Real-world treatment patterns and early outcomes in advanced intrahepatic cholangiocarcinoma: a descriptive case series

Intrahepatic cholangiocarcinoma (ICC) has a poor prognosis, with 70-80% of patients presenting advanced/metastatic disease. Conventional first-line chemotherapies have limitations, and targeted-immunotherapy combinations need real-world validation. This retrospective, single-center case series enrolled 12 advanced ICC patients to describe the real-world clinical characteristics, treatment patterns which including lenvatinib-based targeted-immunotherapy with or without Transarterial Interventional Therapies (TAIT) and conventional chemotherapy, as well as early efficacy and safety outcomes; and to generate preliminary hypotheses for subsequent prospective studies, without any intention to compare the efficacy of different regimens. Among the 12 patients, objective response rate was 33.3%, and disease control rate 83.33%. Nine received lenvatinib + PD-1/PD-L1 inhibitors (targeted-immunotherapy group), and 3 received systemic chemotherapy. Among patients receiving lenvatinib + PD-1/PD-L1 inhibitors, 22.22% experienced grade ≥ 3 treatment-related adverse events (TRAEs) with a median treatment duration of 9.0 months; among patients receiving conventional chemotherapy, 66.7% experienced grade ≥ 3 TRAEs with a median treatment duration of 19.0 months. Early TAIT (≤ 2 weeks) was associated with higher tumor reduction (mean 30.52 vs. 7.69% for non-early), which may help alleviate tumor-related symptoms in patients with high initial tumor burden. Baseline CA19-9 and albumin/globulin ratio showed exploratory potential associations with treatment efficacy without formal statistical analysis; larger initial diameter correlated with better response. This study describes that lenvatinib + Immune Checkpoint Inhibitors (ICIs) combined with early TAIT may be a feasible multimodal strategy used in real-world practice for advanced ICC, with preliminary observations of feasibility. Limitations include small single-center sample and short follow-up. The high heterogeneity of treatment regimens may also limit the comparability of efficacy and safety. Larger prospective studies are needed.