Importance Case series have identified a macular condition hypothesized to be associated with the use of pentosan polysulfate sodium (PPS). Observational studies seeking to quantify this association have yielded equivocal results.

Objective To estimate the association between PPS exposure and maculopathy.

Design, Setting, and Participants This disproportionality analysis was conducted using the US Food and Drug Administration Adverse Event Reporting System from January 2013 through June 2020.

Exposure Adverse event reports for pentosan polysulfate were selected and compared with adverse event reports associated with drugs taken for the following indications: interstitial cystitis, cystitis, bladder disorder, or bladder pain.

Main Outcome Measures Retinal adverse events were identified using the retinal disorders Standardized Medical Dictionary for Regulatory Activities (MedDRA) Query, which includes conditions associated with retinal damage attributable to blockage of its blood supply, nutritional deficiencies, toxins, and diseases affecting the retina.

Results There were 2775 reports available for analysis in the PPS group (of which 1966 were for women [70.9%]) and 6833 reports in the other drugs group (of which 4036 [59.1%] were for women). The proportion of adverse events for any macular event relative to all other events was elevated for the users of PPS compared with those using other interstitial cystitis and bladder pain drugs (proportionate reporting ratio [PRR], 1.21 [95% CI, 1.01-1.44]). With respect to specific retinal conditions, macular degeneration (20 [0.8%] vs 15 [0.2%]), maculopathy (83 [3.4%] vs 2 [0.03%]), retinal dystrophy (3 [0.1%] vs 0), retinal injury (5 [0.2%] vs 0), and retinal toxicity (3 [0.1%] vs 0) were proportionately more common among users of PPS compared with those using other interstitial cystitis and bladder pain drugs, respectively.

Conclusions and Relevance The results of the current study add to the growing evidence that PPS use is associated with an increased risk of maculopathy. Studies that rule out prevalent retinal abnormalities prior to the initiation of PPS would strengthen the current body of literature.

重要性 案例系列已经确定了一种假设与使用戊聚糖多硫酸钠 (PPS) 相关的黄斑病变。试图量化这种关联的观察性研究产生了模棱两可的结果。

目的 评估PPS暴露与黄斑病变的关系。

设计、设置和参与者 这项不成比例分析是使用美国食品和药物管理局不良事件报告系统从 2013 年 1 月到 2020 年 6 月进行的。

选择了多硫酸戊聚糖 的不良事件报告，并将其与用于以下适应症的药物相关的不良事件报告进行比较：间质性膀胱炎、膀胱炎、膀胱疾病或膀胱疼痛。

主要结果指标 视网膜不良事件是使用视网膜疾病监管活动标准化医学词典 (MedDRA) 查询确定的，其中包括与视网膜损伤相关的疾病，这些疾病可归因于血液供应的阻塞、营养缺乏、毒素和影响视网膜的疾病。

结果 PPS 组有 2775 份报告可供分析（其中 1966 份为女性 [70.9%]），其他药物组有 6833 份报告（其中 4036 份 [59.1%] 为女性）。与使用其他间质性膀胱炎和膀胱疼痛药物的患者相比，PPS 使用者的任何黄斑事件相对于所有其他事件的不良事件比例升高（比例报告率 [PRR]，1.21 [95% CI，1.01-1.44] ）。关于特定的视网膜状况，黄斑变性（20 [0.8%] vs 15 [0.2%]），黄斑病变（83 [3.4%] vs 2 [0.03%]），视网膜营养不良（3 [0.1%] vs 0），与使用其他间质性膀胱炎和膀胱疼痛药物的人相比，PPS 使用者的视网膜损伤（5 [0.2%] vs 0）和视网膜毒性（3 [0.1%] vs 0）比例更高。

结论和相关性 当前研究的结果增加了越来越多的证据表明 PPS 的使用与黄斑病变风险增加有关。在 PPS 开始之前排除普遍的视网膜异常的研究将加强当前的文献。