Abstract Background Biliary tract cancer is a rare tumour entity mostly diagnosed at advanced stages with poor prognosis. Since publication of the TOPAZ-1-trial, durvalumab + gemcitabine/cisplatin has become the standard palliative first-line treatment. However, real-world evidence is inconclusive and no systematic review or meta-analysis has yet evaluated the current available literature on this topic. This meta-analysis therefore aimed to assess the effectiveness of durvalumab plus gemcitabine/cisplatin as first-line treatment compared to the previous standard and evaluate the existing evidence of this treatment in real-world cohorts. Methods Trials investigating durvalumab + gemcitabine/cisplatin as palliative first-line treatment in advanced biliary tract cancer and published in PubMed/Medline databases between January 2020 and December 2024 were included. Studies on second line treatment or studies investigating other than the standard chemotherapy backbones were excluded. Selection of the trials and quality assessment was conducted independently by two reviewers. Trials with a two-arm design reporting effect measures were included in the meta-analysis. Results After screening 190 studies, 10 trials encompassing 2877 patients were included. Evidence was heterogeneous but results of the meta-analysis demonstrated a statistically significant difference in overall survival and progression free survival, in favor of patients treated with durvalumab + gemcitabine/cisplatin. Conclusion This systematic review and meta-analysis confirms durvalumab + gemcitabine/cisplatin as best currently available treatment option in patients with advanced biliary tract cancer. Furthermore, multiple real-world cohorts reported similar results even in patients with higher risk factors. However, trials are heterogeneous and further evidence from real-world cohorts is needed to enhance data quality.
Reimann et al. (Wed,) studied this question.