Risk factors for pentosan polysulfate maculopathy

Shiromani S, Martini A, Ou X, Hall BP, Leal CA, Rosengren KJ, Cui X, Jain N · Canadian Journal of Ophthalmology

Cumulative dose dominates the risk — odds ratio 39.8 in the top quartile at 2,000 g or more — but dose per body weight, older age, female sex and inflammatory bowel disease also came out as independent factors, and toxicity prevalence in this screened population ran between 3.8% and 24.5%. Among patients below 800 g cumulative, those who developed maculopathy were older, lighter, and had taken the drug longer at a higher dose per kilogram.

Population

187 PPS-exposed patients screened with questionnaires and fundus imaging at Emory Eye Center, 2018–2024

Intervention

Oral PPS at varying cumulative exposure

Comparator

Lower-exposure quartiles within the same cohort

Limitations

Cross-sectional and single-centre, at a referral practice where patients with symptoms are over-represented, which is why the prevalence estimate spans a sixfold range; cumulative dose is patient-recalled; the wide confidence interval on the top-quartile odds ratio reflects small numbers in the comparison group.