Hyperinsulinism in an individual with an EP300 variant of Rubinstein-Taybi syndrome

Am J Med Genet A. 2021 Apr;185(4):1251-1255. doi: 10.1002/ajmg.a.62085. Epub 2021 Jan 14.

Abstract

Rubinstein-Taybi syndrome (RSTS) is an autosomal dominant genetic syndrome characterized by distinct facial features, broad thumbs, growth restriction, microcephaly, intellectual disability, and developmental delay. Pathogenic variants in both CREBBP and EP300 have been associated with RSTS. Here we present a case of a female with hyperinsulinism and features consistent with RSTS, found to have a pathogenic variant in EP300. While there have been a few rare case reports of hyperinsulinism in RSTS, we suggest that hyperinsulinism might be a more prominent feature in EP300 variant RSTS than previously recognized.

Keywords: EP300; Rubinstein Taybi; hyperinsulinism; hypoglycemia.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural

MeSH terms

  • E1A-Associated p300 Protein / genetics*
  • Female
  • Genetic Predisposition to Disease*
  • Genetic Variation / genetics
  • Genotype
  • Humans
  • Hyperinsulinism / genetics*
  • Hyperinsulinism / pathology
  • Infant
  • Infant, Newborn
  • Mutation / genetics
  • Phenotype
  • Rubinstein-Taybi Syndrome / genetics*
  • Rubinstein-Taybi Syndrome / pathology
  • Sequence Deletion / genetics

Substances

  • E1A-Associated p300 Protein
  • EP300 protein, human