Germline PTPN11 and somatic PIK3CA variant in a boy with megalencephaly-capillary malformation syndrome (MCAP)--pure coincidence?

Eur J Hum Genet. 2015 Mar;23(3):409-12. doi: 10.1038/ejhg.2014.118. Epub 2014 Jun 18.

Abstract

Megalencephaly-capillary malformation (MCAP) syndrome is an overgrowth syndrome that is diagnosed by clinical criteria. Recently, somatic and germline variants in genes that are involved in the PI3K-AKT pathway (AKT3, PIK3R2 and PIK3CA) have been described to be associated with MCAP and/or other related megalencephaly syndromes. We performed trio-exome sequencing in a 6-year-old boy and his healthy parents. Clinical features were macrocephaly, cutis marmorata, angiomata, asymmetric overgrowth, developmental delay, discrete midline facial nevus flammeus, toe syndactyly and postaxial polydactyly--thus, clearly an MCAP phenotype. Exome sequencing revealed a pathogenic de novo germline variant in the PTPN11 gene (c.1529A>G; p.(Gln510Arg)), which has so far been associated with Noonan, as well as LEOPARD syndrome. Whole-exome sequencing (>100 × coverage) did not reveal any alteration in the known megalencephaly genes. However, ultra-deep sequencing results from saliva (>1000 × coverage) revealed a 22% mosaic variant in PIK3CA (c.2740G>A; p.(Gly914Arg)). To our knowledge, this report is the first description of a PTPN11 germline variant in an MCAP patient. Data from experimental studies show a complex interaction of SHP2 (gene product of PTPN11) and the PI3K-AKT pathway. We hypothesize that certain PTPN11 germline variants might drive toward additional second-hit alterations.

MeSH terms

  • Abnormalities, Multiple / diagnosis*
  • Abnormalities, Multiple / genetics*
  • Child
  • Class I Phosphatidylinositol 3-Kinases
  • Comparative Genomic Hybridization
  • Consanguinity
  • Exome
  • Genetic Variation*
  • Germ-Line Mutation*
  • High-Throughput Nucleotide Sequencing
  • Humans
  • Male
  • Megalencephaly / diagnosis*
  • Megalencephaly / genetics*
  • Models, Biological
  • Pedigree
  • Phenotype
  • Phosphatidylinositol 3-Kinases / genetics*
  • Protein Tyrosine Phosphatase, Non-Receptor Type 11 / genetics*
  • Skin Diseases, Vascular / diagnosis*
  • Skin Diseases, Vascular / genetics*
  • Telangiectasis / congenital*
  • Telangiectasis / diagnosis
  • Telangiectasis / genetics

Substances

  • Phosphatidylinositol 3-Kinases
  • Class I Phosphatidylinositol 3-Kinases
  • PIK3CA protein, human
  • PTPN11 protein, human
  • Protein Tyrosine Phosphatase, Non-Receptor Type 11

Supplementary concepts

  • Megalencephaly cutis marmorata telangiectatica congenita