TY - JOUR
T1 - Pentosan-associated maculopathy
T2 - prevalence, screening guidelines, and spectrum of findings based on prospective multimodal analysis
AU - Wang, Derrick
AU - Au, Adrian
AU - Gunnemann, Frederic
AU - Hilely, Assaf
AU - Scharf, Jackson
AU - Tran, Khoi
AU - Sun, Michel
AU - Kim, Ja Hong
AU - Sarraf, David
N1 - Publisher Copyright:
© 2019
PY - 2020/4
Y1 - 2020/4
N2 - Objective: To describe the prevalence and spectrum of multimodal imaging findings of pentosan polysulfate sodium (PPS)–associated maculopathy and to recommend dosage-related screening guidelines. Design: Cross-sectional study. Methods: Patients previously or currently treated with PPS at University of California, Los Angeles, were randomly ascertained and prospectively screened for PPS-associated maculopathy with multimodal retinal imaging. Daily and cumulative dosages of PPS exposure were calculated for each patient. Images were studied to identify the characteristic findings of toxicity. The prevalence of PPS-associated maculopathy and screening guidelines were determined. Results: The prevalence of PPS-associated maculopathy in this cohort was 20% (10/50 patients). Both average duration of PPS therapy and average cumulative dosage were significantly lower in the unaffected (6.3 ± 6.6 years, 691.7 ± 706.6 g) versus the affected groups (20.3 ± 6.6 years, 3375.4 ± 1650.0 g, p < 0.001). Near-infrared reflectance (NIR) illustrated characteristic punctate retinal pigment epithelium (RPE) macular lesions early. Fundus autofluorescence (FAF) showed speckled autofluorescence in the posterior pole with peripapillary extension. Co-localization with optical coherence tomography (OCT) displayed focal RPE thickening and, in more severe cases, RPE atrophy in the macula and even the periphery. Conclusions: A prevalence of 20% in this study cohort suggests a significant risk of macular toxicity for PPS-treated patients. Characteristic alterations are best detected with FAF and NIR. More significant PPS exposure was associated with more severe atrophy. We recommend an initial baseline eye examination to include OCT and, most importantly, NIR and FAF with annual retinal imaging thereafter especially with cumulative dosages approaching 500 g. Patients exposed to greater than 1500 g of PPS are at significant risk of retinal toxicity.
AB - Objective: To describe the prevalence and spectrum of multimodal imaging findings of pentosan polysulfate sodium (PPS)–associated maculopathy and to recommend dosage-related screening guidelines. Design: Cross-sectional study. Methods: Patients previously or currently treated with PPS at University of California, Los Angeles, were randomly ascertained and prospectively screened for PPS-associated maculopathy with multimodal retinal imaging. Daily and cumulative dosages of PPS exposure were calculated for each patient. Images were studied to identify the characteristic findings of toxicity. The prevalence of PPS-associated maculopathy and screening guidelines were determined. Results: The prevalence of PPS-associated maculopathy in this cohort was 20% (10/50 patients). Both average duration of PPS therapy and average cumulative dosage were significantly lower in the unaffected (6.3 ± 6.6 years, 691.7 ± 706.6 g) versus the affected groups (20.3 ± 6.6 years, 3375.4 ± 1650.0 g, p < 0.001). Near-infrared reflectance (NIR) illustrated characteristic punctate retinal pigment epithelium (RPE) macular lesions early. Fundus autofluorescence (FAF) showed speckled autofluorescence in the posterior pole with peripapillary extension. Co-localization with optical coherence tomography (OCT) displayed focal RPE thickening and, in more severe cases, RPE atrophy in the macula and even the periphery. Conclusions: A prevalence of 20% in this study cohort suggests a significant risk of macular toxicity for PPS-treated patients. Characteristic alterations are best detected with FAF and NIR. More significant PPS exposure was associated with more severe atrophy. We recommend an initial baseline eye examination to include OCT and, most importantly, NIR and FAF with annual retinal imaging thereafter especially with cumulative dosages approaching 500 g. Patients exposed to greater than 1500 g of PPS are at significant risk of retinal toxicity.
UR - http://www.scopus.com/inward/record.url?scp=85077986794&partnerID=8YFLogxK
U2 - 10.1016/j.jcjo.2019.12.001
DO - 10.1016/j.jcjo.2019.12.001
M3 - Article
C2 - 31973791
AN - SCOPUS:85077986794
SN - 0008-4182
VL - 55
SP - 116
EP - 125
JO - Canadian Journal of Ophthalmology
JF - Canadian Journal of Ophthalmology
IS - 2
ER -