Pentosan Polysulfate Maculopathy—We Need to Know More

Interstitial cystitis, also known as painful bladder syndrome, is characterized by urinary urgency, frequency, and nocturia, often accompanied by bladder and pelvic pain ranging from mild to severe. Interstitial cystitis affects approximately 1 million people in the US, most of them women. Pentosan polysulfate (PPS) (Elmiron; Janssen Pharmaceuticals) was approved by the US Food and Drug Administration (FDA) in 1996 for the treatment of interstitial cystitis and remains the only FDA-approved drug for this disease. However, it was not until 2018 that Pearce et al reported a case series of 6 women who were long-term users of PPS and had a pigmentary maculopathy with distinctive signs on fundus autofluorescence and near-infrared imaging. The mean (SD) duration of PPS use in patients with the associated maculopathy was 15.0 (5.7) years in a recent comprehensive review that summarized previous studies through 2021,1 and the median (IQR) was 14 (10.2-18.9) years in a large (74 patients), recently published multicenter case series.