledo, Vicky ; Carig, Karen ; Baker, Kirstie ; Ellenbogen, David ; Russell, James ; Bluemel, Daniel ; Moreno, Alex ; Pham, Royal ; Sanford, Theo ; Linares, Daisy ; Tran, Mei. / Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2). In: Ophthalmology. 2016 ; Vol. 123, No. 9. pp. 1887-1897
@article{e969dc2b198841999203fe0517144327,
title = "Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)",
abstract = "Purpose 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. Design Retrospective, multicenter cohort study. Participants A total of 176 patients (332 eyes) with molecularly and clinically confirmed Stargardt disease enrolled from the United States and Europe. Methods 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. Main Outcome Measures Yearly change in VA. Results 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 (P < 0.0001) 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 age >30 years showed 0.4 lines slower change of VA per year (P = 0.01) compared with patients with onset age ≤14 years. Conclusions 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.",
author = "Xiangrong Kong and Strauss, {Rupert W.} and Beatriz Mu{\~n}oz and Sheila West and Scholl, {Hendrik P N} and Xiangrong Kong and Strauss, {Rupert W.} and Michel Michaelides and Strauss, {Rupert W.} and Cideciyan, {Artur V.} and Sahel, {Jos{\'e} Alain} and Scholl, {Hendrik P N} and Strauss, {Rupert W.} and Yulia Wolfson and Millena Bittencourt and Shah, {Syed Mahmood} and Mohammed Ahmed and Etienne Schonbach and Kaoru Fujinami and Justis Ehlers and Meghan Marino and Susan Crowe and Rachael Briggs and Angela Borer and Anne Pinter and Tami Fecko and Nikki Brugnoni and Sunness, {Janet S.} and Carol Applegate and Leslie Russell and Michel Michaelides and Anthony Moore and Andrew Webster and Sophie Connor and Victoria McCudden and Maria Pefkianaki and Jonathan Aboshiha and Gerald Liew and Graham Holder and Anthony Robson and Alexa King and {Cajas Narvaez}, {Daniela Ivanova} and Katy Barnard and Catherine Grigg and Hannah Dunbar and Yetunde Obadeyi and Karine Girard-Claudon and Hilary Swann and Avani Rughani and Charles Amoah and Dominic Carrington and Kanom Bibi and Co, {Emerson Ting} and Andrew Carter and Anne Georgiou and Selma Lewis and Saddaf Shaheen and Harpreet Shinmar and Linda Burton and Paul Bernstein and Kimberley Wegner and Sawyer, {Briana Lauren} and Bonnie Carlstrom and Kellian Farnsworth and Cyrie Fry and Melissa Chandler and Glen Jenkins and Donnel Creel and David Birch and Wang, {Yi Zhong} and Luis Rodriguez and Kirsten Locke and Martin Klein and Paulina Mejia and Cideciyan, {Artur V.} and Jacobson, {Samuel G.} and Schwartz, {Sharon B.} and Rodrigo Matsui and Michaela Gruzensky and Roman, {Alejandro J.} and Eberhart Zrenner and Fadi Nasser and Hahn, {Gesa Astrid} and Barbara Wilhelm and Tobias Peters and Benjamin Beier and Tilman Koenig and Susanne Kramer and Sahel, {Jos{\'e} Alain} and Saddek Mohand-Said and Isabelle Audo and Caroline Laurent-Coriat and Ieva Sliesoraityte and Christina Zeitz and Fiona Boyard and Tran, {Minh Ha} and Mathias Chapon and C{\'e}line Chaumette and Juliette Amaudruz and Victoria Ganem and Serge Sancho and Aurore Girmens and Scholl, {Hendrik P N} and Strauss, {Rupert W.} and Yulia Wolfson and Shah, {Syed Mahmood} and Mohammed Ahmed and Etienne Schonbach and Robert Wojciechowski and Shazia Khan and Emmert, {David G.} and Dennis Cain and Mark Herring and Jennifer Bassinger and Lisa Liberto and Sheila West and Ervin, {Ann Margret} and Beatriz Munoz and Xiangrong Kong and Kurt Dreger and Jennifer Jones and Anamika Jha and Alex Ho and Brendan Kramer and Amirhossein Hariri and Blanquel, {Gloria Rebecca} and Ngoc Lam and Sean Pitetta and Yue Shi and Rita Tawdros and Christine Petrossian and Dennis Jenkins and Muneeswar Gupta and Zhou, {Yong Dong} and Katherine Aguilar and Cynthia Chan and Lisa Santos and Brian Seo and Christopher Sison and Silvia Perez and Stephanie Chao and Kelly Miyasato and Julia Higgins and Zoila Luna and Anita Menchaca and Norma Gonzalez and Vicky Robledo and Karen Carig and Kirstie Baker and David Ellenbogen and James Russell and Daniel Bluemel and Alex Moreno and Royal Pham and Theo Sanford and Daisy Linares and Mei Tran",
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doi = "10.1016/j.ophtha.2016.05.027",
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pages = "1887--1897",
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TY - JOUR

T1 - Visual Acuity Loss and Associated Risk Factors in the Retrospective Progression of Stargardt Disease Study (ProgStar Report No. 2)

AU - Kong,Xiangrong

AU - Strauss,Rupert W.

AU - Muñoz,Beatriz

AU - West,Sheila

AU - Scholl,Hendrik P N

AU - Kong,Xiangrong

AU - Strauss,Rupert W.

AU - Michaelides,Michel

AU - Strauss,Rupert W.

AU - Cideciyan,Artur V.

AU - Sahel,José Alain

AU - Scholl,Hendrik P N

AU - Strauss,Rupert W.

AU - Wolfson,Yulia

AU - Bittencourt,Millena

AU - Shah,Syed Mahmood

AU - Ahmed,Mohammed

AU - Schonbach,Etienne

AU - Fujinami,Kaoru

AU - Ehlers,Justis

AU - Marino,Meghan

AU - Crowe,Susan

AU - Briggs,Rachael

AU - Borer,Angela

AU - Pinter,Anne

AU - Fecko,Tami

AU - Brugnoni,Nikki

AU - Sunness,Janet S.

AU - Applegate,Carol

AU - Russell,Leslie

AU - Michaelides,Michel

AU - Moore,Anthony

AU - Webster,Andrew

AU - Connor,Sophie

AU - McCudden,Victoria

AU - Pefkianaki,Maria

AU - Aboshiha,Jonathan

AU - Liew,Gerald

AU - Holder,Graham

AU - Robson,Anthony

AU - King,Alexa

AU - Cajas Narvaez,Daniela Ivanova

AU - Barnard,Katy

AU - Grigg,Catherine

AU - Dunbar,Hannah

AU - Obadeyi,Yetunde

AU - Girard-Claudon,Karine

AU - Swann,Hilary

AU - Rughani,Avani

AU - Amoah,Charles

AU - Carrington,Dominic

AU - Bibi,Kanom

AU - Co,Emerson Ting

AU - Carter,Andrew

AU - Georgiou,Anne

AU - Lewis,Selma

AU - Shaheen,Saddaf

AU - Shinmar,Harpreet

AU - Burton,Linda

AU - Bernstein,Paul

AU - Wegner,Kimberley

AU - Sawyer,Briana Lauren

AU - Carlstrom,Bonnie

AU - Farnsworth,Kellian

AU - Fry,Cyrie

AU - Chandler,Melissa

AU - Jenkins,Glen

AU - Creel,Donnel

AU - Birch,David

AU - Wang,Yi Zhong

AU - Rodriguez,Luis

AU - Locke,Kirsten

AU - Klein,Martin

AU - Mejia,Paulina

AU - Cideciyan,Artur V.

AU - Jacobson,Samuel G.

AU - Schwartz,Sharon B.

AU - Matsui,Rodrigo

AU - Gruzensky,Michaela

AU - Roman,Alejandro J.

AU - Zrenner,Eberhart

AU - Nasser,Fadi

AU - Hahn,Gesa Astrid

AU - Wilhelm,Barbara

AU - Peters,Tobias

AU - Beier,Benjamin

AU - Koenig,Tilman

AU - Kramer,Susanne

AU - Sahel,José Alain

AU - Mohand-Said,Saddek

AU - Audo,Isabelle

AU - Laurent-Coriat,Caroline

AU - Sliesoraityte,Ieva

AU - Zeitz,Christina

AU - Boyard,Fiona

AU - Tran,Minh Ha

AU - Chapon,Mathias

AU - Chaumette,Céline

AU - Amaudruz,Juliette

AU - Ganem,Victoria

AU - Sancho,Serge

AU - Girmens,Aurore

AU - Scholl,Hendrik P N

AU - Strauss,Rupert W.

AU - Wolfson,Yulia

AU - Shah,Syed Mahmood

AU - Ahmed,Mohammed

AU - Schonbach,Etienne

AU - Wojciechowski,Robert

AU - Khan,Shazia

AU - Emmert,David G.

AU - Cain,Dennis

AU - Herring,Mark

AU - Bassinger,Jennifer

AU - Liberto,Lisa

AU - West,Sheila

AU - Ervin,Ann Margret

AU - Munoz,Beatriz

AU - Kong,Xiangrong

AU - Dreger,Kurt

AU - Jones,Jennifer

AU - Jha,Anamika

AU - Ho,Alex

AU - Kramer,Brendan

AU - Hariri,Amirhossein

AU - Blanquel,Gloria Rebecca

AU - Lam,Ngoc

AU - Pitetta,Sean

AU - Shi,Yue

AU - Tawdros,Rita

AU - Petrossian,Christine

AU - Jenkins,Dennis

AU - Gupta,Muneeswar

AU - Zhou,Yong Dong

AU - Aguilar,Katherine

AU - Chan,Cynthia

AU - Santos,Lisa

AU - Seo,Brian

AU - Sison,Christopher

AU - Perez,Silvia

AU - Chao,Stephanie

AU - Miyasato,Kelly

AU - Higgins,Julia

AU - Luna,Zoila

AU - Menchaca,Anita

AU - Gonzalez,Norma

AU - Robledo,Vicky

AU - Carig,Karen

AU - Baker,Kirstie

AU - Ellenbogen,David

AU - Russell,James

AU - Bluemel,Daniel

AU - Moreno,Alex

AU - Pham,Royal

AU - Sanford,Theo

AU - Linares,Daisy

AU - Tran,Mei

PY - 2016/9/1

Y1 - 2016/9/1

N2 - Purpose 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. Design Retrospective, multicenter cohort study. Participants A total of 176 patients (332 eyes) with molecularly and clinically confirmed Stargardt disease enrolled from the United States and Europe. Methods 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. Main Outcome Measures Yearly change in VA. Results 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 (P < 0.0001) 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 age >30 years showed 0.4 lines slower change of VA per year (P = 0.01) compared with patients with onset age ≤14 years. Conclusions 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.

AB - Purpose 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. Design Retrospective, multicenter cohort study. Participants A total of 176 patients (332 eyes) with molecularly and clinically confirmed Stargardt disease enrolled from the United States and Europe. Methods 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. Main Outcome Measures Yearly change in VA. Results 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 (P < 0.0001) 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 age >30 years showed 0.4 lines slower change of VA per year (P = 0.01) compared with patients with onset age ≤14 years. Conclusions 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.

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U2 - 10.1016/j.ophtha.2016.05.027

DO - 10.1016/j.ophtha.2016.05.027

M3 - Article

VL - 123

SP - 1887

EP - 1897

JO - Ophthalmology

T2 - Ophthalmology

JF - Ophthalmology

SN - 0161-6420

IS - 9

ER -