Pentosan Polysulfate Maculopathy: Pathophysiology, Clinical Features, and Screening Strategies

초록

Pentosan polysulfate (PPS) has been widely used for the treatment of interstitial cystitis, but has been linked to a progressive and potentially irreversible maculopathy. PPS maculopathy is characterized by pigmentary changes, outer retinal atrophy, and visual symptoms such as difficulty reading and dark adaptation, despite relatively preserved central acuity in early stages. Histologic and imaging studies suggest that PPS toxicity primarily targets the retinal pigment epithelium, leading to secondary photoreceptor loss. The risk of toxicity increases with cumulative dose and treatment duration, but reported prevalence varies widely, from approximately 5% or lower in claims-based studies to around 20% in tertiary-center cohorts. Multimodal imaging—including fundus autofluorescence, near-infrared reflectance, and optical coherence tomography—plays a critical role in early detection. Despite this, screening practices remain inconsistent and inadequate in many clinical settings. Recent nationwide data from South Korea revealed that over half of high-risk PPS users did not undergo any ophthalmic examination during the study period. Given the irreversible nature of PPS maculopathy and its progressive course even after drug cessation, early identification is crucial. There is an urgent need to establish standardized screening protocols based on cumulative dose and duration of therapy, similar to existing guidelines for hydroxychloroquine retinopathy. Increased collaboration between urologists and ophthalmologists, along with heightened awareness among patients and providers, is essential to prevent vision loss in this vulnerable population.

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