Prevalence of Maculopathy Associated with Long-Term Pentosan Polysulfate Therapy

Vora, Robin; Patel, Amar; Melles, Ronald B.

doi:10.1016/j.ophtha.2020.01.017

Cited by 52 publications

(41 citation statements)

References 7 publications

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“… 13 Similarly, the present study found a mean cumulative dose of 1.6kg and a mean duration of 13.5 years in the PPS-associated retinopathy group. Vora et al performed a multivariable regression analysis of a cohort of 117 patients and found that the risk of developing PPS-associated maculopathy increased from 13% in those with 500–999g of exposure to 42% with over 1500g; 7 the present study found similar incidences of 12% (3/25) and 50% (10/20), respectively. There was no statistically significant association between the presence or severity of pigmentary retinopathy and a history of liver or renal insufficiency, nor the female height, dosage per weight, BMI, BSA, or LBW, but the present study may not have been large enough to detect a difference.…”

Section: Discussionsupporting

confidence: 70%

Pentosan Polysulfate Sodium-Associated Pigmentary Retinopathy: Risk Factors and Fundus Findings

Leung¹,

Sharma²,

Levie-Sprick³

et al. 2021

OPTH

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Purpose To evaluate the risk factors and fundus findings of patients with potential PPS-associated retinopathy. Patients and Methods A retrospective chart review was performed of patients exposed to PPS who had a dilated fundus examination at a large retina-only practice from 2018–21. Multimodal images were evaluated by masked reviewers. Results A total of 148 patients were included, of whom 33 (22%) had PPS-associated retinopathy, and 115 (78%) did not. The mean age was 60.3 years old, and the mean follow-up was 11.8 months. The PPS-associated retinopathy group had higher mean cumulative doses of PPS (1600g±849 vs 864g±852, P < 0.0001, Mann–Whitney test) and longer duration of PPS use (13.6 years vs 7.48, P < 0.0001). There was no statistically significant difference based on a history of kidney or liver disease or the dosage per day for the weight, body mass index, body surface area, or lean body weight. Of the patients with PPS-associated retinopathy whose genetic results were available, 15 of 16 (93%) were heterozygous for variants of uncertain significance. Conclusion A longer duration of PPS use and higher cumulative dosage of PPS were associated with an increased risk of developing PPS-associated pigmentary retinopathy. The role of genetic mutations in patients exposed to PPS is still to be determined.

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Section: Discussionsupporting

confidence: 70%

Pentosan Polysulfate Sodium-Associated Pigmentary Retinopathy: Risk Factors and Fundus Findings

Leung¹,

Sharma²,

Levie-Sprick³

et al. 2021

OPTH

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“…The median duration of PPS intake was 168 months (range, 36-262), and the median cumulative PPS exposure was 2.04 kg (range, 0.44-3. 19). All patients reported having discontinued PPS prior to the study visit at a median of 9.9 months (range, 0-137.8) prior.…”

Section: Resultsmentioning

confidence: 99%

“…We do believe that a larger study of all PPS users would identify such a relationship. 5,19 Future studies should continue to evaluate other individual differences with testing results to detect any risk factors for vision loss from PPS maculopathy. The mechanism for PPS toxicity is not well understood, and further research into the mechanism of PPS toxicity may aid in identifying risk factors for disease occurrence and progression.…”

Section: Discussionmentioning

confidence: 99%

Visual Function in Pentosan Polysulfate Sodium Maculopathy

Lyons

Brower

Jain

2020

Invest. Ophthalmol. Vis. Sci.

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Individuals with pentosan polysulfate sodium (PPS) maculopathy commonly report symptoms of prolonged dark adaptation and difficulty reading. We hypothesize that PPS maculopathy causes degradation of visual function not fully captured with visual acuity testing. METHODS. Subjects with PPS maculopathy underwent multimodal evaluation of retinal structure and function. Structural changes were graded as moderate or advanced. Patientreported visual function was assessed with the National Eye Institute Visual Function Questionnaire 39 (NEI-VFQ-39) and Low Luminance Questionnaire (LLQ). Objective functional evaluations included Early Treatment of Diabetic Retinopathy Study (ETDRS) bestcorrected visual acuity (BCVA), Pelli-Robson contrast sensitivity, mesopic microperimetry, and dark adaptometry. Functional testing results were correlated with structural disease category. RESULTS. Thirteen patients (26 eyes), median age 62 years (range, 37-76), completed the study. Median ETDRS letter score was 82 (Snellen equivalent 20/25). Median NEI-VFQ-39 and LLQ composite scores were 65 (range, 33-88) and 41 (range, 20-92), respectively. Median contrast sensitivity was 1.65 (range, 0.15-1.95), and median mesopic microperimetry average thresholds and percent reduced thresholds were 26 decibels (range, 0.4-28.6) and 21.6% (range, 0-100%), respectively. Median rod intercept time was 14.1 minutes (range, 4.4-20.0). Eyes with advanced disease based on retinal structure had significantly worse retinal function for several testing modalities. CONCLUSIONS. PPS maculopathy causes considerable visual function degradation that is not fully captured with BCVA testing. There was good correlation between other measures of visual function and disease severity. These findings deepen our concern regarding this patient safety issue.

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“…Only PPS exposure, and no other IC therapies, was significantly associated with a pigmentary maculopathy. In 2020, Vora and coworkers 24 found that 23 out of 117 patients exposed to at least 500 g of PPS showed signs of maculopathy. Cumulative dose was the only significant risk factor and development of maculopathy varied in a dose-dependent manner.…”

Section: Pentosan Maculopathymentioning

confidence: 99%

Age-Related Macular Degeneration Masquerade: A Review of Pentosan Polysulfate Maculopathy and Implications for Clinical Practice

Mukhopadhyay

Boyce

Gehrs

et al. 2022

Asia-Pacific Journal of Ophthalmology

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Pentosan polysulfate (PPS) sodium (Elmiron) is the only Food and Drug Administration (FDA)–approved oral medication to treat interstitial cystitis, also known as bladder pain syndrome. A symptomatic pigmentary maculopathy associated with PPS was reported in 2018. Since then, recognition of this unique drug toxicity has increased rapidly. This potentially sight-threatening side effect prompted the FDA in June 2020 to update the label for PPS to warn about “retinal pigmentary changes.” A challenging feature of pentosan maculopathy is its ability to mimic many other retinal conditions, including inherited retinal dystrophies such as pattern dystrophy, mitochondrially inherited diabetes and deafness, and Stargardt disease, and age-related macular degeneration. In this review, we discuss the history of PPS maculopathy and its implications for thousands of at-risk interstitial cystitis patients. We use published literature and an illustrative case from our institution to highlight the importance of diagnosing PPS maculopathy. We also compare PPS maculopathy to age-related macular degeneration, explain why differentiating between the 2 is clinically important, and highlight avenues for further research. Finally, we highlight the paucity of data on patients of color and why this lack of understanding may impact patient care.

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Prevalence of Maculopathy Associated with Long-Term Pentosan Polysulfate Therapy

Cited by 52 publications

References 7 publications

Pentosan Polysulfate Sodium-Associated Pigmentary Retinopathy: Risk Factors and Fundus Findings

Pentosan Polysulfate Sodium-Associated Pigmentary Retinopathy: Risk Factors and Fundus Findings

Visual Function in Pentosan Polysulfate Sodium Maculopathy

Age-Related Macular Degeneration Masquerade: A Review of Pentosan Polysulfate Maculopathy and Implications for Clinical Practice

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