Abstract 291P
Background
There is currently a rise in the incidence of biliary tract cancers (BTC) among younger individuals (≤50 years). This study investigates the clinical, therapeutical and molecular characteristics and outcomes of young-onset BTC (YO-BTC) patients (pts).
Methods
All consecutive pts with a histologically confirmed BTC treated at two European cancer centers, Gustave Roussy (France) and Vall d’Hebron Institute of Oncology (Spain), from 2011 to 2021 were identified. Clinical data were extracted from medical records. Genomic data, classified according to the ESMO Scale for Clinical Actionability of Molecular Targets (ESCAT), and data regarding the efficacy of targeted therapies were obtained. Particularly, the Growth Modulation Index (GMI), which is the ratio of progression free survival (PFS) with the targeted therapy line to the PFS of the n-1 line, was calculated. Patients were categorized in YO-BTC (≤50 years), 51-69 years and late-onset (LO-BTCs, ≥70 years).
Results
Among 1023 pts with BTC, 184 (18%) had YO-BTC, 561 (54.8%) were 51-69y, and 278 (27.2%) were LO-BTCs. Median OS of patients with metastatic disease was higher in YO-BTC: 22 months (95% CI: 18-26) vs 18 months (95%CI: 17-20) in 51-69 (p = 0.010) and 15 months (95%IC: 13-17) in LO-BTC (p<0.001), despite a higher tumoral burden with more liver bilobar involvement. Molecular profiling was conducted in YO-BTC, 51-69 years and LO-BTCs in 80%, 77% and 62% respectively. Among the actionable molecular alterations, FGFR2 fusions were more frequent in YO-BTC (12% vs 5.7 % and 4.3% respectively, p = 0.038), the rates of other actionable molecular alterations (IDH1, HER2, BRAFV600E, NTRK, MSI) did not differ by age categories. YO-BTC pts received more targeted therapies in second or later lines (48% vs. 37% vs. 29%, p=0.020). Among pts with a molecular-matched targeted therapy, the GMI was higher in YO-BTC (GMI>1.33: 61.1% vs 39.2% vs 33.3% respectively), and OS was longer, especially for the ESCAT I-IIIA alterations: median OS: 35 (27, 43) months vs 21 (19, 38) months in LO-BTC, p =0.016.
Conclusions
YO-BTC patients have improved outcomes in the metastatic setting compared to older patients. They are enriched for FGFR2 fusions, which open opportunities for precision oncology-based approaches.
Legal entity responsible for the study
Gustave Roussy and Vall d'Hebron Institute of Oncology.
Funding
This Research Project was supported by ESMO with the aid of a grant from BMS. Any views, opinions, findings, conclusions, or recommendations expressed in this material are those solely of the author(s) and do not necessarily reflect those of ESMO.
Disclosure
A. Turpin: Financial Interests, Personal and Institutional, Research Grant, This Research Project was supported by ESMO with the aid of a grant from BMS. Any views, opinions, findings, conclusions, or recommendations expressed in this material are those solely of the author(s) and do not necessarily reflect those of ESMO": BMS; Financial Interests, Personal, Invited Speaker: Servier, Viatris, Incyte Biosciences, MSD; Financial Interests, Personal, Expert Testimony: Merck; Financial Interests, Personal, Advisory Board: AstraZeneca. All other authors have declared no conflicts of interest.