Incidence and prognostic value of actionable mutations in early-stage resectable cholangiocarcinoma.

• DOI: 10.1200/jco.2024.42.3_suppl.545
• Publication Date: 2024-01-22T21:12:58Z
• Authors: Kailey Morgan Oppat; Emilie AK Warren; Frances J. Bennett; Amanda Nottke; Hana E. Littleford; Kim M. Phelps; Karen R. Pennington; Ashokkumar A. Patel; Melinda Bachini; Stacie Lindsey; Shishir K. Maithel
• Keywords: 03 medical and health sciences; 0302 clinical medicine

545 Background: Targeted therapy for actionable mutations in advanced cholangiocarcinoma (CCA) has revolutionized second and third-line treatment algorithms. Their application early stage disease is not defined. The incidence prognostic value of pts with resected known. Methods: Cholangiocarcinoma Foundation Ciitizen (a wholly owned subsidiary Invitae Corporation) collaboratively launched a registry platform that directly consents collects comprehensive medical records. Presently, de-identified data including clinical characteristics, molecular testing, interventions, outcomes have been extracted standardized research use on 400 pts. longitudinal regularly planned updates. We identified who underwent resection non-metastatic genetic testing. Actionable recorded included FGFR2 fusion/rearrangement, IDH1 mutation, HER2 amplification, BRAF MSI-high, TMB-high. Primary outcome was recurrence-free survival (RFS). Results: Of pts, 137 CCA. When compared to disease, those had similar frequency testing presence mutations: 94.3% (n=248) vs 83.9% (n=115) 38.7% (n=96) vs. 33.0% (n=38), respectively. considering non-metastatic, (n=104), the mean age 59.2 yrs, 66 (63.5%) were females, 60 (57.7%) intrahepatic (iCCA), 42 (40.4%) extrahepatic 2 (1.9%) otherwise specified. majority received before or at time surgery (n=89, 85.6%). resectable 34 (32.7%) FGFR2: n=7 (6.7%), IDH1: n=16 (15.4%), HER2: n=5 (4.8%), BRAF: n=0 (0%), MSI-high: n=3 (2.9%), TMB-high: n=8 (7.7%). fusion/rearrangement only iCCA. Pts an younger (49.9 59.8 yrs; p=0.03) all female. Although statistically significant, associated nearly doubling median RFS (32.0 17.3 mos; p=0.19) patients Conclusions: In this highly selected cohort resected, stage, CCA, profiling surgery. As expected, present An seen young, female may favorable as suggested by trend increased RFS. Clinical trials are needed assess administering targeted patient population adjuvant neoadjuvant setting.