Expanded clinical spectrum of enhanced S-cone syndrome

JAMA Ophthalmol. 2013 Oct;131(10):1324-30. doi: 10.1001/jamaophthalmol.2013.4349.

Abstract

Importance: New funduscopic findings in patients with enhanced S-cone syndrome (ESCS) may help clinicians in diagnosing this rare autosomal recessive retinal dystrophy.

Objective: To expand the clinical spectrum of ESCS due to mutations in the NR2E3 gene.

Design: Retrospective, noncomparative case series of 31 patients examined between 1983 and 2012.

Setting: Academic and private ophthalmology practices specialized in retinal dystrophies.

Participants: A cohort of patients diagnosed with ESCS and harboring known NR2E3 mutations.

Intervention: Patients had ophthalmic examinations including visual function testing that led to the original diagnosis.

Main outcomes and measures: New fundus features captured with imaging modalities.

Results: New clinical observations in ESCS include (1) torpedo-like, deep atrophic lesions with a small hyperpigmented rim, variably sized and predominantly located along the arcades; (2) circumferential fibrotic scars in the posterior pole with a spared center and large fibrotic scars around the optic nerve head; and (3) yellow dots in areas of relatively normal-appearing retina.

Conclusions and relevance: Enhanced S-cone syndrome has more pleiotropy than previously appreciated. While the nummular type of pigmentation at the level of the retinal pigment epithelium and cystoid or schisis-like maculopathy with typical functional findings remain classic hallmarks of the disease, changes such as circumferential fibrosis of the macula or peripapillary area and "torpedo-like" lesions along the vascular arcades may also direct the clinical diagnosis and focus on screening the NR2E3 gene for a molecular diagnosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Child
  • Child, Preschool
  • Electroretinography
  • Eye Diseases, Hereditary / diagnosis*
  • Eye Diseases, Hereditary / genetics
  • Female
  • Fibrosis
  • Fluorescein Angiography
  • Humans
  • Male
  • Middle Aged
  • Orphan Nuclear Receptors / genetics
  • Photic Stimulation
  • Retinal Cone Photoreceptor Cells / pathology*
  • Retinal Degeneration / diagnosis*
  • Retinal Degeneration / genetics
  • Retinal Pigment Epithelium / pathology*
  • Retrospective Studies
  • Tomography, Optical Coherence
  • Vision Disorders / diagnosis*
  • Vision Disorders / genetics
  • Visual Acuity
  • Young Adult

Substances

  • NR2E3 protein, human
  • Orphan Nuclear Receptors

Supplementary concepts

  • Enhanced S-Cone Syndrome