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
Wilmer Eye Institute, Johns Hopkins University, Baltimore, Maryland, USA.
Moorfields Eye Hospital NHS Foundation Trust, and UCL Institute of Ophthalmology, University College London, London, United Kingdom.
Ophthalmic Res. 2019;61(1):36-43. doi: 10.1159/000488711. Epub 2018 Jun 25.
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.
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).
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.
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.
描述斯塔加特病视杆细胞功能纵向暗视微视野评估（SMART）研究中参与者首次就诊时的研究设计和特征。
在一项多中心自然史研究（ProgStar）中，对一部分经分子学证实为ABCA4相关斯塔加特病（STGD1）的参与者的一只指定研究眼进行暗视微视野检查（sMP）。研究访视每6个月进行一次，为期6至24个月，还包括眼底自发荧光（FAF）检查。
SMART研究招募了118名参与者（118只眼）。在SMART研究的首次就诊时，中视微视野检查的平均敏感度为11.48（±5.05；范围0.00 - 19.88）dB，sMP检查的平均敏感度为11.25（±5.26；0 - 19.25）dB。对于FAF检查，所有眼睛均有自发荧光降低的病变（平均病变面积3.62 [±3.48；0.10 - 21.46] mm²），共有76只眼（65.5%）有明确自发荧光降低的病变，平均病变面积为3.46（±3.60；0.21 - 21.46）mm²。
STGD1患者的视杆细胞功能受损，可通过sMP进行评估。检测视杆细胞功能可能作为未来临床治疗试验的潜在结局指标。这在SMART研究中进行评估。
