Birmingham, AL—Pentosan polysulfate sodium (PPS), marketed as Elmiron, is the only FDA–approved pharmacologic treatment for interstitial cystitis (IC), a chronic bladder condition that causes pressure and pain.

Although the medication received FDA approval in 1996, according to a new study, concerns have only recently been raised about the association between PPS and maculopathy, a condition with unique pathological findings that causes difficulty with visual tasks, such as reading, as well as dark adaptation impairment.

“Based on a number of case series, this condition, now termed PPS-associated maculopathy, has been associated with long-term PPS exposure, the median duration of use being at least 15 years, although some patients have reported shorter durations,” write University of Alabama Birmingham researchers in JAMA Ophthalmology.

The study team sought to estimate the association between PPS exposure and maculopathy using a disproportionality analysis of the FDA’s Adverse Event Reporting System from January 2013 through June 2020. The focus was on adverse event reports for pentosan polysulfate; those were compared with adverse event reports associated with drugs taken for interstitial cystitis, cystitis, bladder disorder, or bladder pain.

At the same time, retinal adverse events were identified using the retinal disorders Standardized Medical Dictionary for Regulatory Activities (MedDRA) Query, which includes conditions associated with retinal damage attributable to blockage of its blood supply, nutritional deficiencies, toxins, and diseases affecting the retina.

Of the 2,775 reports available for analysis in the PPS group, 70.9% were in women, and of the 6,833 reports in the other drugs group, 59.1% were in women.

Results indicate that the proportion of adverse events for any macular event relative to all other events was elevated for the users of PPS compared with those using other interstitial cystitis and bladder pain drugs (proportionate reporting ratio [PRR], 1.21 [95% CI, 1.01-1.44]).

“Specific retinal conditions proportionately more common among users of PPS compared with those using other interstitial cystitis and bladder pain drugs were:

• macular degeneration (20 [0.8%] vs 15 [0.2%]),
• maculopathy (83 [3.4%] vs 2 [0.03%]),
• retinal dystrophy (3 [0.1%] vs 0),
• retinal injury (5 [0.2%] vs 0), and
• retinal toxicity (3 [0.1%] vs 0).

“The results of the current study add to the growing evidence that PPS use is associated with an increased risk of maculopathy,” the researchers conclude. “Studies that rule out prevalent retinal abnormalities prior to the initiation of PPS would strengthen the current body of literature.”

Background information in the articles notes that treatment options for IC, which primarily affects women, include lifestyle changes, such as dietary modifications; bladder training; physical therapy; medications; bladder procedures, such as hydrodistension; and in rare cases, surgery.

Pharmacologic treatment for the condition includes OTC drugs such as antihistamines and nonsteroidal anti-inflammatories, while prescription medications such as tricyclic antidepressants and cyclosporine also are used in addition to PPS.

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