Pentosan Polysulfate Maculopathy: Final Outcomes from a 4-Year Prospective Study of Disease Progression after Drug Cessation - 13/02/25
Highlights |
• | This study investigated pentosan polysulfate maculopathy after drug cessation. |
• | Retinal function and structure exhibited significant decline. |
• | Five eyes developed new-onset atrophy years after drug cessation. |
• | Atrophic areas showed a linearized growth rate of 0.23 mm/year. |
• | Future research is needed to identify therapies that slow disease progression. |
Résumé |
Purpose |
To report the long-term disease course of pentosan polysulfate (PPS) maculopathy following drug cessation.
Design |
Single-institution, prospective case series.
Methods |
23 eyes of 12 participants seen at the Emory Eye Center with a diagnosis of PPS maculopathy were included in our study. Participants were enrolled between December 1, 2018, and December 1, 2019, and data were collected annually for four years.
Main Outcomes and Measures |
Changes in visual function and retinal structure were the primary outcomes measured. Visual function was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA), ETDRS low-luminance visual acuity (LLVA), Minnesota low-vision reading (MNREAD) performance, contrast sensitivity, mesopic and scotopic microperimetry, and dark adaptometry. Patient reported outcomes were assessed with the National Eye Institute Visual Function Questionnaire (NEI-VFQ-39) and Low Luminance Questionnaire (LLQ). Structural outcomes included the presence of complete retinal pigment epithelium and outer retinal atrophy (cRORA), atrophic lesion size (in mm 2 ), macular central subfield thickness (CST), and subfoveal choroidal thickness (SFCT).
Results |
Of the 12 participants, 11 (91.7%) were female, with a median age at enrollment of 58 years. The median ETDRS BCVA letter score at baseline was 83, with a median change of -5 letters over 4 years ( P = 0.005). The median 4-year change in mesopic microperimetry average threshold and percent reduced threshold was -5.4 dB ( P = 0.003) and 48.6% ( P = 0.004), respectively. MNREAD performance (assessed at 2 and 4 years) declined across all measures, with a median maximum reading speed change of -21 words per minute ( P = 0.007). NEI-VFQ-39 and LLQ composite scores significantly decreased over 4 years. At baseline, 9 eyes (39%) had macular cRORA. By the study's end, 5 of the remaining eyes (35.7%) developed new-onset cRORA. The median linearized growth rate of atrophic lesions was 0.23 mm/year. The median 4-year change of CST and SFCT was -7.0 µm ( P = 0.055) and -22.0 µm ( P = 0.610), respectively.
Conclusion |
This prospective study demonstrates continued progression of broad-ranging functional and structural deficits in PPS maculopathy long after drug cessation. These findings should inform PPS prescribing patterns, patient counseling, and disease monitoring strategies.
Le texte complet de cet article est disponible en PDF.Plan
Vol 271
P. 371-380 - mars 2025 Retour au numéro
Article précédent
- Visual Field Progression in the Ocular Hypertension Treatment Study
- Mae O. Gordon, Dale K. Heuer, Eve J. Higginbotham, Richard K. Parrish, Lei Liu, James D. Brandt, Julia B. Huecker, J. Phillip Miller, Chamila Perera, Chris Xie, John L. Keltner, Chris A. Johnson, Stuart K. Gardiner, Jeffrey M. Liebmann, Michael A. Kass
- High Body Mass Index is Associated with Lower Adalimumab Serum Levels and Higher Disease Activity in Noninfectious Uveitis
- Francesco Pichi, Sahar H AlAli, Yanny Perez Jimenez, Piergiorgio Neri
Bienvenue sur EM-consulte, la référence des professionnels de santé.
L’accès au texte intégral de cet article nécessite un abonnement.
Déjà abonné à cette revue ?