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Peer-reviewedCC BY-NC-ND 4.0ESMO Gastrointestinal Oncology

Real-world outcomes of first-line gemcitabine–platinum plus an immune checkpoint inhibitor and subsequent treatment with matched targeted therapy in biliary tract cancer

  • H. Yoon1
  • H. Jeong1
  • K.P. Kim1
  • I. Park1
  • T.J. Song2
  • D. Oh2
  • G. Huh2
  • S.H. Cho2
  • Y. Lee2
  • M. Nuh3
  • M. Hsiang3
  • Q. Kimana4
3 more authors
  • M.M. Javle4
  • S. Lee4
  • C. Yoo1
4 affiliations
  1. 1Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea
  2. 2Department of Gastroenterology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea
  3. 3Department of Internal Medicine, Baylor College of Medicine, Houston, USA
  4. 4Department of Gastrointestinal Medical Oncology, University of Texas MD Anderson Cancer Center, Houston, USA
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Abstract

Background: Gemcitabine plus platinum combined with an immune checkpoint inhibitor (GP-ICI) is standard first-line therapy for advanced biliary tract cancer (BTC); however, tumor-site distribution, molecular epidemiology, and postprogression treatment access vary globally. We compared real-world outcomes and postprogression treatment patterns between US and South Korean tertiary centers.

Patients and methods: Patients with advanced BTC receiving first-line GP-ICI at MD Anderson Cancer Center (MDACC) or Asan Medical Center (AMC) between 2021 and 2025 were retrospectively analyzed. Outcomes included objective response rate (ORR), progression-free survival (PFS), overall survival (OS), and postprogression survival (PPS). Molecular profiling and treatment patterns were evaluated.

Results: Among 375 patients (MDACC, n = 121; AMC, n = 254), intrahepatic cholangiocarcinoma was observed at a higher proportion at MDACC (71.9% versus 42.1%), whereas extrahepatic cholangiocarcinoma was more common at AMC (10.7% versus 41.7%). ORR (21.5% versus 15.7%), median PFS (6.6 versus 7.2 months), and median OS (19.5 versus 14.9 months) were comparable. Median PPS was significantly longer at MDACC (13.1 versus 5.8 months; P = 0.002). IDH1 mutations and FGFR2 fusions were more common at MDACC, whereas HER2 alterations were more frequent at AMC. Among patients with actionable alterations, matched targeted therapy was administered more frequently at MDACC (75.8% versus 12.8%; P < 0.001) and was associated with significantly longer PPS [hazard ratio (HR) 0.15, 95% confidence interval (CI) 0.06-0.41, P < 0.001] and a trend toward longer OS (HR 0.54, 95% CI 0.26-1.10, P = 0.089).

Conclusions: Real-world GP-ICI outcomes were comparable between US and South Korean cohorts. The differences in molecular profiling, matched targeted therapy utilization, and postprogression outcomes underscore the need for broader molecular profiling and improved targeted therapy access in advanced BTC.

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Cite

BibTeXRIS
BibTeX
@article{yoon2026realworld,
  title = {{Real-world outcomes of first-line gemcitabine–platinum plus an immune checkpoint inhibitor and subsequent treatment with matched targeted therapy in biliary tract cancer}},
  author = {H. Yoon and H. Jeong and K.P. Kim and I. Park and T.J. Song and D. Oh and G. Huh and S.H. Cho and Y. Lee and M. Nuh and M. Hsiang and Q. Kimana and M.M. Javle and S. Lee and C. Yoo},
  journal = {ESMO Gastrointestinal Oncology},
  year = {2026},
  month = oct,
  doi = {10.1016/j.esmogo.2026.100437},
  url = {https://doi.org/10.1016/j.esmogo.2026.100437},
  note = {CC BY-NC-ND 4.0},
}
RIS
TY  - JOUR
TI  - Real-world outcomes of first-line gemcitabine–platinum plus an immune checkpoint inhibitor and subsequent treatment with matched targeted therapy in biliary tract cancer
AU  - H. Yoon
AU  - H. Jeong
AU  - K.P. Kim
AU  - I. Park
AU  - T.J. Song
AU  - D. Oh
AU  - G. Huh
AU  - S.H. Cho
AU  - Y. Lee
AU  - M. Nuh
AU  - M. Hsiang
AU  - Q. Kimana
AU  - M.M. Javle
AU  - S. Lee
AU  - C. Yoo
PY  - 2026
DA  - 2026/10/28
JO  - ESMO Gastrointestinal Oncology
DO  - 10.1016/j.esmogo.2026.100437
UR  - https://doi.org/10.1016/j.esmogo.2026.100437
AB  - Background: Gemcitabine plus platinum combined with an immune checkpoint inhibitor (GP-ICI) is standard first-line therapy for advanced biliary tract cancer (BTC); however, tumor-site distribution, molecular epidemiology, and postprogression treatment access vary globally. We compared real-world outcomes and postprogression treatment patterns between US and South Korean tertiary centers.  Patients and methods: Patients with advanced BTC receiving first-line GP-ICI at MD Anderson Cancer Center (MDACC) or Asan Medical Center (AMC) between 2021 and 2025 were retrospectively analyzed. Outcomes included objective response rate (ORR), progression-free survival (PFS), overall survival (OS), and postprogression survival (PPS). Molecular profiling and treatment patterns were evaluated.  Results: Among 375 patients (MDACC, n = 121; AMC, n = 254), intrahepatic cholangiocarcinoma was observed at a higher proportion at MDACC (71.9% versus 42.1%), whereas extrahepatic cholangiocarcinoma was more common at AMC (10.7% versus 41.7%). ORR (21.5% versus 15.7%), median PFS (6.6 versus 7.2 months), and median OS (19.5 versus 14.9 months) were comparable. Median PPS was significantly longer at MDACC (13.1 versus 5.8 months; P = 0.002). IDH1 mutations and FGFR2 fusions were more common at MDACC, whereas HER2 alterations were more frequent at AMC. Among patients with actionable alterations, matched targeted therapy was administered more frequently at MDACC (75.8% versus 12.8%; P < 0.001) and was associated with significantly longer PPS [hazard ratio (HR) 0.15, 95% confidence interval (CI) 0.06-0.41, P < 0.001] and a trend toward longer OS (HR 0.54, 95% CI 0.26-1.10, P = 0.089).  Conclusions: Real-world GP-ICI outcomes were comparable between US and South Korean cohorts. The differences in molecular profiling, matched targeted therapy utilization, and postprogression outcomes underscore the need for broader molecular profiling and improved targeted therapy access in advanced BTC.
SN  - 2949-8198
ER  -