Gastrointestinal stromal tumors (GISTs) are the most common mesenchymal tumors of the gastrointestinal tract and historically were associated with a poor prognosis. Advances in the last 20 years in the diagnosis and treatment of GISTs have led to significant improvements in outcomes, making the treatment of GIST a model for the development of targeted therapy for solid tumors. Historically, GIST were classified as gastrointestinal leiomyoma, leiomyosarcoma, or leiomyoblastomas, but were reclassified in the 1990s after demonstration of the cell of origin as the interstitial cells of Cajal and the driver mutation in c-kit. Two years after the identification of c-kit as the activating mutation in GIST, the first patient with advanced GIST was treated on a clinical trial with imatinib, a tyrosine kinase receptor inhibitor of KIT. Today, imatinib is approved for use in advanced/metastatic GIST and as adjuvant therapy. Future advances require an understanding of the molecular biology of when imatinib is ineffective, which will open opportunities for new treatments.
KeywordsGastrointestinal stromal tumors Imatinib Tyrosine kinase inhibitor Sarcoma c-kit
Gastrointestinal stromal tumor
Tyrosine kinase inhibitor
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