Abstract
Background: Controversy exists regarding whether EGFR-targeted therapy combined with GEMOX (gemcitabine and oxaliplatin) provides additional benefits over GEMOX alone for biliary tract cancer patients. Therefore, this meta-analysis of randomized controlled trials (RCTs) was performed to assess the efficacy and safety of the GEMOX + EGFR-targeted regimen, and subgroup analysis was conducted to identify groups that might benefit from targeted therapy. Methods: The PubMed, Cochrane Library, and ClinicalTrials.gov registries were searched for published studies. Hazard ratios (HRs) for progression-free survival (PFS) and overall survival (OS) were pooled using a fix-effect model. Risk-ratios (RRs) were used to analyse the objective response rate (ORR) and adverse events. Results: Four RCTs were assessed. GEMOX + EGFR-targeted therapy significantly improved PFS (HR = 0.80, 95% CI 0.66-0.94, P = 0.03) and was associated with a better ORR (RR = 1.52, 95% CI 1.13-2.04, P = <0.01), whereas the TKI group achieved a better ORR in subgroup analysis. Patients with cholangiocarcinoma responded well to the GEMOX + EGFR-targeted regimen, leading to a better ORR (RR = 1.78, 95% CI 1.21-2.61, P = <0.01). Unfortunately, PFS benefits were not translated into OS benefits (HR = 0.92, 95% CI 0.75-1.08, P = 0.39). Conclusion: GEMOX + EGFR-targeted therapy is a considerable and tolerable treatment option for patients with advanced BTCs, improving both PFS and ORR but not prolonging patient survival. Patients with cholangiocarcinoma would benefit the most from EGFR-targeted therapy.