Supplementary Material for: The Progression of Stargardt Disease (ProgStar) as determined by spectral-domain optical coherence tomography over a 24-month period (ProgStar Report No. 19)

Introduction: To evaluate the progression of atrophy as determined by spectral-domain optical coherence tomography (SD-OCT) in patients with molecularly confirmed ABCA4-associated Stargardt disease type 1 (STGD1) over a 24-month period in a multicenter prospective cohort study.
Methods: SD-OCT images from 428 eyes of 236 patients were analyzed. Change of mean thickness (MT) and intact area were estimated after semi-automated segmentation for the following individual layers in the central subfield (CS), inner ring (IR) and outer ring (OR) of the ETDRS grid: retinal pigment epithelium (RPE), outer segments (OS), inner segments (IS), outer nuclear layer (ONL) inner retina (IR) and total retina (TR).
Results: Statistically significant decreases of all outer retinal layers (RPE, OS, IS, and ONL) could be observed over a 24-month period both in decline of mean retinal thickness and intact area (p<.0001, respectively); whereas the inner retina showed an increase of retinal thickness in the central subfield and inner ring and remained unchanged in the outer ring.
Conclusions: Significant loss could be detected in outer retinal layers by SD-OCT over a 24-month period in patients with STGD1. Loss of thickness and/or intact area of such layers may serve as potential endpoints for clinical trials that aim to slow down the disease progression of STGD1.

引言：本项多中心前瞻性队列研究旨在评估经分子学确诊的ABCA4相关1型Stargardt病（ABCA4-associated Stargardt disease type 1, STGD1）患者，在24个月随访周期内，经光谱域光学相干断层扫描（spectral-domain optical coherence tomography, SD-OCT）检测的视网膜萎缩进展情况。
方法：本研究对236例患者共428只眼的光谱域光学相干断层扫描图像进行分析。针对早期糖尿病视网膜病变研究（Early Treatment Diabetic Retinopathy Study, ETDRS）网格的中心亚区（central subfield, CS）、内环（inner ring, IR）与外环（outer ring, OR）内的下述各视网膜层，通过半自动分割算法估算其平均厚度（mean thickness, MT）与完整区域面积的变化：视网膜色素上皮（retinal pigment epithelium, RPE）、外节（outer segments, OS）、内节（inner segments, IS）、外核层（outer nuclear layer, ONL）、视网膜内层（inner retina, IR）及全视网膜（total retina, TR）。
结果：在24个月随访周期内，所有视网膜外层（RPE、OS、IS及ONL）的平均视网膜厚度与完整区域面积均出现具有统计学意义的下降（两组均p<0.0001）；而视网膜内层在中心亚区与内环的视网膜厚度出现升高，在外环则无明显变化。
结论：在24个月随访周期内，通过SD-OCT可检测到STGD1患者的视网膜外层出现显著的结构丢失。此类视网膜层的厚度及/或完整区域面积的丢失，可作为旨在延缓STGD1疾病进展的临床试验的潜在终点指标。