State-of-the art therapy for gastrointestinal stromal tumors.

Until recently, there were few effective therapeutic options for patients with gastrointestinal stromal tumors (GISTs). Most patients undergoing even potentially curative resection for early-stage disease recurred if followed for a sufficiently long period, and treatment of advanced tumors with systemic chemotherapy was ineffective. Imatinib mesylate, a molecularly targeted agent that inhibits the KIT receptor tyrosine kinase, has now been demonstrated to be highly effective at inducing objective responses in GIST patients, and it improves overall survival. In locoregional disease, ongoing studies are assessing the use of imatinib pre-or postsurgery. In addition, other agents possessing activity against a variety of molecular targets are being tested in advanced disease. Questions remain about the optimal dose of imatinib, whether to continue drug in the setting of progressive disease, and how best to prevent or overcome resistance.