Predictive factors of the treatment outcome in patients with advanced biliary tract cancer receiving gemcitabine plus cisplatin as first-line chemotherapy

BACKGROUND: Few studies have clearly identified the prognostic factors in patients with advanced biliary tract cancer (BTC) receiving gemcitabine plus cisplatin (GC) which is acknowledged as standard chemotherapy regimen. OBJECTIVES: The aim of this study was to identify predictive factors of the overall survival (OS) in advanced BTC patients receiving GC therapy. METHODS: Data of 307 patients with advanced BTC who received GC therapy as the first-line chemotherapy at our institution from January 2007 to June 2017 were reviewed retrospectively. The patients were randomly assigned to the investigation or the validation dataset at the ratio of 2:1. Multivariate analysis was conducted to identify the prognostic factors, a prognostic index is proposed from the investigation dataset, and the usefulness of this index was confirmed in the validation dataset. RESULTS: Multivariate analysis identified poor performance status, elevated serum lactate dehydrogenase, and elevated neutrophil-to-lymphocyte ratio as independent unfavorable predictors. The patients could be classified into three groups according to these factors, and it was found that the outcomes differed significantly among the three groups (P = 0.0002, good- vs. intermediate-prognosis groups; P = 0.005, intermediate- vs. poor-prognosis groups). When this index was applied to the validation dataset, the OS was confirmed to differ significantly among the three groups (P = 0.04, good- vs. intermediate-prognosis groups, P < 0.0001, intermediate- vs. poor-prognosis groups). CONCLUSIONS: We identified three predictors of the OS in patients with advanced BTC receiving GC therapy in this study, based on which we could classify the patients into three risk groups.