GIST(Gastrointenstinal Stromal Tumor) is the most common form of sarcoma in the gastrointestinal tract. There are thought to be 4,000-5,000 new cases of GIST diagnosed each year in the USA, about 1,500 of them metastatic. Two treatments for GIST has been recently FDA approved. Recently, FDA also approved regorefanib, Stivarga, for colon cancer, based on the CORRECT trial. The application for approval is for GIST is now pending before the FDA. It is based on the recent GRIP study of 132 third line patientswho either received regorafenib 160 mg once daily or placebo. There was no overall survival difference, possibly related to the crossover design of the study (85% crossed over to regorefanib). Progression-free survival, however, another indicator of effectiveness, was almost five times greater in the regorafenib group at 4.8 months compared with 0.9 months in the placebo group. The disease control rate was also significantly greater – 52.6% compared with 9.1% for placebo-treated patients.
Efficacy is also supported by a phase II trial published in 2012 by S. George et al. Bayer has began to offer regorefenib to patients with GIST on a compassionate use basis.
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