Lenvatinib plus toripalimab with local-regional therapy in patients with advanced biliary tract cancer: a retrospective study

Abstract Background: Lenvatinib plus anti-PD-1 antibodies with local-regional therapy which was called stereotactic therapy have demonstrated potent antitumor activity in solid tumors. However, the efficacy and safety of stereotactic therapy in patients with advanced biliary tract cancer (BTC) remains unclear.Methods: This was a retrospective study including patients with advanced BTC who received a combination of Lenvatinib plus toripalimab or Lenvatinib plus toripalimab with local-regional therapy (stereotactic therapy). This study evaluated the efficacy and safety of Lenvatinib plus toripalimab and stereotactic therapy in advanced BTC patients. Tumor tissues were collected to retrospectively evaluate the expression status of PDL1.Results: This study included 55 patients: 24 in Lenvatinib plus toripalimab therapy; 31 in stereotactic therapy. All patients had undergone at least 1 line of antitumor treatment. The stereotactic therapy group showed longer median progression-free survival (mPFS) (9.6 versus 4.6 months, p=0.035), longer overall survival (mOS) (13.7 versus 9.2 months, p=0.023) than the Lenvatinib plus toripalimab group. The ORR was 35.5% [95% confidence interval (CI): 17.6-53.3] with stereotactic therapy versus 25% (95% CI: 6.3-43.7) with toripalimab plus Lenvatinib. Four patients received surgery after the Lenvatinib plus toripalimab therapy or after stereotactic therapy. All patients experienced any-grade adverse events (AEs) without grade 5 AEs. PDL1 expression was associated with improved clinical benefits.Conclusions: Stereotactic therapy had acceptable toxic effects and might improve survival compared with Lenvatinib plus toripalimab for advanced BTC.