About Gastrointestinal Stromal Tumor
Gastrointestinal stromal tumours are the most common mesenchymal tumours of the GI tract, arising from the interstitial cells of Cajal or their precursors, and defined by gain-of-function mutations in KIT (approximately 80%) or PDGFRA (approximately 10%), with the remaining cases being wild-type and often harbouring SDH complex deficiency, NF1 mutations, or BRAF alterations. The advent of imatinib and subsequent generations of tyrosine kinase inhibitors has transformed the management of advanced GIST, but drug resistance through secondary KIT/PDGFRA mutations remains the central clinical challenge. Precise molecular subtyping of the primary driver mutation is essential for predicting drug sensitivity and determining eligibility for targeted therapy trials.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Gastrointestinal Stromal Tumor trials.
Precise molecular characterisation — KIT exon 9, 11, 13, or 17 mutation, PDGFRA mutation including D842V status, or SDH/NF1/BRAF wild-type subtype — is required for virtually all targeted therapy trials and determines which TKI is applicable.
PDGFRA D842V mutations confer resistance to imatinib and sunitinib but sensitivity to avapritinib; confirm D842V status separately as standard KIT/PDGFRA panels do not always report this variant explicitly.
Prior TKI lines of therapy and reasons for discontinuation (progression vs. toxicity) are key eligibility variables; provide a complete treatment chronology with best response and progression dates for each agent.
Patient Resources
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