Pentosan polysulfate maculopathy
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A Prospective cohort study of 741 patients prescribed pentosan polysulfate (PPS) within a large university database, 100 (13.4%) with any consumption agreed to participate in a prospective screening investigation. Multimodal retinal imaging including near infrared reflectance (NIR), fundus autofluorescence (FAF), and spectral domain optical coherence tomography (SD-OCT) was performed in all patients. Characteristic findings of affected patients were identified and affected and unaffected cohorts were compared. Choroidal vascularity index (CVI), defined as stromal choroidal area (SCA) divided by total choroidal area, was analyzed.

The prevalence of PPS maculopathy was 16%. NIR illustrated punctate hyperreflective lesions with early presentation. FAF illustrated a speckled macular network of hypo- and hyperautofluorescence colocalized with multifocal hyperreflective retinal pigment epithelial lesions on SD-OCT. Advanced cases demonstrated varying degrees of atrophy. The affected cohort exhibited significantly greater average PPS therapy duration, average daily dosage, and average cumulative dosage (19.5±5.5 years, 433.9±137.6 milligrams, 3103.1±1402.2 grams) compared to the unaffected cohort (7.1±6.6 years, 291.6±177.6 milligrams, 768.4±754.8 grams). SCA was significantly lower and CVI was significantly greater in the affected versus the unaffected group.

This prospective cohort study identified a prevalence of PPS maculopathy of 15-20% among PPS users who agreed to participate. A spectrum of findings may be observed with multimodal retinal imaging. Significant choroidal abnormalities associated with this characteristic maculopathy may provide surrogate markers of macular toxicity.

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