Pediatric mastocytosis is a clonal disease associated with D816V and other activating c-KIT mutations

J Invest Dermatol. 2010 Mar;130(3):804-15. doi: 10.1038/jid.2009.281. Epub 2009 Oct 29.

Abstract

Adult mastocytosis is an incurable clonal disease associated with c-KIT mutations, mostly in exon 17 (D816V). In contrast, pediatric mastocytosis often spontaneously regresses and is considered a reactive disease. Previous studies on childhood mastocytosis assessed only a few patients and focused primarily on codon 816 mutations, with various results. In this study, we analyzed the entire c-KIT sequence from cutaneous biopsies of 50 children with mastocytosis (ages 0-16 years). A mutation of codon 816 (exon 17) was found in 42% of cases, and mutations outside exon 17 were observed in 44%. Unexpectedly, half of the mutations were located in the fifth Ig loop of c-KIT's extracellular domain, which is encoded by exons 8 and 9. All mutations identified in this study were somatic and caused a constitutive activation of c-KIT. There was no clear phenotype-genotype correlation, no clear relationship between the mutations and familial versus spontaneous disease, and no significant change in the relative expression of the c-KIT GNNK+ and GNNK isoforms. These findings strongly support the idea that, although pediatric mastocytosis can spontaneously regress, it is a clonal disease most commonly associated with activating mutations in c-KIT.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Age of Onset
  • Animals
  • Biopsy
  • COS Cells
  • Child
  • Child, Preschool
  • Chlorocebus aethiops
  • Clone Cells
  • Exons / genetics
  • Female
  • Genomics
  • Genotype
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Mast Cells / pathology*
  • Mast Cells / physiology
  • Mastocytosis, Cutaneous / genetics*
  • Mastocytosis, Cutaneous / pathology*
  • Phenotype
  • Point Mutation*
  • Proto-Oncogene Proteins c-kit / genetics*

Substances

  • Proto-Oncogene Proteins c-kit