How does light sensitivity of the posterior pole change over time in patients with Stargardt disease?
In this cohort study of 359 eyes with ABCA4-related Stargardt disease, microperimetric mean sensitivity (−0.68 dB per year) and deep scotoma points (1.56 points per year) showed a statistically significant and clinically meaningful change after 12 months.
These results suggest that microperimetric mean sensitivity and deep scotoma points may serve as useful end points for clinical trials investigating emerging treatments for Stargardt disease.
Functional end points for clinical trials investigating the efficacy of emerging treatments for Stargardt disease type 1 (STGD1) are needed.
To assess the yearly rate of change of macular function in patients with STGD1 using microperimetry.
Design, Setting, and Participants
This multicenter prospective cohort study was conducted in an international selection of tertiary referral centers from October 21, 2013, to February 15, 2017. The study included participants with ABCA4-related STGD1 who were enrolled in the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study at baseline. Data were analyzed from February 16, 2017, to December 1, 2019.
ABCA4-related STGD1 with a minimum lesion size on fundus autofluorescence and a minimum visual acuity.
Main Outcomes and Measures
Changes in overall macular sensitivity (MS), deep scotoma count, number of points that tested normal, and location-specific sensitivity changes.
Among the 359 eyes from 200 patients (87 [43.5%] men; mean [SD] age, 33.3 [15.2] years) who underwent microperimetry examination graded at baseline and month 12, the mean (SD) yearly change in MS was −0.68 (2.04) dB (95% CI, −0.89 to −0.47 dB; P < .001), and deep scotoma points increased by a mean (SD) of 1.56 (5.74) points per year. The points with sensitivity of 12 dB or higher decreased in sensitivity by a mean (SD) of −3.01 (9.84) dB (95% CI, −4.03 to −1.99 dB; P < .001). The mean (SD) yearly change in MS was not significantly different between the eyes with a grading of good or fair pattern placement at both visits (−0.67 [2.1] dB) and the eyes with a poor pattern placement during at least 1 visit (−0.64 [2.2] dB) (P = .91).
Conclusions and Relevance
This study showed that MS and the number of deep scotoma points had measurably changed after follow-up of approximately 1 year. Microperimetry may serve as a useful functional outcome parameter for clinical trials aimed at slowing the progression of STGD1.
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Schönbach EM, Strauss RW, Muñoz B, et al. Longitudinal Microperimetric Changes of Macular Sensitivity in Stargardt Disease After 12 Months: ProgStar Report No. 13. JAMA Ophthalmol. 2020;138(7):772–779. doi:10.1001/jamaophthalmol.2020.1735
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