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Review
. 2025 Aug;39(4):773-784.
doi: 10.1016/j.hoc.2025.04.006. Epub 2025 May 13.

Gastrointestinal Stromal Tumor: Current Approaches and Future Directions in the Treatment of Advanced Disease

Carlo M Cicala  1 , Sebastian Bauer  2 , Michael C Heinrich  3 , César Serrano  4
Affiliations
Review

Gastrointestinal Stromal Tumor: Current Approaches and Future Directions in the Treatment of Advanced Disease

Carlo M Cicala et al. Hematol Oncol Clin North Am. 2025 Aug.

Abstract

This review discusses the current and future landscape of advanced gastrointestinal stromal tumor (GIST) treatment. It covers the role of tyrosine kinase inhibitors (TKIs), specifically imatinib, and further treatment options, such as sunitinib, regorafenib, and ripretinib, as well as avapritinib for platelet-derived growth factor receptor alpha D842V mutations. In addition, this review emphasizes individualized treatment strategies within multidisciplinary expert teams, including surgery and other locoregional therapies, together with the importance of mutation-guided approaches, particularly for wild-type GISTs. Finally, it explores the potential of next-generation KIT inhibitors, combination therapies, and other investigational approaches.

Keywords: Gastrointestinal stromal tumor (GIST); Imatinib; KIT mutation; PDGFRA mutation; Targeted therapy; Tyrosine-kinase inhibitors (TKIs).

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Conflict of interest statement

Disclosures C.M. Cicala has received honoraria from Deciphera Pharmaceuticals; travel grants from Deciphera Pharmaceuticals, United States, and PharmaMar, Spain. C.M. Cicala receives research funding from the BBVA Foundation, Spain (BBVA, Spain CAIMI II). S. Bauer has received institutional research funding from Incyte, United States, Blueprint Medicines, and von Pfeffel pharmaceuticals; has held a scientific advisory role (noncompensated) for IDRX, von Pfeffel, and Deciphera; received honoraria (CME) from Deciphera; and held an advisory role for Adcendo, Boehringer Ingelheim, and Cogent. M.C. Heinrich has received honoraria from Deciphera Pharmaceuticals, Novartis, Cogent Pharmaceuticals, and von Pfeffel Pharma. M.C. Heinrich is a member of the Scientific Advisory Board and has an equity interest in von Pfeffel Pharma. M.C.Heinrich received partial salary support and/or research support from research grants from a VA Merit Review grant, United States (I01BX005358), an NCI, United States R21 grant (R21CA263400), NIH, United States grant U01CA278470, GIST Cancer Research Fund, and GIST Support International. C. Serrano has received research funding (institution) from IDRX and Blueprint; consulting fees (advisory role) from NewBay, IDRX, Cogent, and Deciphera; payment for lectures from Roche, Switzerland, PharmaMar, and Deciphera; and travel grants from Gilead and PharmaMar.

References

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