Visual Acuity Change over 12 Months in the Prospective Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: ProgStar Report Number 6

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; ProgStar Study Group

doi:10.1016/j.ophtha.2017.04.026

Visual Acuity Change over 12 Months in the Prospective Progression of Atrophy Secondary to Stargardt Disease (ProgStar) Study: ProgStar Report Number 6

Ophthalmology. 2017 Nov;124(11):1640-1651. doi: 10.1016/j.ophtha.2017.04.026. Epub 2017 May 23.

Authors

Affiliations

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.

PMID: 28549516
DOI: 10.1016/j.ophtha.2017.04.026

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.

Trial registration: ClinicalTrials.gov NCT01977846.

Publication types

Multicenter Study

MeSH terms

Adolescent
Adult
Aged
Atrophy
Child
Cohort Studies
Cross-Sectional Studies
Disease Progression
Female
Follow-Up Studies
Humans
Macular Degeneration / complications
Macular Degeneration / congenital*
Macular Degeneration / diagnosis
Macular Degeneration / physiopathology
Male
Middle Aged
Prospective Studies
Retina / pathology*
Risk Factors
Stargardt Disease
Vision Disorders / diagnosis
Vision Disorders / etiology
Vision Disorders / physiopathology*
Visual Acuity / physiology*
Young Adult

Associated data

ClinicalTrials.gov/NCT01977846