The efficacy and safety analysis of immunotherapy combined with chemotherapy with or without targeted therapy versus chemotherapy alone as second-line treatment for advanced biliary tract malignant tumors

Objective The treatment of biliary tract cancer (BTC) keep still limited, especially after the progress of first-line treatment. Immune checkpoint inhibitors (ICIs) have been proved effects in various of solid tumors, so as BTC. But their efficacy and safety in patients with advanced BTC are still need further exploration. Methods The clinical information of 126 patients diagnosed with advanced BTC between 2014 and 2024 who received the first-line therapy or neoadjuvant therapy but assessing progress or intolerance were retrospectively reviewed. All patients were treated with chemotherapy, while 57 of them were treated with ICIs in combination targeted therapy or not, the other 69 of them were not. Therefore, we divided the patients into two groups, SC (simple chemotherapy) and CT (combined treatment), then we analyzed the benefit of adding ICIs with or without targeted therapy according to efficacy, adverse events, progression-free survival (PFS), overall survival (OS), progressive disease (PD), and the influence of various factors and effectiveness. Results The mean PFS was 4.68 months for CT group and 3.50 months for SC group. And the mean OS was 30.26 months for CT group and 17.14 months for SC group. The PFS and OS were prolonged by 1.18 months and 13.12 months with ICIs with or without targeted therapy respectively, and the difference were statistically significant (p = 0.0012; p < 0.001). The objective response rate (ORR) was 5.26% (3/57) for the CT group versus 1.45% (1/69) for the SC group, and the disease control rate (DCR) was 54.39% (31/57) versus 33.33% (23/69), respectively. Regression analysis demonstrated that factors such as TNM stage, T stage, histological grade, whether CA199 decreased, and therapeutic evaluation grade were related to OS (p < 0.05). For the treatment-related adverse events (TRAEs), the highest grade 3–4 adverse effects was hemoglobin count decreased (29.37%). Conclusion Our study showed that ICIs combined with chemotherapy with or without targeted therapy exhibited good antitumor activity with acceptable safety and could be recommended as second-line treatment for patients with advanced BTC.