Loss of SMARCB1/INI1 expression in poorly differentiated chordomas

Acta Neuropathol. 2010 Dec;120(6):745-53. doi: 10.1007/s00401-010-0767-x. Epub 2010 Oct 30.

Abstract

Chordomas are malignant neoplasms that typically arise in the axial spine and primarily affect adults. When chordomas arise in pediatric patients they are more likely to display unusual histological features and aggressive behavior. We noted the absence of SMARCB1/INI1 expression by immunohistochemistry in an index case of poorly differentiated chordoma of the sacrum, leading us to further examine SMARCB1/INI1 expression as well as that of brachyury, a highly specific marker of notochordal differentiation, in 3 additional poorly differentiated chordomas of the clivus, 10 typical chordomas, and 8 atypical teratoid/rhabdoid tumors (AT/RTs). All 4 poorly differentiated chordomas and all AT/RTs lacked nuclear expression of SMARCB1/INI1, while the 10 typical chordomas maintained strong nuclear SMARCB1/INI1 immunoreactivity. All 10 typical and 4 poorly differentiated chordomas expressed brachyury; all 8 AT/RTs were brachyury immunonegative. Cytogenetic evaluation utilizing FISH probes near the SMARCB1/INI1 locus on chromosome 22q was also performed in all of the poorly differentiated chordomas in this series. Three of the four poorly differentiated chordomas had evidence for deletion of this region by FISH. Analysis of the SMARCB1/INI1 gene sequence was performed using formalin-fixed paraffin-embedded tissue in all cases and no point mutations were observed. In summary, all poorly differentiated chordomas in this series showed the absence of SMARCB1/INI1 expression, and were reliably distinguished from AT/RTs, clinically by their characteristic primary sites of origin and pathologically by strong nuclear brachyury expression. Our findings reveal a likely role for SMARCB1/INI1 in a subset of chordomas with aggressive features.

MeSH terms

  • Cell Differentiation / genetics
  • Child
  • Child, Preschool
  • Chordoma / genetics*
  • Chordoma / metabolism
  • Chordoma / pathology*
  • Chromosomal Proteins, Non-Histone / antagonists & inhibitors*
  • Chromosomal Proteins, Non-Histone / biosynthesis*
  • Chromosomal Proteins, Non-Histone / genetics
  • DNA-Binding Proteins / antagonists & inhibitors*
  • DNA-Binding Proteins / biosynthesis*
  • DNA-Binding Proteins / genetics
  • Female
  • Gene Deletion*
  • Humans
  • Infant
  • Male
  • Mutation / genetics*
  • Neoplasm Invasiveness / genetics
  • Neoplasm Invasiveness / pathology
  • SMARCB1 Protein
  • Spinal Neoplasms / genetics*
  • Spinal Neoplasms / metabolism
  • Spinal Neoplasms / pathology*
  • Transcription Factors / antagonists & inhibitors*
  • Transcription Factors / biosynthesis*
  • Transcription Factors / genetics

Substances

  • Chromosomal Proteins, Non-Histone
  • DNA-Binding Proteins
  • SMARCB1 Protein
  • SMARCB1 protein, human
  • Transcription Factors