Prognosis of Advanced Cholangiocarcinoma in the Palliative Care Setting: A Series of 201 Cases

Prognosis of cholangiocarcinoma is poor, and palliative treatment options are limited in China. This study aimed to analyze prognostic factors affecting survival in patients with advanced cholangiocarcinoma.

Clinical data on 201 consecutive patients with cholangiocarcinoma who received treatment at a single center from May 2014 to December 2018 were analyzed retrospectively. Survival curves were plotted using the Kaplan-Meier method. Survival analyses were performed using a log-rank test.

For first-line therapy, the disease control rate was 56% (85/152) and the overall response rate was 16% (24/152). The total disease control rate was 34% (23/67) for second-line therapy. The median progression-free survival was 7 months, and the median overall survival was 17 months. Next-generation sequencing was performed for 59 patients. The most frequently mutated genes were TP53 and PI3KCA. No significant association was found between gene mutations and treatment response or survival. Of 5 patients with high levels of microsatellite instability, 4 (80%) were sensitive to anti-programmed death 1 antibodies and remained in partial remission at last follow-up.

Macroscopic tumor characteristics, rather than gene mutations, determine the prognosis of advanced cholangiocarcinoma. High microsatellite instability may be a favorable predictor of response to immunotherapy for cholangiocarcinoma.