Gastrointestinal stromal tumor: a review of current and emerging therapies

Gastrointestinal stromal tumors (GIST) are rare neoplasms arising from the interstitial cell of Cajal in the gastrointestinal tract. Two thirds of GIST in adult patients have c-Kit mutation and smaller fractions have platelet derived growth factor receptor alpha (PDGFRA) mutation. Surgery is the only curative treatment for localized disease. Imatinib improves survival when used adjuvantly and in advanced disease. Several targeted therapies have also improved survival in GIST patients after progression on imatinib including sunitinib and regorafenib. Recently, United States Federal and Drug Administration (FDA) approved two new tyrosine kinase inhibitors for the treatment of heavily pretreated advanced/unresectable GIST including avapritinib (a selective inhibitor for PDGFRA exon 18 mutation including D842V mutations) and ripretinib (a broad-spectrum kinase inhibitor of c-Kit and PDGFRA). In this article, we will provide a comprehensive review of GIST including the current standard of care treatment and exploring future paradigm shifts in therapy.

Automated summary

GIST is a rare tumor originating from the interstitial cells of Cajal in the gastrointestinal tract, with surgery being the only curative treatment for localized disease. Targeted therapies like imatinib have shown improved survival rates in patients. FDA-approved new tyrosine kinase inhibitors, avapritinib and ripretinib, provide options for heavily pretreated advanced GIST. Future shifts in therapy paradigms are likely to occur in the treatment of GIST.