Institute for Vision Research, Carver College of Medicine, University of Iowa, Iowa City, IA, US.
Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA, US.
Asia Pac J Ophthalmol (Phila). 2022;11(2):100-110. doi: 10.1097/APO.0000000000000504.
Pentosan polysulfate (PPS) sodium (Elmiron) is the only Food and Drug Administration (FDA)-approved oral medication to treat interstitial cystitis, also known as bladder pain syndrome. A symptomatic pigmentary maculopathy associated with PPS was reported in 2018. Since then, recognition of this unique drug toxicity has increased rapidly. This potentially sight-threatening side effect prompted the FDA in June 2020 to update the label for PPS to warn about "retinal pigmentary changes." A challenging feature of pentosan maculopathy is its ability to mimic many other retinal conditions, including inherited retinal dystrophies such as pattern dystrophy, mitochondrially inherited diabetes and deafness, and Stargardt disease, and age-related macular degeneration. In this review, we discuss the history of PPS maculopathy and its implications for thousands of at-risk interstitial cystitis patients. We use published literature and an illustrative case from our institution to highlight the importance of diagnosing PPS maculopathy. We also compare PPS maculopathy to age-related macular degeneration, explain why differentiating between the 2 is clinically important, and highlight avenues for further research. Finally, we highlight the paucity of data on patients of color and why this lack of understanding may impact patient care.
戊聚糖多硫酸酯钠（Elmiron）是唯一被美国食品和药物管理局（FDA）批准用于治疗间质性膀胱炎（也称为膀胱疼痛综合征）的口服药物。2018 年报道了一种与戊聚糖有关的症状性色素性黄斑病变。此后，人们对这种独特的药物毒性的认识迅速提高。这种潜在的致盲副作用促使 FDA 于 2020 年 6 月更新了戊聚糖的标签，以警告“视网膜色素变化”。戊聚糖黄斑病变的一个具有挑战性的特征是它能够模仿许多其他视网膜疾病，包括遗传性视网膜营养不良，如图案性营养不良、线粒体遗传糖尿病和耳聋以及斯塔加特病，以及年龄相关性黄斑变性。在这篇综述中，我们讨论了 PPS 黄斑病变的历史及其对数千名有风险的间质性膀胱炎患者的影响。我们使用已发表的文献和我们机构的一个说明性病例来强调诊断 PPS 黄斑病变的重要性。我们还将 PPS 黄斑病变与年龄相关性黄斑变性进行了比较，解释了为什么区分这两种疾病在临床上很重要，并强调了进一步研究的途径。最后，我们强调了关于有色人种患者的数据不足，以及这种缺乏理解可能如何影响患者的护理。
