Regorafenib Active in Refractory Gastrointestinal Stromal Tumors

For patients with metastatic gastrointestinal stromal tumors (GISTs), the use of tyrosine kinase inhibitors (TKIs) that target KIT and PDGFR improves disease control and survival. Adjuvant therapy with these TKIs after resection of high-risk primary disease also improves survival. However, despite the success of the TKIs imatinib as first-line therapy and sunitinib as second-line therapy, virtually all patients with metastatic GISTs ultimately develop resistance to these agents.

Investigators now report the results of an industry-supported, global, double-blinded, placebo-controlled, phase III trial of the novel, multitargeted TKI regorafenib in patients with GISTs that were refractory to both imatinib and sunitinib. A total of 199 patients …