What three diseases are the main indications for regorafenib?

Regorafenib is a multi-target oral tyrosine kinase inhibitor with multiple mechanisms of anti-tumor angiogenesis, anti-tumor cell proliferation and inhibition of the tumor microenvironment. It can inhibit multiple kinase pathways involved in angiogenesis (such as VEGFR1-3), tumor growth (such as KIT, RET) and metastasis (such as PDGFR, FGFR), and therefore has become one of the effective targeted therapeutic drugs for the clinical treatment of advanced malignant tumors. Currently, the three main indications for regorafenib that are widely recognized worldwide are: metastatic colorectal cancer (mCRC), gastrointestinal stromal tumor (GIST), and hepatocellular carcinoma (HCC).

For metastatic colorectal cancer, regorafenib is mainly used for patients who have failed standard treatment regimens, that is, patients who have previously received fluorouracil, oxaliplatin, irinotecan, anti-VEGF therapy, and RAS wild-type patients who still have disease progression after receiving anti-EGFR therapy. Such patients have low response to traditional chemotherapy, but regorafenib can control tumor progression and prolong survival time through a multi-target mechanism.

In gastrointestinal stromal tumors (GIST), regorafenib is suitable for patients who have failed or cannot tolerate imatinib and sunitinib. Regorafenib has an inhibitory effect on KIT and PDGFRA mutations and can control the disease process of GIST that progresses slowly but has a high recurrence rate.

For advanced hepatocellular carcinoma (HCC), regorafenib is widely recommended in multiple guidelines around the world as a second-line targeted drug after failure of sorafenib treatment. It has a good inhibitory effect on the angiogenesis characteristics of liver cancer, helps prolong the progression-free survival of patients, and its tolerance is relatively controllable.

In summary, the three core indications for regorafenib are metastatic colorectal cancer, gastrointestinal stromal tumors, and hepatocellular carcinoma. For these tumors, its use needs to be decided based on a comprehensive assessment of previous treatment experience, molecular biology characteristics and the patient's overall status, reflecting the development direction of precision medicine and targeted therapy.

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