Extraordinary Benefit of Regorafenib in Metastatic Cholangiocarcinoma: A Case Report.

INTRODUCTION/

BACKGROUND

Cholangiocarcinoma (CC) is a rare, aggressive cancer of the bile ducts with limited treatment advancements over recent decades. The five-year survival rate for CC remains low, around 10%, and even lower for advanced cases.

CASE PRESENTATION: This case report discusses a 46-year-old woman with metastatic CC who achieved remarkable progression-free survival with regorafenib, a multikinase inhibitor typically used in other cancers. After multiple lines of treatment, regorafenib was given as a 7thline therapy. Despite initial intolerance requiring dose reduction, the patient achieved 22 months of progression-free survival (PFS) with stable disease and partial regression in some areas. Her response to regorafenib significantly exceeded typical outcomes in the literature, where PFS generally ranges from 3 to 4 months. This improvement may be attributed to an FGFR2 mutation identified via next-generation sequencing, which regorafenib may effectively target.

CONCLUSION

This case suggests that patients with FGFR2 mutations might benefit from regorafenib, warranting further studies to explore this associationAs can be applied as a promising target gene to develop drug resistance and remedial efficacy.