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The following is a summary of “Pentosan Polysulfate Maculopathy: Final Outcomes from a 4-Year Prospective Study of Disease Progression after Drug Cessation,” published in the December 2024 issue of Ophthalmology by Hall et al. 

Researchers conducted a retrospective study to report the long-term disease course of pentosan polysulfate (PPS) maculopathy after discontinuation of the drug.  

They included 23 eyes from 12 participants diagnosed with PPS maculopathy at the Emory Eye Center, enrolled between December 1, 2018, and December 1, 2019, with data collected annually for 4 years. Visual function was evaluated using the 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 measured using the National Eye Institute Visual Function Questionnaire (NEI-VFQ-39) and the Low Luminance Questionnaire (LLQ). Structural outcomes assessed included complete retinal pigment epithelium and outer retinal atrophy (cRORA), atrophic lesion size (in mm²), macular central subfield thickness (CST), and subfoveal choroidal thickness (SFCT).  

 The results showed that 11 of the 12 participants (91.7%) were female, with a median age of 58 years. The median ETDRS BCVA letter score at baseline was 83, with a −5 letter median change over 4 years (P = 0.005). The median 4-year changes in mesopic microperimetry were −5.4 dB for the average threshold (P = 0.003) and 48.6% for the reduced threshold (P = 0.004). The MNREAD performance declined, with a median maximum reading speed change of −21 words per minute (P = 0.007), NEI-VFQ-39 and LLQ composite scores significantly decreased. At baseline, 9 eyes (39%) had macular cRORA, with 5 additional eyes (35.7%) developing new-onset cRORA by the study’s end. The median linearized growth rate of atrophic lesions was 0.23 mm/year. The median 4-year changes in macular CST and SFCT were −7.0 μm (P = 0.055) and −22.0 μm (P = 0.610), respectively.  

Investigators concluded the persistent functional and structural impairments in patients with PPS maculopathy, even after discontinuing the medication, were revealed.

Source: ajo.com/article/S0002-9394(24)00562-2/abstract