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Regorafenib
Source: Gastrointestinal Stromal Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: December 13, 2024 · Captured 2026-09-09.
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The FDA has approved regorafenib for the treatment of GISTs that are refractory to first-line therapy. Regorafenib is a multikinase inhibitor with activity against KIT, PDGFRA, and VEGFR, among others. Regorafenib has demonstrated anti-GIST activity in phase II and phase III studies.[39,40]
Evidence (regorafenib):
The phase III double-blind GRID trial (NCT01271712) included 199 patients with advanced GISTs who did not respond to previous imatinib and sunitinib therapy. Patients were randomly assigned in a 2:1 ratio to receive either 160 mg daily of regorafenib (3-weeks-on/1-week-off regimen) (n = 133) or placebo (n = 66). Crossover to open-label regorafenib was allowed for patients who had disease progression on the placebo arm.[40]
The phase III double-blind GRID trial (NCT01271712) included 199 patients with advanced GISTs who did not respond to previous imatinib and sunitinib therapy. Patients were randomly assigned in a 2:1 ratio to receive either 160 mg daily of regorafenib (3-weeks-on/1-week-off regimen) (n = 133) or placebo (n = 66). Crossover to open-label regorafenib was allowed for patients who had disease progression on the placebo arm.[40]
After a median treatment duration of 23 weeks for regorafenib and 7 weeks for placebo, the median PFS was longer in patients who received regorafenib (4.8 months) compared with patients who received placebo (0.9 months) (HR, 0.27; 95% CI, 0.19–0.39; P < .0001).[40][Level of evidence B1] The OS did not differ between arms (HR, 0.77; 0.4–1.41; P = .199). However, 56 patients in the placebo arm (85%) did cross over to receive regorafenib at the time of disease progression.
Source links and citations
The phase III double-blind GRID trial (NCT01271712) included 199 patients with advanced GISTs who did not respond to previous imatinib and sunitinib therapy. Patients were randomly assigned in a 2:1 ratio to receive either 160 mg daily of regorafenib (3-weeks-on/1-week-off regimen) (n = 133) or placebo (n = 66). Crossover to open-label regorafenib was allowed for patients who had disease progression on the placebo arm.[40]
Adverse events were more common in the regorafenib arm (98.5%) compared with the placebo arm (68.2%). The most common grade 3 or greater events were hypertension (23.5%), hand-foot syndrome (19.7%), and diarrhea (5.3%).
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