Efficacy and safety of durvalumab and gemcitabine-based chemotherapy combined with or without lenvatinib in advanced biliary tract cancer: A retrospective real-world study

Abstract Background Immunotherapy in combination with gemcitabine-based chemotherapy has become the standard first-line treatment for advanced biliary tract cancer (BTC). Some small sample studies have shown that immune checkpoint inhibitors (ICIs), when used in combination with lenvatinib and chemotherapy as a first-line therapy, exhibit high BTC antitumour activity. However, real-world data showing the efficacy and safety of durvalumab combined with chemotherapy and lenvatinib are lacking. Therefore, we conducted a retrospective study to assess the efficacy and safety of durvalumab and gemcitabine-based chemotherapy with or without lenvatinib for advanced BTC therapy. Methods We conducted a retrospective analysis of the efficacy and safety of durvalumab and gemcitabine-based chemotherapy with or without lenvatinib in advanced BTC cancer patients between January 2021 and June 2023. The study endpoints were overall survival (OS), progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), and safety. Results Thirty patients with advanced BTC were included in this study, among which 14 (46.7%) received durvalumab combined with lenvatinib plus chemotherapy. For all the patients, the median OS was 11.82 months, the median PFS was 6.23 months, the ORR was 43.4%, and the DCR was 76.7%. Patients who received lenvatinib treatment had a longer median PFS (7.42 vs. 4.98 months, P = 0.875) than did those in the non-lenvatinib group. However, the median OS was shorter in patients in the lenvatinib treatment group (10.60 vs. 14.35 months, P = 0.759). The ORR and DCR in patients receiving lenvatinib were 57.1% and 92.9%, respectively. However, in patients that did not receive lenvatinib, the ORR and DCR were only 31.2% and 62.5%, respectively. All patients experienced adverse events (AEs), but the inclusion of lenvatinib did not increase the risk of AEs. The data are still being updated. Conclusion Durvalumab and gemcitabine-based chemotherapy with or without lenvatinib has been shown to be effective and safe in routine practice. The addition of lenvatinib may improve only the DCR, ORR, and PFS but may not prolong OS.