Longitudinal Microperimetric Changes of Macular Sensitivity in Stargardt Disease After 12 Months ProgStar Report No. 13
Longitudinal Microperimetric Changes of Macular Sensitivity in Stargardt Disease After 12 Months ProgStar Report No. 13
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DOI：
10.1001/jamaophthalmol.2020.1735
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发表时间：
2020-07-01
影响因子：
8.1
通讯作者：
Scholl, Hendrik P. N.
中科院分区：
文献类型：
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作者：
Schonbach, Etienne M.;Strauss, Rupert W.;Scholl, Hendrik P. N.
重要性功能性终点临床试验调查的新兴治疗方法的疗效Stargardt病1型（STGD 1）是needed.Objective评估黄斑功能的变化在STGD 1患者使用microperimetry年率设计，设置，和参与者这项多中心前瞻性队列研究进行了国际选择的三级转诊中心从2013年10月21日，到2017年2月15日该研究包括ABCA 4相关STGD 1的参与者，他们在基线时参加了Stargardt病继发性萎缩进展的自然史（ProgStar）研究。分析2017年2月16日至2019年12月1日的数据。曝光ABCA 4相关STGD 1，眼底自发荧光显示最小病变大小和最小视力。主要结果和测量总体黄斑敏感度（MS），深暗点计数，测试正常的点数，结果200例359眼患者中，（87名[43.5%]男子;平均[SD]年龄，33.3 [15.2]岁），在基线和第12个月接受微视野检查分级，MS的平均（SD）年变化为-0.68（2.04）dB（95%CI，-0.89至-0.47 dB; P <0.001），深暗点平均（SD）每年增加1.56（5.74）点。灵敏度为12 dB或更高的点的灵敏度平均（SD）降低了-3.01（9.84）dB（95% CI，-4.03至-1.99 dB; P < .001）。在两次访视时，具有良好或一般图案放置分级的眼睛之间MS的平均（SD）年变化没有显著差异（-0.67 [2.1] dB）和至少1次访视期间图案放置不良的眼睛（-0.64 [2.2] dB）（P = 0.91）。结论和相关性该研究表明，在随访约1年后，MS和深暗点的数量发生了可测量的变化。微视野可作为一个有用的功能结果参数的临床试验，旨在减缓STGD 1的进展。
Importance Functional end points for clinical trials investigating the efficacy of emerging treatments for Stargardt disease type 1 (STGD1) are needed.Objective To assess the yearly rate of change of macular function in patients with STGD1 using microperimetry.Design, Setting, and Participants This multicenter prospective cohort study was conducted in an international selection of tertiary referral centers from October 21, 2013, to February 15, 2017. The study included participants with ABCA4-related STGD1 who were enrolled in the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) study at baseline. Data were analyzed from February 16, 2017, to December 1, 2019.Exposure ABCA4-related STGD1 with a minimum lesion size on fundus autofluorescence and a minimum visual acuity.Main Outcomes and Measures Changes in overall macular sensitivity (MS), deep scotoma count, number of points that tested normal, and location-specific sensitivity changes.Results Among the 359 eyes from 200 patients (87 [43.5%] men; mean [SD] age, 33.3 [15.2] years) who underwent microperimetry examination graded at baseline and month 12, the mean (SD) yearly change in MS was -0.68 (2.04) dB (95% CI, -0.89 to -0.47 dB; P < .001), and deep scotoma points increased by a mean (SD) of 1.56 (5.74) points per year. The points with sensitivity of 12 dB or higher decreased in sensitivity by a mean (SD) of -3.01 (9.84) dB (95% CI, -4.03 to -1.99 dB; P < .001). The mean (SD) yearly change in MS was not significantly different between the eyes with a grading of good or fair pattern placement at both visits (-0.67 [2.1] dB) and the eyes with a poor pattern placement during at least 1 visit (-0.64 [2.2] dB) (P = .91).Conclusions and Relevance This study showed that MS and the number of deep scotoma points had measurably changed after follow-up of approximately 1 year. Microperimetry may serve as a useful functional outcome parameter for clinical trials aimed at slowing the progression of STGD1.