ArticleJHEP reports : innovation in hepatology2025
Recent advances in systemic therapy for advanced biliary tract cancer: A systematic review and meta-analysis using reconstructed RCT survival data.
Article in JHEP reports : innovation in hepatology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
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Who cites it
15 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Observational
- Gemcitabine-cisplatin plus durvalumab in advanced intrahepatic cholangiocarcinoma: effectiveness outcomes and characterization of the tumor microenvironment.Hepatology international · 2026Article
- Perioperative approaches for patients with biliary tract cancer.Nature reviews. Clinical oncology · 2026Review
- The allosteric IDH1 inhibitor ivosidenib overcomes chemoresistance in intrahepatic cholangiocarcinoma models expressing wild-type IDH1.The Journal of clinical investigation · 2026Article
- Synergistic anti-tumor activity of andrographolide and gemcitabine in intrahepatic cholangiocarcinoma through inhibition of RRM2 and the JAK/STAT3 pathway.Cancer chemotherapy and pharmacology · 2026Article
- Liver resection with and without vascular resection versus transplantation for de novo perihilar cholangiocarcinoma.Hepatology (Baltimore, Md.) · 2026Article
- How to efficiently establish animal models of cholangiocarcinoma: challenges and inspiration.Medical review (2021) · 2026Review
- MAPK Pathway Mutations Emerge in Mutant IDH1 Inhibitor-Resistant Cholangiocarcinoma and Attenuate the IFN Response.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026Article
- Prognostic Impact of Metastatic Sites in Patients with Biliary Tract Cancer Treated with Cisplatin, Gemcitabine, and Durvalumab.Targeted oncology · 2026Article
- Present and future of liver transplantation for cholangiocellular carcinoma: moving toward personalized multiparametric transplantability patterns.Transplant international : official journal of the European Society for Organ Transplantation · 2026Review
- Cholangiocarcinoma in the Era of Precision Medicine: Emerging Insights and Therapeutic Strategies.Current pharmaceutical design · 2026Review
- A pathomics-integrated multimodal model to evaluate chemoimmunotherapy efficacy in unresectable intrahepatic cholangiocarcinoma.JHEP reports : innovation in hepatology · 2025Article
- Conversion to Resection After Neoadjuvant Gemcitabine, Cisplatin, and Nab-Paclitaxel for Borderline Resectable CCA: A Real-World Exploratory Analysis.Annals of surgical oncology · 2025Article
- Clinical Utility and Prognostic Implications of Circulating Cell-Free DNA in Biliary Tract Cancer.JCO precision oncology · 2025Article
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background & Aims: Gemcitabine/cisplatin (GemCis) was the long-standing first-line treatment for advanced biliary tract cancers (BTCs). Following positive results from the TOPAZ-01 and KEYNOTE-966 trials, immune checkpoint inhibitors (ICIs) combined with chemotherapy are now the standard of care. We aim to compare the efficacy of first-line therapies for advanced BTCs. Methods: Our systematic review included studies from five databases focusing on English-language articles published between January 2010 and June 2024. We included randomized clinical trials (RCTs) that featured GemCis in a treatment arm for treatment-naive adults with advanced BTCs. The primary endpoints were overall survival (OS) and progression-free survival. We conducted a one-stage meta-analysis using reconstructed survival data, Cox-based models, and restricted mean survival time (RMST). Results: After screening 8,797 studies, 17 RCTs were selected, involving a total of 4,584 patients. Of these, 2,140 (46.7%) received GemCis. The majority (68.9%) were diagnosed with intrahepatic or extrahepatic cholangiocarcinoma, and 80% had metastatic disease at the time of treatment. The pooled median OS in the GemCis group was 11.6 months (95% CI 11.3-12.2 months). GemCis plus pembrolizumab (hazard ratio [HR] 0.99, 95% CI 0.98-0.99; Conclusions: Despite incremental advancements, a breakthrough in advanced BTC treatment remains elusive. Further improvements in treatment efficacy may require biomarker identification to optimize combinational therapies for better patient selection. Impact and implications: This study analyzed recent RCTs, including KEYNOTE-966, TOPAZ-1, NIFE, and SWOG 1815, involving 4,584 patients with advanced biliary tract cancer. A meta-analysis of 17 treatment arms, using reconstructed survival data, confirmed the modest survival benefit of GemCis plus ICIs, supporting its guideline adoption. The findings, however, highlight the need for biomarker identification and better patient selection.
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