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Prior to the introduction of imatinib, rare mesenchymal tumors known as gastrointestinal stromal tumors (GISTs) were highly resistant to therapy and were associated with poor prognosis. GISTs harbor mutations in the KIT or PDGFRA genes that encode tyrosine kinase receptors, which are targeted effectively by the tyrosine kinase inhibitor imatinib. Indeed, imatinib markedly improves progression-free and overall survival in patients with advanced GISTs and improves progression free-survival in the adjuvant setting after surgical resection of GISTs.
In an earlier industry-funded, open-label, multicenter, randomized, phase III trial (BFR14), researchers in France compared outcomes of 58 patients with nonprogressive GISTs who were randomized to co…