• CHOLANGIOCARCINOMA
• CHEMOTHERAPY

Cholangiocarcinomas, the second most frequent hepatic tumour after hepatocellular carcinoma, make up around 2% of all gastrointestinal malignancies. It is believed that these tumours have been occurring more frequently recently, at least in Western countries, and that this is mostly due to an increase in intrahepatic cholangiocarcinoma (iCCA).1 The combination of cisplatin and gemcitabine was established as the first-line therapy for patients with advanced cholangiocarcinoma for several years based on the findings of the ABC-02 trial. Recently, the phase III TOPAZ-1 and KEYNOTE-966 studies found a survival benefit with durvalumab or pembrolizumab in combination with gemcitabine and cisplatin, respectively.2 In a real-world scenario, phase III TOPAZ-1 trial data were validated.3 With very few clinical data, the relevance of second-line chemotherapy following progression is still up for debate. Fluorouracil and oxaliplatin regimen was recently reported by the ABC-06 study to be related with better overall survival in patients with advanced cholangiocarcinoma. With a 5-year survival rate of about 2%, traditional treatment used in advanced stages still produces disappointing results.

In this still devastating context, the use of next-generation sequencing (NGS) technologies has recently surpassed the use of conventional diagnostic methods, enabling extensive genomic profiling investigations of intrahepatic CCA and the identification of potentially targetable mutations. …