Prevalence and Dose Dependency Analysis of Pentosan Polysulfate Sodium Maculopathy

ABSTRACT

Purpose

We describe a retrospective cohort study investigating the prevalence of pentosan polysulfate sodium (PPS) maculopathy in patients with PPS exposure, as well as the relationship between cumulative PPS exposure and the presence of PPS-maculopathy.

Methods

Patients were identified through review of the electronic medical record system. Available diagnostic imaging was reviewed for signs of PPS-maculopathy. Patients were also contacted to determine cumulative exposure.

Results

Of the 335 identified eligible patient records, 84 had sufficient diagnostic imaging. Sixteen patients had definitive signs of PPS-maculopathy, 6 had likely signs of PPS-maculopathy, and 62 had no signs. The mean cumulative PPS exposure and standard error of the mean (SEM) for patients with any signs of PPS-maculopathy was 1946.0 g (396.0 g), significantly higher than the mean cumulative PPS exposure for patients without such signs of 782.3 g (105.3 g). No significant difference in BCVA was noted. The odds ratio (OR, 95% confidence interval (95% CI)) of PPS-maculopathy was significantly elevated in patients with cumulative PPS exposures of 1500–2000 g [OR 4.72 (0.856–26.02 95% CI)] and greater than 2000 g [OR 28.33 (2.388–336.1, 95% CI)]. Logistic regression analysis confirmed a positive dose response relationship.

Conclusions

We describe the concerning incidence of PPS-maculopathy in a multispecialty ophthalmology practice’s patient population and investigate the dose-dependency of PPS-maculopathy. Patients with PPS-maculopathy were shown to have a higher average exposure to PPS than those without the maculopathy. Patients with cumulative PPS exposures greater than 1500 g were shown to have an increased risk of PPS-maculopathy.

KEYWORDS:

• Pentosan polysulfate
• maculopathy
• Elmiron
• dose
• toxicity

Disclosure statement

No potential conflict of interest was reported by the author(s).

Publication statement

This manuscript is not being simultaneously considered for publication by any other journal. This manuscript has previously been submitted to Ophthalmology Retina, with the reviewer comments and our responses included as supplemental material for the editors, and to the British Journal of Ophthalmology, who said that the article did not meet the ”journal’s priorities” at that point in time, and encouraged us to submit to their open access companion journal.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.