Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) Study: Design and Baseline Characteristics (Report No. 1).

Strauss, Rupert W.; Kong, Xiangrong; Bittencourt, Millena G.; Ho, Alexander; Jha, Anamika; Schönbach, Etienne M.; Ahmed, Mohamed I.; Muñoz, Beatriz; Ervin, Ann-Margret; Michaelides, Michel; Birch, David G.; Sahel, José-Alain; Sunness, Janet S.; Zrenner, Eberhart; Bagheri, Saghar; Ip, Michael; Sadda, SriniVas; West, Sheila; Scholl, Hendrik P.N.

Purpose: To describe the study design and characteristics at first visit of participants in the longitudinal Scotopic Microperimetric Assessment of Rod Function in Stargardt Disease (SMART) study. Methods: Scotopic microperimetry (sMP) was performed in one designated study eye in a subset of participants with molecularly proven ABCA4-associated Stargardt disease (STGD1) enrolled in a multicenter natural history study (ProgStar). Study visits were every 6 months over a period ranging from 6 to 24 months, and also included fundus autofluorescence (FAF). Results: SMART enrolled 118 participants (118 eyes). At the first visit of SMART, the mean sensitivity in mesopic microperimetry was 11.48 (±5.05; range 0.00–19.88) dB and in sMP 11.25 (±5.26; 0–19.25) dB. For FAF, all eyes had a lesion of decreased autofluorescence (mean lesion size 3.62 [±3.48; 0.10–21.46] mm2), and a total of 76 eyes (65.5%) had a lesion of definitely decreased autofluorescence with a mean lesion size of 3.46 (±3.60; 0.21–21.46) mm2. Conclusions: Rod function is impaired in STGD1 and can be assessed by sMP. Testing rod function may serve as a potential outcome measure for future clinical treatment trials. This is evaluated in the SMART study.

BIOFLUORESCENCE; NATURAL history; CLINICAL trials; EYE

Ophthalmic Research, 2019, Vol 61, Issue 1, p36

0030-3747

Academic Journal

10.1159/000488711