First-line chemotherapy or in combination with programmed cell death protein-1 antibody in patients with metastatic or recurrent biliary tract cancer

Background Although immune checkpoint blockade therapy has achieved great success in various types of cancers, studies on biliary tract cancer are limited. This study aimed to assess the efficacy and tolerability of immune checkpoint inhibitors (ICIs) combined with chemotherapy in Chinese patients with BTC. Methods We collected medical records of 130 pathologically diagnosed metastatic or recurrent BTC patients who had not received chemotherapy in the advanced stage. Eligible patients who received first-line chemotherapy +/- ICIs were enrolled in the efficacy and safety analysis. We compared progression-free survival (PFS), overall survival (OS), objective response rate (ORR), and duration of response (DoR) between the ICI plus chemotherapy group and chemotherapy alone group. Results Of 90 enrolled patients, 45 received ICIs plus chemotherapy and 45 received chemotherapy. The median follow-up times were 18.7 and 19.6 months, respectively. The median PFS was 5.9 months (95% CI: 4.3-7.5) with ICIs plus chemotherapy, which was significantly longer than the 4.2 months (95% CI: 2.1-6.5) with chemotherapy (hazard ratio [HR] 0.62, 95% CI: 0.39-0.94; P = 0.0306). The median OS was 14.7 months (95% CI: 11.4-18.0) compared with 14.2 months (95% CI: 12.5-15.9) (HR 0.93; 95% CI: 0.57-1.50; P = 0.765). Grade 3 or 4 treatment-related adverse events were similar between these two groups (71.1% and 64.4%, respectively). Conclusion Although first-line ICI therapy plus chemotherapy showed a significant improvement in the median PFS compared with chemotherapy in metastatic or recurrent BTC, the benefit did not translate into a statistically significant OS prolongation. The safety profile for ICIs plus chemotherapy was similar to chemotherapy alone.

Automated summary

The study aimed to evaluate the efficacy and tolerability of immune checkpoint inhibitors (ICIs) combined with chemotherapy in Chinese patients with biliary tract cancer. Results showed that the median progression-free survival (PFS) with ICIs plus chemotherapy was significantly longer than with chemotherapy alone, but there was no statistically significant difference in overall survival (OS) between the two groups. The safety profile for ICIs plus chemotherapy was similar to chemotherapy alone.