FRIDAY, Nov. 23 (HealthDay News) -- For patients with metastatic colorectal cancer or gastrointestinal stromal tumors (GIST) who have progressed in spite of treatment, the multikinase inhibitor regorafenib may improve survival, according to the results of two phase 3 studies published online Nov. 22 in The Lancet.

Axel Grothey, M.D., from the Mayo Clinic in Rochester, Minn., and colleagues conducted a phase 3 trial at 114 centers in 16 countries to assess regorafenib in patients with metastatic colorectal cancer and progression during or within three months after the last standard therapy. Seven hundred sixty patients were randomized to regorafenib or placebo in a 2:1 ratio. The researchers found that the median overall survival was 6.4 months for regorafenib-treated and 5.0 months for placebo-treated patients (hazard ratio, 0.77). Treatment-related adverse events occurred in 93 percent of regorafenib-treated patients and 61 percent of placebo-treated patients.

In a second study, George D. Demetri, M.D., from the Dana-Farber Cancer Institute in Boston, and colleagues conducted a phase 3 trial at 57 hospitals in 17 countries to evaluate the efficacy and safety of regorafenib in patients with metastatic or unresectable GIST progressing after failure of at least imatinib and sunitinib. One hundred ninety-nine patients were randomized in a 2:1 ratio to regorafenib or placebo. The researchers found that the median progression-free survival was 4.8 months for regorafenib and 0.9 months for placebo (hazard ratio, 0.27). Drug-related adverse events were reported by 98 percent of regorafenib-treated and 68 percent of placebo-treated patients.

"On the basis of the reported data from these two clinical trials, regorafenib would seem to have a future in the treatment of gastrointestinal malignant disease," write the authors of an accompanying editorial.

Several authors from both studies disclosed financial ties to the pharmaceutical industry, including Bayer HealthCare Pharmaceuticals, which funded both studies.

Abstract - Grothey
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Abstract - Demetri
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Editorial (subscription or payment may be required)