Role of the immunotherapy in the treatment of biliary tract cancers: Review of current clinical trials and retrospective analysis

Introduction. Biliary tract cancers (BTC) are aggressive malignancies with limited therapeutic options and poor overall survival. In recent years, the introduction of immunotherapy has shifted the treatment paradigm for BTC, particularly in combination with chemotherapy. This study presents a detailed analysis of current clinical data and evaluates the efficacy of chemoimmunotherapy in patients with unresectable and/or metastatic cholangiocarcinoma in routine clinical practice. Aim. To assess the clinical outcomes of combined chemoimmunotherapy (GemCis + durvalumab/pembrolizumab) in patients with BTC in Russian clinical settings, including overall survival (OS), progression-free survival (PFS), objective response rate (ORR), and toxicity profiles. Mat erials and methods. This retrospective analysis included 23 patients with histologically confirmed cholangiocarcinoma treated between 2020 and 2024 at three centers of JSC “MEDSI Group”. The standard treatment regimen consisted of gemcitabine, cisplatin, and a PD-1/PD-L1 inhibitor followed by maintenance immunotherapy. Treatment efficacy was evaluated according to RECIST v1.1 criteria, and survival analysis was performed using the Kaplan–Meier method. Results. The median PFS was 8.0 months (95% CI: 6.6–9.3), and the median OS was 14.0 months (95% CI: 8.4–19.5). The ORR was 25%, with five partial responses and no complete responses; the disease control rate was 68.5%. Grade 3–4 adverse events occurred in 39% of patients, including hematologic toxicity and rare immune-related adverse events. Conclusion. The findings of this analysis support the real-world efficacy of chemoimmunotherapy and are consistent with data from large international trials. Incorporation of PD-1/PD-L1 inhibitors into first-line treatment regimens for BTC provides a clinically meaningful improvement in survival outcomes and disease control, particularly among patients demonstrating favorable responses to therapy.