Elmiron (pentosan polysulfate sodium): rare risk of pigmentary maculopathy

Cases of pigmentary maculopathy leading to visual impairment have been reported with pentosan polysulfate, particularly after long-term use at high doses. Ensure patients taking pentosan polysulfate have regular ophthalmic examinations and ask them to promptly seek medical advice in case of visual changes.

Advice for healthcare professionals:

  • rare cases of pigmentary maculopathy have been reported in patients using pentosan polysulfate, particularly after long-term use at high doses
  • given the potentially irreversible nature of visual loss in pigmentary maculopathy, ensure patients taking pentosan polysulfate have regular ophthalmic examinations during treatment (for example, at baseline and annually)
  • advise patients on pentosan polysulfate to promptly seek medical advice in case of visual changes such as reading difficulty or slow adjustment to low or reduced light environments
  • consider stopping treatment in patients with pigmentary maculopathy
  • report suspected adverse drug reactions to pentosan polysulfate sodium on a Yellow Card, including any visual problems or unusual findings in ophthalmic tests

Risk of pigmentary maculopathy

Pentosan polysulfate is indicated for the treatment of bladder pain syndrome (interstitial cystitis) characterised by either glomerulations or Hunner’s lesions in adults with moderate to severe pain, urgency, and frequency of micturition. The recommended dose of pentosan polysulfate sodium is 300 mg per day, taken as one 100 mg capsule orally three-times daily.

A recent review of cumulative safety information identified rare cases of pigmentary maculopathy after use of pentosan polysulfate in patients with a diagnosis of interstitial cystitis (also known as bladder pain syndrome).1 2 3 The product information has been updated to include this risk.

In most cases, patients had used pentosan polysulfate long-term and at a dosage exceeding the recommended dose. In a recent retrospective study,3 patients with pigmentary maculopathy had a median length of exposure to pentosan polysulfate of 18.3 years with a range of 3.0–21.9 years.

Unique characteristics of pigmentary maculopathy associated with Elmiron

The pigmentary maculopathy described differs from other forms. Fundus examination showed unique subtle paracentral hyperpigmentation at the level of the retinal pigment epithelium (RPE) with associated areas of RPE atrophy. Multi-modal retinal imaging demonstrated abnormalities of the RPE and overlying retina generally contained in multiple well-delineated areas. This unique maculopathy has only been observed with use of pentosan polysulfate.

Monitor patients via regular ophthalmological examinations

The pathogenesis for pigmentary maculopathy with pentosan polysulfate is unclear and it is not known whether drug cessation will halt or alter the course of this retinal disorder. Nevertheless, as a precautionary measure, treatment cessation should be considered in affected patients.

Given the potentially irreversible nature of visual loss in pigmentary maculopathy, all patients taking pentosan polysulfate should have regular ophthalmological examinations (for example, at baseline and annually). This monitoring may allow early detection of pigmentary maculopathy, potentially at a reversible stage. Monitoring is particularly important for patients who are, or have been, taking pentosan polysulfate long-term or at a high dose.

Report suspected adverse drug reactions with pentosan polysulfate

Suspected adverse drug reactions, including any visual problems or unusual findings in ophthalmic tests should be reported to the Yellow Card Scheme. Remember only a suspicion is needed to report – if in doubt, please complete a Yellow Card.

Healthcare professionals, patients, and caregivers can report suspected side effects via the Yellow Card website or via the Yellow Card app. Download the app today via iTunes Yellow Card for iOS devices or via PlayStore Yellow Card for Android devices.

Further information

European Medicines Agency. Scientific Conclusions. June 2019.

Direct Healthcare Professional Communication. July 2019.

Article citation: Drug Safety Update volume 13, issue 2: September 2019: 3.

Published 19 September 2019