Prenatal and postnatal findings in serpentine fibula polycystic kidney syndrome and a review of the NOTCH2 spectrum disorders

Am J Med Genet A. 2014 Oct;164A(10):2490-5. doi: 10.1002/ajmg.a.36656. Epub 2014 Jul 3.

Abstract

Serpentine fibula polycystic kidney syndrome (SFPKS; OMIM600330) is a rare skeletal dysplasia with a characteristic phenotype that includes polycystic kidneys, S-shaped fibulas, and abnormal craniofacial features. SFPKS shares features with Alagille (AGS; OMIM) and Hajdu-Cheney (HCS; OMIM10250) syndromes. All three syndromes result from mutations in the gene that encodes NOTCH2, one of the receptors involved in Notch signaling. Notch signaling is a major developmental signaling pathway, as well as a key regulator of numerous cellular processes. In this report, we present the prenatal ultrasound and postnatal findings in a 23-week fetus with severe manifestations of SPKS and heterozygosity for a de novo mutation in exon 34 of NOTCH2. These findings expand the phenotypic spectrum of NOTCH2 mutations and demonstrate the findings in the prenatal period.

Keywords: Hadju-Cheney; NOTCH2; alagille syndrome; prenatal ultrasound; serpentine fibula polycystic kidney syndrome.

Publication types

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

MeSH terms

  • Exons / genetics
  • Fetus / pathology
  • Hajdu-Cheney Syndrome / genetics*
  • Hajdu-Cheney Syndrome / pathology*
  • Heterozygote
  • Humans
  • Mutation / genetics
  • Prenatal Diagnosis / methods
  • Receptor, Notch2 / genetics*
  • Receptors, Notch / genetics
  • Signal Transduction / genetics

Substances

  • NOTCH2 protein, human
  • Receptor, Notch2
  • Receptors, Notch

Supplementary concepts

  • Serpentine fibula polycystic kidney syndrome