Sarcoma (Gastrointestinal Stromal Tumors, GIST)

    Actionable Targets Abnormality Prevalence Clinical Experience with Targeted Agent
    BRAF Mutation 4% Dabrafenib (BRAF inhibitor):
    • Case report: SD for 8+ mo (first report of BRAF+ GIST) (1)
    ETV6-NTRK3 Fusion 1.7% Larotrectinib (NTRK inhibitor):
    • Case report: PR for 4+ months in WT GIST (2)
    KIT Mutation 85% (3,4) Imatinib (KIT/BCL/ABL/PDGFR inhibitor):•
    • Phase 2: PR 83% for KIT exon 11 mutations vs. 47.8% for KIT exon 9 mutations, no KIT or PDGFR mutations (4)
    Sunitinib (KIT/PDGFR/VEGFR/RET/FLT3 inhibitor):
    • Phase 1/2: PR 37% for KIT exon 9 mutations vs. 5% for tions, 56% for WT KIT and PDGFR-A (5)
    Other topics in Targets by Organ Site