Evaluating for pentosan polysulfate maculopathy at a single academic institution

Purpose: To report the practices and outcomes of ophthalmic evaluations for pentosan polysulfate sodium (PPS) maculopathy at a single institution. Methods: This study was conducted on patients of Massachusetts Eye and Ear who had documented PPS exposure and an ophthalmic encounter from 2019 through 2022. The main outcomes were examination components performed and identification of PPS maculopathy. Image analysis confirmed findings. Results: Thirty-seven patients were included. Of the initial encounters, optical coherence tomography (OCT) was documented for 29 (78.4%) patients, fundus autofluorescence (FAF) for 13 (35.1%), and color fundus photography (CFP) for 12 (32.4%). Four cases (10.8%) of PPS maculopathy were observed. Mean (range) duration of exposure was 17 (15–20), and mean (range) cumulative exposure was 2418 (2190–2628) mg. Maculopathy did not occur until after 15 years of exposure and greater than 2000 mg of cumulative exposure. Three cases (8.1%) of PPS maculopathy were evaluated following drug cessation over a mean of 18.3 months. Two cases (5.4%) of PPS maculopathy progression post-cessation over durations of 1.1 and 4.3 years were described. Conclusion: We found inadequate imaging and documentation of OCT, CFP, and FAF to evaluate for toxicity in patients with a history of current or past PPS exposure. This study contributes four cases of PPS maculopathy to the growing literature reporting the phenotypic spectrum of toxicity, including two cases of maculopathy progression following drug cessation. There is a need for evaluations post-cessation due to possible progression of maculopathy, so patients are not treated inappropriately for differential diagnoses.