Abstract

Background Advanced cholangiocarcinoma (CCA) patients have a poor survival rate, even when receiving standard chemotherapy. No targeted therapy has been proven to benefit patients with CCA. The molecular alterations in Western and Eastern population are different. The aim of this study was to describe the natural history and clinical behaviour of the disease, including determining prognostic factors and molecular alterations of CCA in the Thai population. Methods A computerised search of a tumour registry database and tissue archive database in Ramathibodi Hospital from November 2007, to December 2011, identified 157 CCA patients who had adequate tumour tissue for DNA/RNA extraction. Data on the natural history and clinical behaviour were collected. The association and predictive ability of patient and tumour characteristics with overall survival (OS) and progression-free survival (PFS) outcomes were examined. ALK expression and ROS1 rearrangement were analysed. Findings Four significant prognostic factors for survival outcomes identified from multivariable-analysis included staging, performance status, surgical resection, and CA19-9 pre-treatment level. Median follow-up was 6.77 months. The median OS and PFS were 6.96 and 4.5 months, respectively. Advance stage disease, poor performance status, high CA19-9 level (⩾100 U/ml), and inoperability correlated with poor survival outcomes. CA19-9 ⩾100 U/ml was the optimal cut point, predicting both OS and PFS. There was a trend for better OS in patients without metastatic lymph nodes. One out of 50 patients had ALK expression, and this patient had OS of 30 months. No ROS1 gene rearrangement was found in 50 patients. Interpretation These data provided strong evidence for staging, performance status, surgical resection, and CA19-9 pre-treatment level as prognostic factors for CCA in the Thai population. Larger studies to elucidate the oncogenic drive genes of CCA are urgently needed.

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