AUTHORS
Etienne M Schönbach, Lucas Janeschitz-Kriegl, Rupert W Strauss, Marco E G V Cattaneo, Kaoru Fujinami, David G Birch, Artur V Cideciyan, Janet S Sunness, Richard G Weleber, Michael S Ip, SriniVas R Sadda, Hendrik P N Scholl, ProgStar Study Group
Am J Ophthalmol. 2021 Oct;230:123-133. doi: 10.1016/j.ajo.2021.04.015. Epub 2021 May 2.
ABSTRACT
PURPOSE: To report the yearly rate of change in macular function in patients with Stargardt disease type 1 (STGD1) over 24 months and to establish a new volumetric visual function index for use in clinical trials investigating the efficacy on retinal sensitivity.
METHODS: Design: International, multicenter, prospective cohort study with 5 study visits every 6 months over 24 months.
PARTICIPANTS: A total of 233 individuals with genetically confirmed STGD1 (≥1 disease-causing ABCA4 variant).
MAIN OUTCOME MEASURES: The total volume (VTOT) beneath the sensitivity surface of a 3-D model of the hill of vision and mean sensitivity (MS) derived from mesopic microperimetry performed with a white stimulus. Changes of VTOT over time and its correlation with the ABCA4 genotype and baseline features.
RESULTS: At baseline, 440 eyes (233 patients) with a mean (SD) age of 33.7 (15.0) years, mean (SD) visual acuity of 46.08 (16.03) ETDRS letters were analyzed with an average VTOT of 0.91 decibel-steradian (dB-sr) and an MS of 10.73 dB. The overall mean rate of decrease in sensitivity [95% confidence interval] was 0.077 [0.064, 0.090] dB-sr/y for VTOT and 0.87 [0.72, 1.02] dB/year for MS. The progression rate of VTOT depended on baseline visual function (0.029 dB-sr/year for low and 0.120 dB-sr/year for high baseline VTOT; P < .001) and exhibited a difference in the first vs second year of follow-up (0.065 dB-sr/year vs 0.089 dB-sr/year, respectively; P < .001). The absence of pigmentary abnormalities of the retinal pigment epithelium at baseline was found to be associated with a faster progression rate (P < .001), whereas a significant association with the genotype was not detected (P = .7).
CONCLUSION: In STGD1, both microperimetric outcomes demonstrate statistically significant and clinically meaningful changes after relatively short follow-up periods. Volumetric modeling may be useful in future interventional clinical trials that aim to improve retinal sensitivity or to slow down its decline and for structure-function correlations.
PMID:33951446 | DOI:10.1016/j.ajo.2021.04.015
