4.5 Review

Complete response of advanced rectal gastrointestinal stromal tumors after imatinib treatment: A case report and literature review

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Summary: The discovery of oncogenic mutations in the KIT and PDGFRA tyrosine kinase receptor was a crucial step in the development of tyrosine kinase inhibitors (TKIs). GIST became a model for molecular-targeted therapy, leading to improved overall survival, but further progress is needed for TKI resistant mutations. Combination therapies and immunotherapy have not fully delivered on their promise in GIST. The development of novel approved TKIs like ripretinib and avapritinib, as well as NTRK inhibitors for ultrarare NTRK fusions, highlight the ongoing need for adequate molecular profiling of high-risk GISTs before treatment.

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