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Original Investigation
July 9, 2020

Disease Course in Patients With Pentosan Polysulfate Sodium–Associated Maculopathy After Drug Cessation

Author Affiliations
  • 1Department of Ophthalmology, Emory University School of Medicine, Atlanta, Georgia
  • 2Department of Ophthalmology, Oregon Health & Science University Casey Eye Institute, Portland
  • 3Emory University School of Medicine, Atlanta, Georgia
  • 4Department of Ophthalmology and Visual Sciences, University of Michigan Kellogg Eye Center, Ann Arbor
JAMA Ophthalmol. Published online July 9, 2020. doi:10.1001/jamaophthalmol.2020.2349
Key Points

Question  How does the maculopathy associated with pentosan polysulfate sodium evolve after drug cessation?

Findings  In this case series of 11 adults observed for at least 6 months after pentosan polysulfate sodium cessation, there was no evidence of disease regression in any eye. Fundus autofluorescence imaging demonstrated expansion of the area of involved tissue in most eyes, including area of retinal pigment epithelium atrophy, and optical coherence tomography demonstrated collapse of nodular lesions at the level of the retinal pigment epithelium.

Meaning  These findings suggest that the maculopathy associated with pentosan polysulfate sodium may continue to evolve after drug cessation.

Abstract

Importance  Recent studies have linked a vision-threatening maculopathy with long-term use of pentosan polysulfate sodium (PPS).

Objective  To evaluate the disease course in PPS-associated maculopathy after drug cessation.

Design, Setting, and Participants  In this retrospective case series, patients diagnosed with PPS-associated maculopathy with at least 6 months of follow-up after drug cessation who were treated at the Emory Eye Center, Atlanta, Georgia, or the Casey Eye Institute, Portland, Oregon, were included. Data were collected from April 2014 through November 2019.

Main Outcomes and Measures  Change in visual acuity and retinal imaging characteristics over time.

Results  Of the 11 included patients, all were female, and the median (interquartile range [IQR]) age was 53 (44-63) years. Participants had a baseline visit at a median (IQR) of 2 (0-4) months after drug cessation and were subsequently observed for a median (IQR) of 12 (8-26) months. The median (IQR) cumulative PPS exposure was 1.97 (1.55-2.18) kg. No eyes exhibited a demonstrable improvement in disease after discontinuing PPS. A total of 9 of 11 patients (82%) reported worsening visual symptoms at the final visit. The mean (SD) logMAR visual acuity was 0.14 (0.23) and 0.14 (0.34) at the baseline and final visit, respectively. Visual acuity improved by 2 or more Snellen lines in 1 eye (5%) and declined by 2 or more Snellen lines in 2 eyes of 1 patient (9%). There was evolution in the pattern of fundus autofluorescence changes and/or optical coherence tomography findings in all eyes. A total of 17 eyes (77%) exhibited expansion of the area of involved tissue. A total of 7 eyes (32%) had macular retinal pigment epithelium atrophy at the baseline visit, and atrophy enlarged after discontinuation of PPS in all 7 eyes, with a median (IQR) growth rate of 0.32 (0.13-0.38) mm per year.

Conclusions and Relevance  These retrospective data among 11 patients suggest PPS-associated maculopathy continues to evolve after drug cessation for at least 10 years. In some cases, progressive retinal pigment epithelium atrophy encroaches on the foveal center and thus may pose a long-term threat to central vision.

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