Abstract 120P
Background
Biliary tract cancers (BTC) including cholangiocarcinoma (CC) and gallbladder cancer (GBC) are rare cancers with poor prognosis. Few data are available on early-onset BTC (EOBTC) defined as patients (pts) under age of 50.
Methods
A retrospective chart review was performed in pts treated for BTC in 14 French centers between 2003 and 2021. Data on demographic characteristics, therapeutic management, and molecular profile were collected. Progression-free survival (PFS) and overall survival (OS) were estimated by the Kaplan Meier method. Prognostic factors were assessed by univariate and multivariate analyses by Cox regression.
Results
Overall, 1256 pts with BTC were included. Patients with EOBTC (n=188, 15%) had less comorbidities according to Charlson score (63.5% vs 84.4%, p<0.0001), higher tumor stage (cT3-4: 49.9% vs 32.17%, p=0.0126), bilobar liver involvement (47.7% vs 32.1%, p=0.0002) and metastatic disease (67.5% vs 57.49%, p=0.0097) compared to older pts, but did not differ regarding primary tumor location (intrahepatic vs extrahepatic CC vs GBC), WHO performance status (PS 0-1: 94.4% vs 85.5%, p=0.15), and sex-ratio (50.8% vs 53.4% of males). First-line systemic therapy for advanced BTC (n=818, 65.2%) was mostly a doublet by GEMCIS (45.5% vs 32.1%, p=0.0091) or GEMOX (43.3% vs 56.5%, p=0.0091) in EOBTC vs non-EOBTC respectively. EOBTC pts received more frequently a 2nd line therapy (89.5% vs 80.9%, p=0.02). For advanced BTC pts, median (m)OS was 17.0 mo vs 16.2 mo (p=0.08) and mPFS was 5.8 mo vs 6.0 mo (p=0.89), in EOBTC vs older pts respectively. Molecular profiling was performed in 72.6% of EOBTC pts vs 52.4% in older pts (p=0.0019), and less actionable alterations were found (e.g. IDH1 mutations, 7.8% vs 16.6%; p=0.029; FGFR2-fusion, 11.7% vs 8.9%; p=0.029). We did not find any usual prognostic factors in BTC (CEA, CA19-9, PS, neutrophile-lymphocyte ratio, number of extra-hepatic metastases) associated with EOBTC survival.
Conclusions
Pts with EOBTC have a more advanced disease at diagnosis, are treated more heavily at an advanced stage, and have similar PFS and OS in comparison to older BTC pts. Molecular profiling was more often performed in EOBTC pts but less actionable alterations were found.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
GERCOR.
Funding
ACABI: Association pour l'étude des cancers et affections des voies biliaires.
Disclosure
J. Edeline: Financial Interests, Personal, Invited Speaker: Roche, AstraZeneca, MSD, Bayer, Boston Scientific, Eisai, Ipsen, Servier; Financial Interests, Personal, Advisory Board: Roche, AstraZeneca, MSD, Bayer, Merck Serono, Eisai, Ipsen, BMS, Basilea, Servier, Incyte, BeiGene, Taiho; Financial Interests, Personal, Steering Committee Member: MSD; Financial Interests, Institutional, Coordinating PI: BMS, BeiGene; Financial Interests, Institutional, Local PI: BMS, MSD, Roche, BeiGene, Bayer, Novartis, Taiho, Servier, Agios; Non-Financial Interests, Principal Investigator: Unicancer. C. Tournigand: Non-Financial Interests, Leadership Role: GERCOR; Non-Financial Interests, Advisory Role: Fondation APHP. T. Lecomte: Financial Interests, Personal, Invited Speaker: Ipsen, Pierre Fabre, AstraZeneca, BMS; Financial Interests, Personal, Advisory Board: Sanofi, Merck Serono, Servier, Amgen, Deciphera, Advanced Accelerator Application Pierre Fabre; Financial Interests, Institutional, Local PI: AstraZeneca, Mirati, ALX Oncology; Financial Interests, Institutional, Funding: Leopharma, Pierre Fabre. D. Tougeron: Financial Interests, Personal, Advisory Board: AstraZeneca, Sanofi, AMGEN, MSD, Roche, Servier, Pierre Fabre, BMS, Bayer; Non-Financial Interests, Member of Board of Directors: Federation francophone de cancerologie digestive. V. Hautefeuille: Financial Interests, Personal, Invited Speaker: Novartis, Merck, Amgen; Financial Interests, Personal, Advisory Board: AAA, Ipsen, Pierre Fabre; Financial Interests, Institutional, Invited Speaker: Deciphera, Esteve. N. Williet: Financial Interests, Personal, Advisory Board: Accord healthcare, AstraZeneca, Mayoly, MSD, Pierre Fabre, Servier, Viatris, LeoPharma; Financial Interests, Personal, Non-financial benefits: Ipsen, Merck, MSD, Servier. J. Bachet: Financial Interests, Personal, Advisory Board: Amgen, AstraZeneca, Bayer, BMS, GSK, Merck-Serono, MSD, Pierre Fabre, Roche, Sanofi, Servier; Non-Financial Interests, Personal, Other: Amgen, Merck Serono, Roche, Servier. A. hollebecque: Financial Interests, Personal, Invited Speaker: Servier, Incyte, Eisai; Financial Interests, Personal, Advisory Board: Basilea, Tahio, Relay Theraeutics, QED Therapeutics, Debiopharm, MSD, Boehringer Ingelheim; Financial Interests, Institutional, Funding: Incyte; Financial Interests, Institutional, Research Grant: AstraZeneca; Non-Financial Interests, Principal Investigator, M19-345: AbbVie; Non-Financial Interests, Principal Investigator, CO42216; WP42627; CO40939: Roche; Non-Financial Interests, Principal Investigator, MCLA-158: Merus; Non-Financial Interests, Principal Investigator, SGNB6A: Seattle Genetics; Non-Financial Interests, Principal Investigator, TAS-120-202: Tahio; Non-Financial Interests, Principal Investigator, Krystal-10: Mirati; Non-Financial Interests, Principal Investigator, ADP-0033: Adaptimmune; Non-Financial Interests, Principal Investigator, ACT16902: Sanofi; Non-Financial Interests, Principal Investigator, C4201002: Pfizer; Non-Financial Interests, Principal Investigator, RLY-4008: Relay Therapeutics; Non-Financial Interests, Principal Investigator, CC-90011: Celgene/BMS; Non-Financial Interests, Principal Investigator, Loxo-IDH: Loxo/Lilly; Non-Financial Interests, Principal Investigator: AstraZeneca; Non-Financial Interests, Principal Investigator, SN-201 study: Sotio; Non-Financial Interests, Principal Investigator, Tropics-03: Gilead; Non-Financial Interests, Principal Investigator, BI1403: Boehringer Ingelheim. D. Malka: Financial Interests, Personal, Advisory Board: AbbVie, Agios, Amgen, AstraZeneca, BMS, Bayer, HalioDx, Incyte, MSD, Merck Serono, Pierre Fabre Oncologie, Roche, Servier, Taiho; Financial Interests, Personal, Invited Speaker: Amgen, AstraZeneca, BMS, Bayer, Foundation Medicine, HalioDx, Incyte, Leo Pharma, MSD, Merck Serono, Pierre Fabre Oncologie, Roche, Sanofi, Servier, Viatris, Veracyte; Financial Interests, Personal, Writing Engagement: Medscape; Other,bTravel expenses for medical congresses: Amgen, Bayer, BMS, Merck Serono, Pierre Fabre Oncologie, Roche, Sanofi, Servier, Viatris. C. Neuzillet: Financial Interests, Personal, Advisory Board: Amgen, AstraZeneca, Baxter, BMS, Fresenius Kabi, Incyte Biosciences, Merck, MSD, MundiPharma, Viatris, Novartis, Nutricia, OSE Immunotherapeutitcs, Pierre Fabre, Roche, Sanofi, Servier; Financial Interests, Institutional, Research Funding: AstraZeneca, BMS, Fresenius Kabi, Nutricia, OSE Immunotherapeutics, Roche, Servier, Viatris. A. Turpin: Financial Interests, Personal, Expert Testimony: Merck Serono, Viatris, Incyte Biosciences; Financial Interests, Personal, Advisory Board: Servier, AstraZeneca, MSD, BMS. All other authors have declared no conflicts of interest.
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