Gastrointestinal stromal tumor: a review of current and emerging therapies

• Published in: Cancer and metastasis reviews Vol. 40; no. 2; pp. 625 - 641
• Main Authors: Al-Share, Bayan, Alloghbi, Abdulrahman, Al Hallak, Mohammed Najeeb, Uddin, Hafiz, Azmi, Asfar, Mohammad, Ramzi M., Kim, Steve H, Shields, Anthony F., Philip, Philip A
• Format: Journal Article
• Language: English
• Published: New York Springer US 01.06.2021; Springer; Springer Nature B.V
• Subjects: Biomedical and Life Sciences; Biomedicine; c-Kit protein; Cancer Research; Clinical; Drug approval; Drug therapy; Enzyme inhibitors; Gastrointestinal cancer; Gastrointestinal system; Gastrointestinal tract; Imatinib; Inhibitor drugs; Mutation; Neoplasia; Nervous system; Oncology; Patients; Platelet-derived growth factor; Protein-tyrosine kinase; Surgery; Targeted cancer therapy; Tumors; Tyrosine; mutation; GIST; Imatinib; Sunitinib; PDGFR; Ripretinib; Regorafinib; c-Kit mutation
• ISSN: 0167-7659; 1573-7233; 1573-7233
• DOI: 10.1007/s10555-021-09961-7

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.