Comparison analysis of ICIs and chemotherapy combined with or without lenvatinib as first-line treatment of unresectable intrahepatic cholangiocarcinoma.

BACKGROUND

Effective first-line treatments for unresectable intrahepatic cholangiocarcinoma (ICC) remain limited. This real-world study aimed to compare the efficacy of immune checkpoint inhibitors (ICIs) plus chemotherapy combined with or without Lenvatinib as first-line treatment in unresectable ICC patients and identify predictors of treatment response and prognosis.

METHODS

In this retrospective cohort study, 58 patients with unresectable ICC received either dual therapy (ICIs plus chemotherapy) or triple therapy (ICIs plus chemotherapy and Lenvatinib) as first-line treatment. The endpoints were progression-free survival (PFS), overall survival (OS), objective response rate (ORR), and disease control rate (DCR). Survival curve was plotted by the Kaplan-Meier method. A Cox proportional hazards model was performed to investigate risk factors of PFS and OS.

RESULTS

No significant differences were observed between triple therapy and dual therapy as first-line treatment for unresectable ICC patients in terms of PFS (median PFS: 10.3 vs. 11.1 months, P > 0.05) and OS (median OS: 14.0 vs. 15.0 months, P > 0.05). The ORR (39.4% vs. 30.4%) and DCR (90.9% vs. 73.9%) were comparable between the triple therapy group and dual therapy group (P > 0.05). In the multivariate analysis, tumor burden score (TBS, ≥ 8) and tumor number (≥ 2) were associated with prolonged PFS (P < 0.05), while TBS was an independent factor for OS (P < 0.05).

CONCLUSIONS

Triple therapy did not demonstrate any benefit on both PFS and OS compared to dual therapy as first-line treatment for patients with unresectable ICC. TBS and tumor number may guide treatment stratification.