Retrospective study of incidence/prevalence of pigmentary maculopathy and retinopathy in patients receiving pentosan polysulfate sodium

PLoS One. 2025 Jan 9;20(1):e0313497. doi: 10.1371/journal.pone.0313497. eCollection 2025.

Abstract

Purpose: To evaluate prevalence and incidence rates of pigmentary maculopathy and retinopathy (PM/PR), and visual acuity (VA) changes in patients exposed to pentosan polysulfate sodium (PPS) and in patients with interstitial cystitis (IC) not exposed to PPS.

Methods: This is a retrospective cohort study (January 2015-March 2021) which included adult de-identified patients from the American Academy of Ophthalmology IRIS® Registry (Intelligent Research in Sight) and Komodo Health database. Three patient cohorts were identified: two PPS-exposed patient cohorts, and Non-PPS-exposed IC patient cohort. Key study outcomes included PM/PR/Any (defined based on prior literature regardless of PPS exposure) and PM/PR/PPS (further defined by an algorithm that was based on clinical notes and other protocol-prespecified criteria in PPS-exposed patients), and VA changes in each respective cohort.

Results: Prevalence of PM/PR/Any was relatively common in patients prior to PPS exposure (4.16%-8.43%). Incidence rate of PM/PR/PPS was uncommon in both PPS-exposed cohorts (0.13-0.15 per 100 person-years). Crude incidence rates of PM/PR/Any (based on ITT analysis) varied slightly across 3 study cohorts (2.13-2.81 and 2.38 per 100-person-years for PPS-exposed cohorts and Non-PPS-exposed IC cohort, respectively). Across all 3 study cohorts, patients with PM/PR/Any appeared to have approximately 2-fold higher proportion of 3 lines of VA worsening than those without PM/PR/Any.

Conclusion: Prevalence of PM/PR was common in patients prior to PPS exposure. Incidence of PM/PR/PPS that may be associated with PPS exposure was relatively uncommon. Crude incidence rates of PM/PR appeared similar across all patient cohorts regardless of PPS exposure.

MeSH terms

  • Adult
  • Aged
  • Cystitis, Interstitial* / drug therapy
  • Cystitis, Interstitial* / epidemiology
  • Female
  • Humans
  • Incidence
  • Macular Degeneration* / chemically induced
  • Macular Degeneration* / epidemiology
  • Male
  • Middle Aged
  • Pentosan Sulfuric Polyester* / adverse effects
  • Prevalence
  • Retinal Diseases / chemically induced
  • Retinal Diseases / epidemiology
  • Retrospective Studies
  • Visual Acuity / drug effects

Substances

  • Pentosan Sulfuric Polyester