Insights into levator muscle dysfunction in a cohort of patients with molecularly confirmed blepharophimosis-ptosis-epicanthus inversus syndrome using high-resolution imaging, anatomic examination, and histopathologic examination

Arch Ophthalmol. 2011 Dec;129(12):1564-9. doi: 10.1001/archophthalmol.2011.348.

Abstract

Objective: To study the basis of defective levator palpebrae superioris (LPS) function in blepharophimosis-ptosis-epicanthus inversus syndrome (BPES), an autosomal dominant eyelid malformation sometimes associated with ovarian dysfunction.

Methods: Eight patients with molecularly proved BPES underwent high-resolution surface-coil 3-T magnetic resonance imaging before surgical intervention. The features of LPS muscle and adjoining connective tissue were compared with an age-matched control subject. During LPS resection for ptosis repair, detailed anatomic examination of the LPS was performed. Histopathologic characteristics were compared with normal control samples from a cadaver and a patient with simple severe congenital ptosis.

Results: The most striking feature shown on magnetic resonance imaging was the thin, long anterior part of the LPS. During the operation, this consisted of a disorganized, thin, long aponeurosis. However, in the posterior part of the LPS, there was an organized thick structure suggestive of a muscle belly. Histopathologic examination revealed posteriorly well-formed striated muscle fibers in all patients with BPES but not in the control sample from the patient with simple severe congenital ptosis. These striated muscle fibers were comparable to those of the normal control tissue but were more intermixed with collagenous tissue and little fatty degeneration.

Conclusions: The presence of striated muscle fibers in LPS of patients with BPES contrasts with the fatty degeneration in patients with simple severe congenital ptosis. To our knowledge, this is the first study providing novel insights into the pathogenesis of the eyelid malformation in BPES through extensive imaging, anatomic study, and histopathologic testing in a unique cohort of patients with molecularly proved BPES.

MeSH terms

  • Adolescent
  • Blepharophimosis / genetics
  • Blepharophimosis / physiopathology*
  • Blepharophimosis / surgery
  • Blepharoptosis / congenital
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Eyelids / abnormalities
  • Female
  • Forkhead Box Protein L2
  • Forkhead Transcription Factors / genetics
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Menopause, Premature / genetics
  • Oculomotor Muscles / physiopathology*
  • Oculomotor Muscles / surgery
  • Skin Abnormalities / genetics
  • Skin Abnormalities / physiopathology*
  • Skin Abnormalities / surgery

Substances

  • FOXL2 protein, human
  • Forkhead Box Protein L2
  • Forkhead Transcription Factors

Supplementary concepts

  • Blepharophimosis syndrome type 1