Tepotinib in Cholangiocarcinoma with MET Amplification: A Case Report

Cholangiocarcinoma is a malignant tumor that affects the bile ducts and is usually aggressive with poor prognosis. The treatment of cholangiocarcinoma depends on the stage and location of the tumor as well as the patient's overall health status. Systemic therapy, such as chemotherapy using gemcitabine and cisplatin, is the first choice for patients with cholangiocarcinoma who were inoperable. After no response to first-line chemotherapy, second-line chemotherapy or targeted therapy focusing on signaling pathway inhibition are subsequent treatment. The present report described a case of cholangiocarcinoma involving bilateral lobes of liver. He received one cycle of chemotherapy with gemcitabine plus cisplatin and exhibited rapid progression. Next-generation sequencing was performed, and the results showed that MET amplification had a gene copy number of 68. After that, he underwent tepotinib and tumor shrinkage occurred. After a follow‑up period of 12 months, the treatment response was partial response, and the benefit of tepotinib is ongoing. The development of precision medicine has expanded the paradigm of targeted therapies to increasingly favorable options in the second line and beyond, and prolong overall survival. Detecting druggable mutations (mutations potentially amenable to treatment with) for identifying a landscape of therapeutic options is imperative for managing cholangiocarcinoma.