Abstract
Purpose :
Long term use of pentosan polysulfate (PPS) is associated with a distinctive vision-threatening maculopathy. We are conducting a prospective study to assess the long-term impact of PPS maculopathy on retinal function and structure following drug cessation; herein, we report the 3-year outcomes.
Methods :
Thirteen participants with PPS maculopathy were evaluated annually with multimodal assessments of retinal function and structure. Twelve participants (23 eyes) completed the 3-year visit. Functional outcomes included Early Treatment of Diabetic Retinopathy Study (ETDRS) best corrected visual acuity (BCVA) and mesopic microperimetry average threshold and percent reduced threshold (PRT). Structural outcomes included macular central subfield thickness (CST) and presence and extent of complete retinal pigment epithelium and outer retinal atrophy (cRORA). Descriptive statistics, including all medians and IQRs, were reported on a per-eye basis, and to account for high inter-eye correlation, p-values were calculated on a per-participant basis using two-sided Wilcoxon signed rank tests.
Results :
Eleven participants (92%) were female, and median (IQR) age at baseline was 58 y (47-64 y). Median time between drug cessation and initial study visit was 0.6 y (0.4-1.9 y). Median ETDRS BCVA letter score was 83 (80-87) at baseline and 82 (75-85) at 3-y, with a median change of -3 (-7 to 1; p=0.2). Median microperimetry average threshold was 26.2 dB (25-27.7 dB) at baseline and 23.4 dB (19.2-25 dB) at 3-y, with a median change of -3.8 dB (-5.5 to -2.6 dB; p<0.01). Median microperimetry PRT was 18.9% (10.8-29.7%) at baseline and 64.9% (48.6-94.6%) at 3-y, with a median change of 37.8% (23-54.1%; p<0.01). Median CST was 284 μm (253-291 μm) at baseline and 273 μm (234-294 μm) at 3-y, with a median change of -8 μm (-13 to 5 μm; p=0.02). At baseline, 9 eyes had cRORA, which continued to expand in all affected eyes over the 3 years (Figure), with a median linearized growth rate of 0.21 mm/y (0.15-0.23 mm/y). Four eyes without atrophy at baseline developed new-onset cRORA by 3-y.
Conclusions :
This study demonstrates continued progression of deficits in retinal function and structure in patients with PPS maculopathy for over 3 years after drug cessation.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.