PURPOSE To report the long-term disease course of pentosan polysulfate (PPS) maculopathy following drug cessation. DESIGN Single-institution, prospective case series. METHODS 23 eyes of 12 participants seen at the Emory Eye Center with a diagnosis of PPS maculopathy were included in our study. Participants were enrolled between December 1, 2018, and December 1, 2019, and data were collected annually for four years. MAIN OUTCOMES AND MEASURES Changes in visual function and structure were the primary outcomes measured. Visual function was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA), ETDRS low-luminance visual acuity (LLVA), Minnesota low-vision reading (MNREAD) performance, contrast sensitivity, mesopic and scotopic microperimetry, and dark adaptometry. Patient reported outcomes were assessed with the National Eye Institute Visual Function Questionnaire (NEI-VFQ-39) and Low Luminance Questionnaire (LLQ). Structural outcomes included the presence of complete retinal pigment epithelium and outer retinal atrophy (cRORA), atrophic lesion size (in mm2), macular central subfield thickness (CST), and subfoveal choroidal thickness (SFCT). RESULTS Of the 12 participants, 11 (91.7%) were female, with a median age at enrollment of 58 years. The median ETDRS BCVA letter score at baseline was 83, with a median change of -5 letters over 4 years (P = 0.005). The median 4-year change in mesopic microperimetry average threshold and percent reduced threshold was -5.4 dB (P = 0.003) and 48.6% (P = 0.004), respectively. MNREAD performance (assessed at 2 and 4 years) declined across all measures, with a median maximum reading speed change of -21 words per minute (P = 0.007). NEI-VFQ-39 and LLQ composite scores significantly decreased over 4 years. At baseline, 9 eyes (39%) had macular cRORA. By the study's end, 5 of the remaining eyes (35.7%) developed new-onset cRORA. The median linearized growth rate of atrophic lesions was 0.23 mm/year. The median 4-year change of CST and SFCT was -7.0 μm (P = 0.055) and -22.0 μm (P = 0.610), respectively. CONCLUSION This prospective study demonstrates continued progression of broad-ranging functional and structural deficits in PPS maculopathy long after drug cessation. These findings should inform PPS prescribing patterns, patient counseling, and disease monitoring strategies.

中文翻译：

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戊聚糖多硫酸盐黄斑病：停药后疾病进展 4 年前瞻性研究的最终结果。


目的 报告停药后戊聚糖多硫酸盐 （PPS） 黄斑病的长期病程。设计 单机构、前瞻性病例系列。方法 我们的研究包括在埃默里眼科中心就诊的 12 名诊断为 PPS 黄斑病变的参与者的 23 只眼睛。参与者在 2018 年 12 月 1 日至 2019 年 12 月 1 日期间入组，每年收集一次数据，持续四年。主要结局和测量 视觉功能和结构的变化是测量的主要结局。使用早期治疗糖尿病视网膜病变研究 （ETDRS） 最佳矫正视力 （BCVA） 、ETDRS 低亮度视力 （LLVA）、明尼苏达低视力阅读 （MNREAD） 性能、对比敏感度、中视和暗视显微视野计以及暗适应测定法评估视觉功能。使用国家眼科研究所视觉功能问卷 （NEI-VFQ-39） 和低亮度问卷 （LLQ） 评估患者报告的结果。结构结局包括存在完全视网膜色素上皮和视网膜外萎缩 （cRORA） 、萎缩病变大小 （mm2） 、黄斑中央子视野厚度 （CST） 和中心凹下脉络膜厚度 （SFCT）。结果 在 12 名参与者中，11 名 （91.7%） 为女性，入组年龄中位数为 58 岁。基线时的中位 ETDRS BCVA 字母评分为 83,4 年内中位变化为 -5 个字母 （P = 0.005）。中间微观视野平均阈值和降低阈值百分比的中位 4 年变化分别为 -5.4 dB （P = 0.003） 和 48.6% （P = 0.004）。所有指标的 MNREAD 性能 （在 2 年和 4 年评估） 均下降，最大阅读速度变化的中位数为每分钟 -21 个单词 （P = 0.007）。 NEI-VFQ-39 和 LLQ 综合评分在 4 年内显着下降。基线时，9 只眼 （39%） 患有黄斑 cRORA。到研究结束时，剩余的 5 只眼睛 （35.7%） 出现了新发的 cRORA。萎缩病变的中位线性化增长率为 0.23 mm/年。CST 和 SFCT 的中位 4 年变化分别为 -7.0 μm （P = 0.055） 和 -22.0 μm （P = 0.610）。结论 这项前瞻性研究表明，在停药后很长一段时间内，PPS 黄斑病变的广泛功能和结构缺陷仍在继续发展。这些发现应为 PPS 处方模式、患者咨询和疾病监测策略提供信息。