Abstract
Purpose :
Recent studies have demonstrated an association between long-term pentosan polysulfate (PPS) use and a novel maculopathy. No study has prospectively evaluated long-term disease course after drug cessation. Here, we report year 1 outcomes of a long-term prospective study of retinal structure and function in PPS maculopathy.
Methods :
Thirteen PPS maculopathy patients were followed prospectively with multimodal assessments of retinal structure and function; 11 (22 eyes) completed year 1 visits. Functional testing endpoints at year 1 included: microperimetry (MP) mean and percent reduced thresholds, and ETDRS best corrected visual acuity (BCVA). Structural endpoints included: central subfield retinal thickness (CST), subfoveal choroidal thickness (SFCT), and geographic atrophy area (defined as complete retinal and outer retinal atrophy). Baseline and year 1 outcomes were compared using a Wilcoxon signed rank test. To account for inter-eye correlation, mean results were computed for the two eyes for each patient.
Results :
Patients had a median age of 63 (range 38-77) and median 2.04 kg (IQR 1.15 – 2.58) PPS exposure. Patients stopped PPS use a median of 9.9 (IQR 5.6 – 20.5) months prior to baseline. Median change in ETDRS BCVA during the study period was -3 letters (IQR -4.75 – 1.5) [baseline: 81.5 letters (IQR 79 – 86.3), year 1: 78.5 letters (IQR 74.5 – 86.8) (p = 0.07)]. Seven of 11 (64%) patients had BCVA decline. Four (18%) eyes, each with progressive atrophy, lost ≥ 10 letters. Median MP average thresholds declined from 25.7 (IQR 17.8 – 26.7) to 25.2 (IQR 16.5 – 27.6) (p = 0.18). Median MP percent reduced thresholds increased from median 23.0% (IQR 9.5% – 54.1%) to 25.2% (IQR 14.2% – 28.6%) (p = 0.88). Fifteen eyes (68%) had atrophy at baseline; with an increase from a median of 5.64 mm2 (IQR 0.36 – 17.9) to a median of 7.23 mm2 (IQR 0.45 – 20.8) (p < 0.01) and median linearized increase of 0.21 mm (IQR 0.08 – 0.39) per eye. Median CST decreased from 278 μm (IQR 243 – 287) to 267 μm (IQR 238 – 290) (p = 0.08). Median SFCT decreased from 268 μm (IQR 159 – 324) to 261 μm (IQR 161 - 334) (p = 0.47).
Conclusions :
This prospective study demonstrates continued evolution of structural and functional deficits in patients with PPS maculopathy even after drug cessation. Geographic atrophy enlarged in all eyes with atrophy present at baseline, and thus may pose a long-term threat to central vision.
This is a 2021 ARVO Annual Meeting abstract.