Impact of immune checkpoint inhibitors (ICI) in biliary tract cancer (BTC): Real-world outcomes and predictive factors.

• DOI: 10.1200/jco.2025.43.16_suppl.e14653
• Publication Date: 2025-05-28T17:57:49Z
• Authors: Amir Sara; Anne M. Noonan; Arjun Mittra; Ning Jin; Pannaga Malalur; Shafia Rahman; Sameek Roychowdhury; John L. Hays; Ashish Manne
• Keywords: Biliary Tract Cancer; Immune checkpoint

e14653 Background: ICI with gemcitabine and cisplatin (GC-ICI) has become a first-line (FL) standard of care in BTC following the success TOPAZ KEYNOTE-966 trials. A clear understanding factors impacting efficacy will help selecting optimal treatment strategies. This single institute retrospective study is aimed at achieving this. Methods: Patients receiving from 1/2017 6/2023 Ohio State University were evaluated. The patient's baseline characteristics (BL, first dose ICI) extracted details immune-related adverse events (irAE) chart review. Descriptive statistics, Cox proportional hazard (for univariate (UVA) multivariate (MVA) analysis), Wilcoxon test (compare survival) used for survival outcomes that assess time to progression (TTP, (PD) or death) overall (OS). Results: cohort comprised 41 patients, median age 64 (range: 39–88), 51% male, predominantly Caucasian (88%). Nineteen patients received FL alone ICI-based therapy, while remaining second-line (SL) later therapy. Treatment regimens (TR) included single-agent (Sg, n = 25), GC-ICI (n 13), other drug combinations (Ot, 3). 9 MSI-H, 4 FGFR2, 1 IDH1-positive present cohort. Durvalumab was agent all patients. Other key BLs are neutrophil-to-lymphocyte ratio (NLR) 4.77 albumin (Alb) level 3.6 mg/dL. IrAE observed 13 (5 ≥ grade group's TTP OS 3 2-5) 18 (15-26) months, respectively. Factors significantly associated UVA neutrophil count (NC), white cell count, Alb, hemoglobin, trends noted NLR irAE incidence (p 0.06–0.7). For OS, significant line use, stage diagnosis, NLR, TR, MVA. Survival comparisons select described table below. At PD, notable changes rise NC by 2.09 0.06) 8.9 0.01). In group, 4.2 0.04) onset. Conclusions: highlights role irAEs as potential prognostic biomarkers treated ICIs. settings demonstrated modest PFS compared regimens. Notable disparities based on TR BL underscore need personalized strategies optimize BTC. Further research warranted validate these findings. TTP* OS* vs. Ot Sg (0.9) 40 25 (0.0002) SL 5 (0.4) 34 11 (0.0006) irAE, yes no 6 (0.01) 15.5 (0.1) (≤4.77 > 4.77) 2.5 (0.04) 22 Alb (<3.6 ≥3.6) 2 (0.03) 15 26 (0.3) *in months (p-value).