Biallelic PMS2 mutations and a distinctive childhood cancer syndrome

J Pediatr Hematol Oncol. 2008 Mar;30(3):254-7. doi: 10.1097/MPH.0b013e318161aa20.

Abstract

Biallelic mutations in PMS2, a gene usually associated in heterozygous form with hereditary nonpolyposis colorectal cancer (HNPCC), results in a recently described childhood cancer syndrome. The tumor spectrum encompasses atypical brain cancers, hematologic malignancies, and colonic polyposis and cancer. Cutaneous stigmata resembling café-au-lait macules with more diffuse margins are frequently seen. Onset is as young as 2 years. The risk of second malignancy is high. Evidence exists for surveillance for bowel cancer, but surveillance for the wider tumor spectrum is of uncertain benefit. We report a consanguineous Australian-Lebanese family with multiple affected individuals shown to be homozygous for a PMS2 exon 7 deletion. We also review published cases of biallelic mutations in HNPCC-related genes. Early recognition of this familial cancer syndrome is critical, and should prompt investigation for familial HNPCC mutations.

Publication types

  • Case Reports

MeSH terms

  • Adenosine Triphosphatases / genetics*
  • Adolescent
  • Brain Neoplasms / diagnosis
  • Brain Neoplasms / genetics*
  • Brain Neoplasms / therapy
  • Child
  • Colorectal Neoplasms, Hereditary Nonpolyposis / diagnosis
  • Colorectal Neoplasms, Hereditary Nonpolyposis / genetics*
  • Colorectal Neoplasms, Hereditary Nonpolyposis / therapy
  • Consanguinity
  • DNA Mismatch Repair
  • DNA Repair Enzymes / genetics*
  • DNA-Binding Proteins / genetics*
  • Exons
  • Fatal Outcome
  • Female
  • Follow-Up Studies
  • Glioblastoma / diagnosis
  • Glioblastoma / genetics*
  • Glioblastoma / therapy
  • Homozygote
  • Humans
  • Immunohistochemistry
  • Male
  • Mismatch Repair Endonuclease PMS2
  • Mutation, Missense*
  • Neoplasms, Multiple Primary / diagnosis
  • Neoplasms, Multiple Primary / genetics*
  • Neoplasms, Multiple Primary / therapy
  • Sequence Deletion
  • Syndrome

Substances

  • DNA-Binding Proteins
  • Adenosine Triphosphatases
  • PMS2 protein, human
  • Mismatch Repair Endonuclease PMS2
  • DNA Repair Enzymes