ArticleBMC medicine2024
Genomic characterization and immunotherapy for microsatellite instability-high in cholangiocarcinoma.
Article in BMC medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03892577 (Real-world Study for Targeted Therapy and Immunotherapy in Patients With Advanced Hepatobiliary Tumors), which is not on this map. Cited by 32 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Real-world Study for Targeted Therapy and Immunotherapy in Patients With Advanced Hepatobiliary Tumors: a Multi-centers, Open-assess Observational Study.
Who cites it
32 citing papers in PubMed, 1 synthesis or guideline pooled it, 40 citations in OpenAlex.
- Durvalumab and pembrolizumab in advanced biliary tract cancer: a reconstructed patient-level mimic head-to-head comparative analysis.Frontiers in immunology · 2024Pooled it
- Efficacy and biomarker analysis of second-line nab-paclitaxel plus sintilimab in patients with advanced biliary tract cancer.Cancer science · 2024Trial
- Long-Term Outcomes and Surgical Conversion After Immunotherapy in Microsatellite Instability-H Biliary Tract Cancers.JCO precision oncology · 2026Observational
- Clinicopathologic spectrum and diagnostic strategies of intrahepatic neoplasms with prominent inflammatory background.Virchows Archiv : an international journal of pathology · 2026Article
- Efficacy and safety of immune checkpoint inhibitors in patients with high microsatellite instability/mismatch repair-deficient advanced cholangiocarcinoma: A propensity score-matched study.JHEP reports : innovation in hepatology · 2026Article
- Tumor mutation burden predicts aggressiveness and prognosis of gastrointestinal stromal tumor.Translational cancer research · 2026Article
- Molecular landscape and biomarker associations of functionalTranslational cancer research · 2026Article
- Article
- Recent advances in molecular classification and multimodal treatment of intrahepatic cholangiocarcinoma.World journal of surgical oncology · 2026Review
- Immune checkpoint blockade and thrombo-inflammatory remodeling in microsatellite instability-high intrahepatic cholangiocarcinoma with Trousseau syndrome: a case report.Frontiers in medicine · 2026Article
- The central role of radiotherapy in remodeling the tumor immune microenvironment: mechanisms and therapeutic implications.Frontiers in cell and developmental biology · 2026Review
- From the invasive front to organotropic pre-metastatic niches: spatial immune regulatory networks governing cholangiocarcinoma dissemination and metastasis-intercepting immunotherapy.Frontiers in immunology · 2026Review
- From PD-1/PD-L1 to tertiary lymphoid structures: Paving the way for precision immunotherapy in cholangiocarcinoma treatment.Human vaccines & immunotherapeutics · 2025Article
- KDM4A-driven SMAD5/TGFβ axis activation as a therapeutic target in cholangiocarcinoma: mechanistic insights and translational implications.Cell & bioscience · 2025Article
- Circulating miRNAs as potentially predictive biomarkers for chemoimmunotherapy in advanced biliary tract cancer: a post-hoc analysis of the phase II T1219 study.NPJ precision oncology · 2025Article
- A multimodal fusion system predicting survival benefits of immune checkpoint inhibitors in unresectable hepatocellular carcinoma.NPJ precision oncology · 2025Article
- Predicting Immunotherapy Response in Unresectable Hepatocellular Carcinoma: A Comparative Study of Large Language Models and Human Experts.Journal of medical systems · 2025Article
- Deficient Mismatch Repair and Microsatellite Instability in Solid Tumors.International journal of molecular sciences · 2025Review
- Interview with the World Class Authorities Frontiers of Cancer Research: An exclusive interview with Professor Luis Diaz.Annals of gastroenterological surgery · 2025Article
- Molecular and Pathological Heterogeneity of Synchronous Small and Large Duct Intrahepatic Cholangiocarcinoma-A Case Series.Current oncology (Toronto, Ont.) · 2025Article
Corrections and comments
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Authors and funding
33 authors at 6 institutions in 2 countries.
Funding
Abstract
backgroundMicrosatellite instability-high (MSI-H) is a unique genomic status in many cancers. However, its role in the genomic features and immunotherapy in cholangiocarcinoma (CCA) is unclear. This study aimed to systematically investigate the genomic characterization and immunotherapy efficacy of MSI-H patients with CCA.
methodsWe enrolled 887 patients with CCA in this study. Tumor samples were collected for next-generation sequencing. Differences in genomic alterations between the MSI-H and microsatellite stability (MSS) groups were analyzed. We also investigated the survival of PD-1 inhibitor-based immunotherapy between two groups of 139 patients with advanced CCA.
resultsDifferential genetic alterations between the MSI-H and MSS groups included mutations in ARID1A, ACVR2A, TGFBR2, KMT2D, RNF43, and PBRM1 which were enriched in MSI-H groups. Patients with an MSI-H status have a significantly higher tumor mutation burden (TMB) (median 41.7 vs. 3.1 muts/Mb, P < 0.001) and more positive programmed death ligand 1 (PD-L1) expression (37.5% vs. 11.9%, P < 0.001) than those with an MSS status. Among patients receiving PD-1 inhibitor-based therapy, those with MSI-H had a longer median overall survival (OS, hazard ratio (HR) = 0.17, P = 0.001) and progression-free survival (PFS, HR = 0.14, P < 0.001) than patients with MSS. Integrating MSI-H and PD-L1 expression status (combined positive score ≥ 5) could distinguish the efficacy of immunotherapy.
conclusionsMSI-H status was associated with a higher TMB value and more positive PD-L1 expression in CCA tumors. Moreover, in patients with advanced CCA who received PD-1 inhibitor-based immunotherapy, MSI-H and positive PD-L1 expression were associated with improved both OS and PFS.
trial registrationThis study was registered on ClinicalTrials.gov on 07/01/2017 (NCT03892577).
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.