Background/Aims A series at a single clinical centre recently demonstrated an association between the interstitial cystitis drug pentosan polysulfate sodium (PPS) and a vision-threatening pigmentary maculopathy. The aim of this study was to determine if an association exists between PPS use and macular disease in a large national cohort.
Methods A retrospective, matched cohort study using data from a large US medical claims database from 2002 to 2016 was performed. A total of 3012 and 1604 PPS users were compared with 15 060 and 8017 matched controls at 5 and 7 years, respectively. The primary outcome measures included (1) any new diagnosis of a hereditary or secondary pigmentary maculopathy (atypical maculopathy outcome), and (2) any new diagnosis of dry age-related macular degeneration (AMD) or drusen in addition to the aforementioned diagnoses (atypical maculopathy+AMD outcome).
Results At the 5-year and 7-year follow-up, 9 (0.3%) and 10 (0.6%) PPS patients progressed to the atypical maculopathy outcome compared with 32 (0.2%) and 25 (0.3%) control patients, respectively. 103 (3.4%) and 87 (5.4%) PPS patients developed the atypical maculopathy+AMD outcome compared with 440 (2.9%) and 328 (4.1%) control patients at 5 and 7 years, respectively. At 5 years, multivariate analysis showed no significant association (p>0.13). At 7 years, PPS users had significantly increased odds of having the atypical maculopathy+AMD outcome (OR=1.41, 95% CI 1.09 to 1.83, p=0.009).
Conclusions PPS exposure was associated with a new diagnosis of macular disease at the 7-year follow-up in a large national cohort.
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Contributors NJ: study design and conception, data analysis and interpretation, writing manuscript, manuscript revision, final approval and agreement to be accountable. AL: data analysis and interpretation, writing manuscript, manuscript revision, final approval and agreement to be accountable. YY: data acquisition, data analysis and interpretation, manuscript revision, final approval and agreement to be accountable. BLVB: study design and conception, data acquisition, data analysis and interpretation, writing manuscript, manuscript revision, final approval and agreement to be accountable.
Funding Foundation Fighting Blindness (CD-C-0918-0748-EEC) (NJ); NIH Core Grant P30 EY006360 (Emory Eye Center); National Institutes of Health K23 Award (1K23EY025729-01) (BLVB) and University of Pennsylvania Core Grant for Vision Research (2P30EY001583) (BLVB, YY). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Additional funding was provided by Research to Prevent Blindness and the Paul and Evanina Mackall Foundation. Funding from each of the above sources was received in the form of block research grants to the Scheie Eye Institute. The sponsors or funding organisations had no role in the design or conduct of this research.
Competing interests None declared.
Patient consent for publication Not required.
Ethics approval Ethics approval was not required due to the deidentified nature of the dataset. It met the eligibility criteria for Institutional Review Board review exemption (Protocol No. 819924).
Provenance and peer review Not commissioned; externally peer reviewed.
Data availability statement Data may be obtained from a third party and are not publicly available.