Pentosan Polysulfate Maculopathy: Final Outcomes from a 4-Year Prospective Study of Disease Progression after Drug Cessation.

IF 4.1 1区 医学 Q1 OPHTHALMOLOGY
Brian P Hall, Sakshi Shiromani, Emily H Jung, Riley J Lyons, Judith Tribe, Nieraj Jain
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引用次数: 0

Abstract

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 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 mm2), 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.

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来源期刊
CiteScore
9.20
自引率
7.10%
发文量
406
审稿时长
36 days
期刊介绍: The American Journal of Ophthalmology is a peer-reviewed, scientific publication that welcomes the submission of original, previously unpublished manuscripts directed to ophthalmologists and visual science specialists describing clinical investigations, clinical observations, and clinically relevant laboratory investigations. Published monthly since 1884, the full text of the American Journal of Ophthalmology and supplementary material are also presented online at www.AJO.com and on ScienceDirect. The American Journal of Ophthalmology publishes Full-Length Articles, Perspectives, Editorials, Correspondences, Books Reports and Announcements. Brief Reports and Case Reports are no longer published. We recommend submitting Brief Reports and Case Reports to our companion publication, the American Journal of Ophthalmology Case Reports. Manuscripts are accepted with the understanding that they have not been and will not be published elsewhere substantially in any format, and that there are no ethical problems with the content or data collection. Authors may be requested to produce the data upon which the manuscript is based and to answer expeditiously any questions about the manuscript or its authors.
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