Ervin Ann-Margret, Strauss Rupert W, Ahmed Mohamed I, Birch David, Cheetham Janet, Ferris Frederick L, Ip Michael S, Jaffe Glenn J, Maguire Maureen G, Schönbach Etienne M, Sadda SriniVas R, West Sheila K, Scholl Hendrik P N
Wilmer Eye Institute, The Johns Hopkins School of Medicine, Johns Hopkins University, Baltimore, MD, USA.
Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA.
Transl Vis Sci Technol. 2019 Apr 12;8(2):16. doi: 10.1167/tvst.8.2.16. eCollection 2019 Apr.
The Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies were designed to measure the progression of Stargardt disease through the use of fundus autofluorescence imaging, optical coherence tomography, and microperimetry. The overarching objectives of the studies were to document the natural course of Stargardt disease and identify the most appropriate clinical outcome measures for clinical trials assessing the efficacy and safety of upcoming treatments for Stargardt disease. A workshop organized by the Foundation Fighting Blindness Clinical Research Institute was held on June 11, 2018, in Baltimore, MD, USA. Invited speakers discussed spectral-domain optical coherence tomography, fundus autofluorescence, and microperimetry methods and findings in the ProgStar prospective study. The workshop concluded with a panel discussion of optimal endpoints for measuring treatment efficacy in Stargardt disease. We summarize the workshop presentations in light of the most current literature on Stargardt disease and discuss potential clinical outcome measures and endpoints for future treatment trials.
继发于斯塔加特病的萎缩进展(ProgStar)研究旨在通过使用眼底自发荧光成像、光学相干断层扫描和微视野计来测量斯塔加特病的进展。这些研究的总体目标是记录斯塔加特病的自然病程,并确定用于评估即将到来的斯塔加特病治疗方法的疗效和安全性的临床试验中最合适的临床结局指标。由抗盲基金会临床研究所组织的一次研讨会于2018年6月11日在美国马里兰州巴尔的摩举行。受邀演讲者讨论了ProgStar前瞻性研究中的光谱域光学相干断层扫描、眼底自发荧光和微视野计方法及研究结果。研讨会最后进行了小组讨论,内容是关于测量斯塔加特病治疗疗效的最佳终点。我们根据关于斯塔加特病的最新文献总结了研讨会的报告,并讨论了未来治疗试验潜在的临床结局指标和终点。