Affiliation
Department of Medical Oncology, Hospital Universitario, 12 de Octubre, Madrid, SpainIssue Date
2019
Metadata
Show full item recordAbstract
Most of patients with cholangiocarcinoma (CCA) present with advanced (inoperable or metastatic) disease, and relapse rates are high in those undergoing potentially-curative resection. Previous treatment nihilism of patients with advanced disease has been replaced by active clinical research with the advent of randomized clinical trials (RCTs) and a much greater effort at understanding molecular mechanisms underpinning CCA. Three RCTs have recently been reported evaluating adjuvant chemotherapy following curative resection; only one of these has the potential to change practice. The BILCAP study failed to meet its primary endpoint by intention-to-treat (ITT) analysis; however, a survival benefit was seen in a pre-planned sensitivity analysis (predominantly adjusting for lymph nodes status). This, along with the numerical difference in median overall survival has led to uptake of adjuvant capecitabine by many clinicians. In patients with advanced disease, the only level 1 data available supports the use of cisplatin and gemcitabine for the first-line treatment of patients with advanced disease; there is no established second-line chemotherapy. Previous forays into targeted therapy have proven unfruitful (namely targeting the epithelial growth factor receptor [EGFR] and vascular endothelial growth factor [VEGF] pathways). An increasing number of genomic subtypes are being defined; for some of these on-target therapeutic options are under active investigation. The most developed are studies targeting IDH-1 (isocitrate dehydrogenase) mutations and FGFR-2 (fibroblast growth factor receptor) fusions, with promising early results. Several other pathways are under evaluation, along with early studies targeting the immune environment; these are too premature to change practice to date. These emerging treatments are discussed.Citation
Adeva J, Sangro B, Salati M, Edeline J, La Casta A, Bittoni A, et al. Medical treatment for cholangiocarcinoma. Liver Int. 2019.Journal
Liver InternationalDOI
10.1111/liv.14100PubMed ID
30892822Additional Links
https://dx.doi.org/10.1111/liv.14100Type
ArticleLanguage
enae974a485f413a2113503eed53cd6c53
10.1111/liv.14100