To investigate the clinical efficacy and safety of Pingxiao capsule combined with gemcitabine and cisplatin in the treatment of advanced cholangiocarcinoma.

Seventy-eight patients with advanced cholangiocarcinoma who received treatment in Haiyang People's Hospital from January 2017 to June 2019 were included in this study. They were randomly assigned to receive treatment with either Pingxiao capsule combined with gemcitabine and cisplatin (treatment group, n = 39) or gemcitabine combined with cisplatin (control group, n = 39). Short-term and long-term curative effects as well as drug safety were compared between the treatment and control groups.

Effective rate in the treatment group was significantly higher than that in the control group [28.2% (11/39) vs. 18.0% (7/31)]. Short-term curative effect in the treatment group was slightly, but not significantly, higher than that in the control group (χ² = 3.961, P = 0.266). Median overall survival time in the treatment and control groups was 12.5 months (95% CI: 8.4-16.6 months) and 9.0 months (95% CI: 5.0-13.0 months), respectively (HR = 2.506, 95%CI: 1.437-4.369). Median progression-free survival in the treatment and control groups was 8.6 months (95% CI: 6.6-10.6 months) and 4.8 months (95% CI: 4.0-5.6 months), respectively (HR = 2.555, 95%CI: 1.459-4.472). There was no significant difference in the incidence of adverse reactions between the two groups (χ² = 6.986, P = 0.072).

Pingxiao capsule combined with gemcitabine and cisplatin in the treatment of advanced cholangiocarcinoma can increase long-term effective rate and prolong survival time, but it does not obviously increase adverse reactions compared with gemcitabine combined with cisplatin. Therefore, it can be used as a treatment option for patients with advanced cholangiocarcinoma.

Bile duct neoplasms; Antineoplastic combined chemotherapy protocols; Progression-free survival; Pingxiao capsule; Gemcitabine; Cisplatin