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. 2020 Aug 1;138(8):894-900.
doi: 10.1001/jamaophthalmol.2020.2349.

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

Rachel Shah  1 , Joseph M Simonett  2 , Riley J Lyons  3 , Rajesh C Rao  4 , Mark E Pennesi  2 , Nieraj Jain  1
Affiliations

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

Rachel Shah et al. JAMA Ophthalmol. .

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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Conflict of interest statement

Conflict of Interest Disclosures: Dr Rao has received grants from the National Institutes of Health and Research to Prevent Blindness as well as research funds from the Leonard G. Miller Endowed Professorship and Ophthalmic Research Fund, Elaine Sandman Research Fund, Grossman Research Fund, and Marek and Maria Spatz Endowed Research Fund. Dr Jain has received grants from Foundation Fighting Blindness. No other disclosures were reported.

Figures

Figure 1.
Figure 1.. Disease Expansion Documented With Fundus Autofluorescence (AF) Imaging
Fundus AF images from the left eye of patient 8 demonstrating expansion in the area of AF abnormality over 11 months after recent discontinuation of pentosan polysulfate sodium (A and B). Imaging in patient 1 demonstrates similar expansion of AF abnormality over 9 months (C and D), several years after drug cessation.
Figure 2.
Figure 2.. Growth of Retinal Pigment Epithelium Atrophy by Multimodal Imaging
Fundus autofluorescence (AF) imaging of the right eye from patient 2 demonstrates growth in area of RPE atrophy from 4 months after drug cessation (A) to 18 months after drug cessation (B). Corresponding optical coherence tomography (OCT) demonstrates progressive atrophy (C) and associated choroidal thinning (D).
Figure 3.
Figure 3.. Retinal Pigment Epithelium Degeneration Documented With Optical Coherence Tomography (OCT)
Serial OCT from the left eye of patient 10, each obtained at the same retinal location, demonstrates evolution of nodular retinal pigment epithelium lesions and progression to impending retinal pigment epithelium atrophy 27 months after drug cessation.
See this image and copyright information in PMC

Comment in

References

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