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 & 30 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, Fiona Boyard, Aurore Girmens

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

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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; Boyard, Fiona; Girmens, Aurore.

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, Boyard, F & Girmens, A 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 ; Boyard, Fiona ; Girmens, Aurore. / 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
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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 - Boyard,Fiona

AU - Girmens,Aurore

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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