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

Abstract

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.

Correlation of Outer Retinal Degeneration and Choriocapillaris Loss in Stargardt Disease Using En Face Optical Coherence Tomography and Optical Coherence Tomography Angiography

“…Our results are in contradiction to the findings of de Carlo and colleagues, but indirectly supported by the microperimetry studies of Strauss, et al, showing extra-lesional areas of decreased cone and rod-dependent sensitivity. 48 Taken together with the findings of Strauss and colleagues, our results suggest that relative photoreceptor dysfunction with mild choriocapillaris attenuation may precede lesion expansion in STGD.…”

The Progression of the Stargardt Disease Type 4 (ProgStar-4) Study: Design and Baseline Characteristics (ProgStar-4 Report No. 1)

“…Moreover, clinical trials that aim to slow progression and/or to restore vision require validated outcome measures to prove treatment efficacy. Such an endeavor has been undertaken for ABCA4-related retinopathy in the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies; these consist of a retrospective chart review (ProgStar-1), a prospective cohort study (ProgStar-2), and an ancillary study evaluating scotopic microperimetry (MP; scotopic assessment of rod function in Stargardt disease SMART) [9,11]. In keeping with these studies, the "Natural History of the Progression of Atrophy Secondary to Stargardt Disease Type 4 (STGD4): A Prospective Longitudinal Observational Study of Stargardt Disease Type 4, PROM1-Related Dystrophy" (ProgStar-4 study) has been launched in order to determine the natural history of PROM1-related retinopathy.…”

Macular dystrophies: clinical and imaging features, molecular genetics and therapeutic options

“…14 15 In addition to ERG, function has been shown to be impaired with scotopic microperimetry. 16 Retinal imaging…”