Prenatal and postnatal presentations of corpus callosum agenesis with polymicrogyria caused by EGP5 mutation

Am J Med Genet A. 2017 Mar;173(3):706-711. doi: 10.1002/ajmg.a.38061. Epub 2017 Feb 7.

Abstract

EPG5-related Vici syndrome is a rare multisystem autosomal recessive disorder characterized by corpus callosum agenesis (ACC), hypopigmentation, cataracts, acquired microcephaly, failure to thrive, cardiomyopathy and profound developmental delay, and immunodeficiency. We report here the first case of prenatally diagnosed Vici syndrome with delayed gyration associated with ACC. Trio based exome sequencing allowed the identification of a compound heterozygous mutation in the EPG5 gene. Our patient subsequently demonstrated severe developmental delay, hypopigmentation, progressive microcephaly, and failure to thrive which led to suspicion of the diagnosis. Her MRI demonstrated ACC with frontoparietal polymicrogyria, severe hypomyelination, and pontocerebellar atrophy. This prenatal presentation of malformations of cortical development in combination with ACC expands the EPG5-related phenotypic spectrum. Our report supports the idea that EPG5-related Vici syndrome is both a neurodevelopmental and neurodegenerative disorder. © 2017 Wiley Periodicals, Inc.

Keywords: autophagy; EPG5; corpus callosum agenesis; microcephaly; migration disorder.

Publication types

  • Case Reports

MeSH terms

  • Agenesis of Corpus Callosum / diagnosis*
  • Agenesis of Corpus Callosum / genetics*
  • Autophagy-Related Proteins
  • Exome
  • Female
  • Genetic Association Studies
  • Genetic Testing
  • Genotype
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Lysosomal Membrane Proteins
  • Magnetic Resonance Imaging
  • Male
  • Mutation*
  • Phenotype*
  • Polymicrogyria / diagnosis*
  • Polymicrogyria / genetics*
  • Pregnancy
  • Prenatal Diagnosis
  • Proteins / genetics*
  • Ultrasonography
  • Vesicular Transport Proteins

Substances

  • Autophagy-Related Proteins
  • EPG5 protein, human
  • Lysosomal Membrane Proteins
  • Proteins
  • Vesicular Transport Proteins