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More than shots in the dark: improving patient stratification to move closer to personalised therapies in intrahepatic cholangiocarcinoma
  1. Maria Arechederra1,2,3,
  2. Andrea Casadei Gardini4,5,
  3. Chiara Raggi6
  1. 1Hepatology Laboratory, Solid Tumors Program, CIMA, CCUN, University of Navarra, Pamplona, Spain
  2. 2CIBERehd, Instituto de Salud Carlos III, Madrid, Spain
  3. 3Instituto de Investigaciones Sanitarias de Navarra IdiSNA, Pamplona, Spain
  4. 4Medical Oncology Department, IRCSS San Raffaele Scientific Institute, Milan, Italy
  5. 5Department of Oncology, Vita-Salute San Raffaele University, Milan, Italy
  6. 6Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy
  1. Correspondence to Dr Chiara Raggi, Department of Experimental and Clinical Medicine, University of Florence, Firenze, Italy; chiara.raggi{at}

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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. …

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  • Contributors Conception writing, review and final approval of manuscript: MA, ACG and CR.

  • Funding Author’s research is partially supported by Instituto de Salud Carlos III (SCIII) cofinanced by ‘Fondo Europeo de Desarrollo Regional’ (FEDER) ‘Una Manera de Hacer Europa’ PI22/00471 and AECC investigador fellowship INVES223049AREC to MA. Partially is provided by grants from Associazione Italiana per la Ricerca sul Cancro (AIRC) (IG23117) to CR. CR is member of the European Network for the Study of Cholangiocarcinoma (ENSCCA) and participates in the initiative COST Action EURO-CHOLANGIO-NET granted by the COST Association (CA18122).

  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.

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