June 2020
Volume 61, Issue 7
ARVO Annual Meeting Abstract  |   June 2020
Pentosan Polysulfate Maculopathy: Disease Course after Drug Cessation
Author Affiliations & Notes
  • Rachel Shah
    Emory, Atlanta, Georgia, United States
  • Riley Lyons
    Emory, Atlanta, Georgia, United States
  • Joseph Simonett
    Oregon Health & Science University, Oregon, United States
  • Mark E Pennesi
    Oregon Health & Science University, Oregon, United States
  • Rajesh C Rao
    University of Michigan, Michigan, United States
  • Nieraj Jain
    Emory, Atlanta, Georgia, United States
  • Footnotes
    Commercial Relationships   Rachel Shah, None; Riley Lyons, None; Joseph Simonett, None; Mark Pennesi, None; Rajesh Rao, None; Nieraj Jain, None
  • Footnotes
    Support  Foundation Fighting Blindness Career Development Award CD-C-0918-0748-EEC (Nieraj Jain)
Investigative Ophthalmology & Visual Science June 2020, Vol.61, 1063. doi:
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      Rachel Shah, Riley Lyons, Joseph Simonett, Mark E Pennesi, Rajesh C Rao, Nieraj Jain; Pentosan Polysulfate Maculopathy: Disease Course after Drug Cessation. Invest. Ophthalmol. Vis. Sci. 2020;61(7):1063.

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      © ARVO (1962-2015); The Authors (2016-present)

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Purpose : This study aims to evaluate long-term outcomes, both structural changes as seen on multimodal imaging, and functional outcomes, after drug cessation in subjects with confirmed PPS maculopathy.

Methods : This retrospective chart review across three institutions (Emory Eye Center, Casey Eye Institute, and Kellogg Eye Center) identified subjects with a diagnosis of PPS maculopathy who reported discontinuation of PPS use. Patients with at least 6 months of follow-up and at least two visits while off the medication were included. Twelve patients were included in the study. Expert image reviewers assessed retinal imaging characteristics across consecutive follow-up visits to assess structural changes over time. Change in visual acuity as well as subjective visual complaints were also evaluated over the course of follow-up.

Results : Twelve subjects, all female, with a median age of 57 years (range, 37 - 74 years), met the eligibility criteria. Patients had a baseline visit at a median of 2.5 months (range, 0 - 480 months) after drug cessation, and were subsequently followed for a median of 12.6 months (range, 7 - 65 months). The median cumulative exposure was 1.81 kg. No eyes exhibited a demonstrable improvement in retinal structure or function while off PPS. Ten subjects (83%) reported worsening of visual symptoms at the final visit. Visual acuity declined by greater than 1 line in two eyes of two subjects (8%). All eyes demonstrated evolution in the pattern of fundus autofluorescence (FAF) abnormalities, and a majority of eyes demonstrated subtle expansion of the area of involved retina noted on FAF imaging. Seven (29%) eyes had macular RPE atrophy at the baseline visit, and atrophy enlarged in all seven eyes (100%). Two eyes (8%) of one subject developed new onset macular RPE atrophy while off PPS.

Conclusions : PPS maculopathy continues to evolve even after drug cessation. Areas of RPE atrophy continue to grow, coalesce, and encroach on the foveal center. This may pose a long-term threat to central vision, even remote from the time of medication use. Affected patients should be counseled appropriately, and screening programs should be instituted to promote early detection.

This is a 2020 ARVO Annual Meeting abstract.


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