Background: Although gemcitabine and platinum-based agents (GP) are currently regarded as the standard chemotherapy for advanced biliary tract cancer (BTC), the prognosis remains poor. Combinations with fluoropyrimidines and targeted therapy have demonstrated modest benefits. Therefore, we conducted a meta-analysis of randomized controlled trials to evaluate the efficacy of different chemotherapy regimens.
Methods: The PubMed, EMBASE, Cochrane Library, Scopus, and ClinicalTrials.gov registries were searched for studies published until April 2016. A meta-analysis was conducted to calculate the pooled effect size by using random effects models. Treatment efficacies were measured using progression-free survival (PFS) and overall survival. The secondary outcomes included the objective response rate (ORR), 1-year survival rate, quality of life, disease control rate, and adverse events.
Results: Fifteen trials that involved examining 1775 patients were reviewed. Patients who received epidermal growth factor receptor (EGFR)-targeted therapy in addition to standard GP chemotherapy exhibited a significantly higher median PFS (weighted mean difference = -1.49; 95% confidence interval -2.56 to -0.43), PFS (hazard ratio = 0.79; 95% confidence interval 0.63-0.99), and ORR (odd ratio = 0.56; 95% confidence interval 0.38-0.82). Combining GP with fluoropyrimidines or vascular EGFR inhibitors (VEGFR) did not improve patient outcomes.
Conclusion: Combining EGFR-targeted therapy with the current standard GP chemotherapy is a safe and viable option that may improve the median PFS, PFS, and ORR in patients with advanced BTC. Further research investigating the optimal dosage and drug type of EGFR inhibitors for specific BTC patient groups is warranted.