Gastrointestinal stromal tumors (GIST): C-kit mutations, CD117 expression, differential diagnosis and targeted cancer therapy with Imatinib

Pathol Oncol Res. 2003;9(1):13-9. doi: 10.1007/BF03033708. Epub 2003 Apr 18.

Abstract

Gastrointestinal stromal tumors (GISTs) have been recognised as a biologically distinctive tumor type, different from smooth muscle and neural tumors of the gastrointestinal tract. They constitute the majority of gastrointestinal mesenchymal tumors. They are defined and diagnosed by the expression of a protooncogene protein called CD117 detected by immunohistochemistry. It is now believed that GISTs originate from gastrointestinal pacemaker cells known as interstitial cells of Cajal, that control gut motility or from a precursor of these cells. The identification of mutations mostly in exon 11 and to a lesser extent in exons 9 and 13 of the c-kit protooncogene coding for c-kit (CD117) in many GISTs, has resulted in a better understanding of their oncogenic mechanisms. The finding of remarkable antitumor effects of the molecular inhibitor, imatinib (Glivec trade mark ) in metastatic and inoperable GISTs, has necessitated accurate diagnosis of GISTs and their distinction from other gastrointestinal mesenchymal tumors. To achieve this, pathologists need to be familiar with the spectrum of histological appearances shown by GISTs and have a high index of suspicion for these tumors. This review summarises recent advances in knowledge regarding the histogenesis, pathology, molecular biology, genetics and differential diagnosis of GISTs and the basis for the novel targeted cancer therapy with imatinib.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Benzamides
  • Diagnosis, Differential
  • Enzyme Inhibitors
  • Gastrointestinal Neoplasms* / diagnosis
  • Gastrointestinal Neoplasms* / drug therapy
  • Gastrointestinal Neoplasms* / genetics
  • Gastrointestinal Neoplasms* / metabolism
  • Humans
  • Imatinib Mesylate
  • Incidence
  • Mutation
  • Piperazines / therapeutic use*
  • Prognosis
  • Protein-Tyrosine Kinases
  • Proto-Oncogene Proteins c-kit / genetics*
  • Proto-Oncogene Proteins c-kit / metabolism*
  • Pyrimidines / therapeutic use*
  • Stromal Cells / pathology

Substances

  • Antineoplastic Agents
  • Benzamides
  • Enzyme Inhibitors
  • Piperazines
  • Pyrimidines
  • Imatinib Mesylate
  • Protein-Tyrosine Kinases
  • Proto-Oncogene Proteins c-kit