Congenital ichthyosis in Prader-Willi syndrome associated with maternal chromosome 15 uniparental disomy: Case report and review of autosomal recessive conditions unmasked by UPD

Am J Med Genet A. 2020 Oct;182(10):2442-2449. doi: 10.1002/ajmg.a.61792. Epub 2020 Aug 20.

Abstract

Prader-Willi syndrome (PWS) is a prototypic genetic condition related to imprinting. Causative mechanisms include paternal 15q11-q13 deletion, maternal chromosome 15 uniparental disomy (UPD15), Prader-Willi Syndrome/Angelman Syndrome (PWS/AS) critical region imprinting defects, and complex chromosomal rearrangements. Maternal UPD15-related PWS poses risks of concomitant autosomal recessive (AR) disorders when the mother carries a pathogenic variant in one of the genes on chromosome 15 associated with autosomal recessive inherited disease. Co-occurrence of autosomal recessive conditions in the setting of UPD leads to increased complexity of the clinical phenotype, and may delay the diagnosis of PWS. We report a patient with PWS and associated congenital ichthyosis due to maternal UPD15, and a homozygous novel pathogenic variant in ceramide synthase 3 (CERS3). We also review the literature of associated disorders reported in the setting of maternal UPD15-related PWS and provide a summary of the previously described CERS3 variants. This represents the second case of autosomal recessive congenital ichthyosis (ARCI) in the setting of PWS and UPD15. There needs to be a high index of suspicion of this genetic mechanism when there is unexpected phenotype or evolution of the clinical course in a patient with PWS.

Keywords: CERS3-related ichthyosis; Prader-Willi syndrome; autosomal recessive disorders; uniparental disomy.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Adult
  • Angelman Syndrome / genetics*
  • Angelman Syndrome / pathology
  • Child
  • Child, Preschool
  • Chromosomes, Human, Pair 15 / genetics
  • Congenital Abnormalities / diagnosis
  • Congenital Abnormalities / genetics
  • Congenital Abnormalities / pathology
  • Female
  • Genes, Recessive / genetics
  • Genomic Imprinting / genetics
  • Humans
  • Ichthyosis / complications
  • Ichthyosis / genetics*
  • Ichthyosis / pathology
  • In Situ Hybridization, Fluorescence
  • Infant
  • Infant, Newborn
  • Maternal Inheritance / genetics
  • Prader-Willi Syndrome / diagnosis
  • Prader-Willi Syndrome / genetics*
  • Prader-Willi Syndrome / pathology
  • Sphingosine N-Acyltransferase / genetics*
  • Uniparental Disomy / diagnosis
  • Uniparental Disomy / genetics
  • Uniparental Disomy / pathology
  • Young Adult

Substances

  • CERS3 protein, human
  • Sphingosine N-Acyltransferase