biostudies-literatureSun DNational Natural Science Foundation of China1527-1535https://www.ebi.ac.uk/biostudies/studies/S-EPMC6768892biostudies-literatureUnknown68(9)<h4>Background</h4>Evidence for the efficacy of immunotherapy in biliary tract cancer (BTC) is limited and unsatisfactory.<h4>Methods</h4>Chinese BTC patients receiving a PD-1 inhibitor with chemotherapy, PD-1 inhibitor monotherapy or chemotherapy alone were retrospectively analyzed. The primary outcome was overall survival (OS). The key secondary outcomes were progression-free survival (PFS) and safety. Patients previously treated with any agent targeting T cell costimulation or immune checkpoints were excluded.<h4>Results</h4>The study included 77 patients (a PD-1 inhibitor plus chemotherapy, n = 38; PD-1 inhibitor monotherapy, n = 20; chemotherapy alone, n = 19). The median OS was 14.9 months with a PD-1 inhibitor plus chemotherapy, significantly longer than the 4.1 months with PD-1 inhibitor monotherapy (HR 0.37, 95% CI 0.17-0.80, P = 0.001) and the 6.0 months with chemotherapy alone (HR 0.63, 95% CI 0.42-0.94, P = 0.011). The median PFS was 5.1 months with a PD-1 inhibitor plus chemotherapy, significantly longer than the 2.2 months with PD-1 inhibitor monotherapy (HR 0.59, 95% CI 0.31-1.10, P = 0.014) and the 2.4 months with chemotherapy alone (HR 0.61, 95% CI 0.45-0.83, P = 0.003). Grade 3 or 4 treatment-related adverse events were similar between the anti-PD-1 combination group and the chemotherapy alone group (34.2% and 36.8%, respectively).<h4>Conclusions</h4>Anti-PD-1 therapy plus chemotherapy is an effective and tolerable approach for advanced BTC.Cancer immunology, immunotherapy : CIIAnti-PD-1 therapy combined with chemotherapy in patients with advanced biliary tract cancer.PMC67688928167299681402552Cui PZheng XCai SHu YZhang JChen GDu WWang JSong JGao CZhao XLi XWu ZQian YSun DMa JHan CChen SYue ZfalseAnti-PD-1 therapy combined with chemotherapy in patients with advanced biliary tract cancer.<h4>Background</h4>Evidence for the efficacy of immunotherapy in biliary tract cancer (BTC) is limited and unsatisfactory.<h4>Methods</h4>Chinese BTC patients receiving a PD-1 inhibitor with chemotherapy, PD-1 inhibitor monotherapy or chemotherapy alone were retrospectively analyzed. The primary outcome was overall survival (OS). The key secondary outcomes were progression-free survival (PFS) and safety. Patients previously treated with any agent targeting T cell costimulation or immune checkpoints were excluded.<h4>Results</h4>The study included 77 patients (a PD-1 inhibitor plus chemotherapy, n = 38; PD-1 inhibitor monotherapy, n = 20; chemotherapy alone, n = 19). The median OS was 14.9 months with a PD-1 inhibitor plus chemotherapy, significantly longer than the 4.1 months with PD-1 inhibitor monotherapy (HR 0.37, 95% CI 0.17-0.80, P = 0.001) and the 6.0 months with chemotherapy alone (HR 0.63, 95% CI 0.42-0.94, P = 0.011). The median PFS was 5.1 months with a PD-1 inhibitor plus chemotherapy, significantly longer than the 2.2 months with PD-1 inhibitor monotherapy (HR 0.59, 95% CI 0.31-1.10, P = 0.014) and the 2.4 months with chemotherapy alone (HR 0.61, 95% CI 0.45-0.83, P = 0.003). Grade 3 or 4 treatment-related adverse events were similar between the anti-PD-1 combination group and the chemotherapy alone group (34.2% and 36.8%, respectively).<h4>Conclusions</h4>Anti-PD-1 therapy plus chemotherapy is an effective and tolerable approach for advanced BTC.2019-01-01T00:00:00Z2019 Sep2025-04-21T23:21:10.459Z2019-10-30T08:17:34ZS-EPMC67688923153516010.1007/s00262-019-02386-w