Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population

Abstract Background Biliary tract cancers (BTCs) are a heterogeneous group of malignant cancers with an overall poor prognosis. For more than a decade, the standard palliative first-line therapy was cytotoxic chemotherapy with gemcitabine/cisplatin. The results of the TOPAZ-1 and KEYNOTE-966 trials...

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Main Authors: Florian Gerhardt, Christian Müller, Marino Venerito, Jack Chater, Raphael Mohr, Mara Egerer, Udo Lindig, Aaron Schindler, Sebastian Ebel, Janett Fischer, Maik Schwarz, Sonja Gehring, Thomas Berg, Florian van Bömmel
Format: Article
Language:English
Published: Springer 2025-06-01
Series:Journal of Cancer Research and Clinical Oncology
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Online Access:https://doi.org/10.1007/s00432-025-06239-1
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author Florian Gerhardt
Christian Müller
Marino Venerito
Jack Chater
Raphael Mohr
Mara Egerer
Udo Lindig
Aaron Schindler
Sebastian Ebel
Janett Fischer
Maik Schwarz
Sonja Gehring
Thomas Berg
Florian van Bömmel
author_facet Florian Gerhardt
Christian Müller
Marino Venerito
Jack Chater
Raphael Mohr
Mara Egerer
Udo Lindig
Aaron Schindler
Sebastian Ebel
Janett Fischer
Maik Schwarz
Sonja Gehring
Thomas Berg
Florian van Bömmel
author_sort Florian Gerhardt
collection DOAJ
description Abstract Background Biliary tract cancers (BTCs) are a heterogeneous group of malignant cancers with an overall poor prognosis. For more than a decade, the standard palliative first-line therapy was cytotoxic chemotherapy with gemcitabine/cisplatin. The results of the TOPAZ-1 and KEYNOTE-966 trials have now introduced immune checkpoint inhibitors (ICIs) into first-line therapy. Methods Between July 2022 and March 2024, we retrospectively analyzed patients with advanced BTC who were treated with gemcitabine/cisplatin and durvalumab (GCD) at collaborating German university hospitals, tertiary hospitals, and outpatient oncology practices. Results A total of 90 patients were enrolled. The median overall survival (mOS) was 16 months, and the median progression-free survival (mPFS) was 5 months. The overall response rate (ORR) was 11.1%, and the disease control rate (DCR) was 41.1%. A perihilar primary tumor was significantly associated with better mPFS, while age group between 70 and 75 years and performance status of ECOG 2 at treatment initiation were significantly associated with poorer mOS. Adverse events (AEs) occurred in a total of 64% of patients. The most common grade 1 and grade 2 AEs included anemia (23%), thrombocytopenia (16%), neutropenia (10%), nausea (14%), and fatigue (16%). Grade 3 and grade 4 AEs included anemia (10%), thrombocytopenia (5%), and neutropenia (11%). Only one case of immune-mediated hypothyroidism (imAE) was documented. Conclusion Our real-world data support previously reported findings and further validate ICI based therapy as the standard of care for patients with advanced BTCs.
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spelling doaj-art-6800e98f7a43476fafedc1fc2d0af07b2025-08-20T03:42:37ZengSpringerJournal of Cancer Research and Clinical Oncology1432-13352025-06-01151611010.1007/s00432-025-06239-1Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient populationFlorian Gerhardt0Christian Müller1Marino Venerito2Jack Chater3Raphael Mohr4Mara Egerer5Udo Lindig6Aaron Schindler7Sebastian Ebel8Janett Fischer9Maik Schwarz10Sonja Gehring11Thomas Berg12Florian van Bömmel13Division of Hepatology, Department of Medicine II, Leipzig University Medical CenterDepartment of Gastroenterology, Hepatology and Infectious Diseases, Otto von Guericke University HospitalDepartment of Gastroenterology, Hepatology and Infectious Diseases, Otto von Guericke University HospitalMedical Clinic III, Klinikum ChemnitzDepartment of Hepatology and Gastroenterology, Charité University Medicine BerlinDepartment of Hepatology and Gastroenterology, Charité University Medicine BerlinMedical Clinic II, University Hospital JenaDivision of Hepatology, Department of Medicine II, Leipzig University Medical CenterDepartment of Diagnostic and Interventional Radiology, Leipzig University Medical CenterDivision of Hepatology, Department of Medicine II, Leipzig University Medical CenterMVZ SchöneckDepartment of Gastroenterology, University Hospital MarburgDivision of Hepatology, Department of Medicine II, Leipzig University Medical CenterDivision of Hepatology, Department of Medicine II, Leipzig University Medical CenterAbstract Background Biliary tract cancers (BTCs) are a heterogeneous group of malignant cancers with an overall poor prognosis. For more than a decade, the standard palliative first-line therapy was cytotoxic chemotherapy with gemcitabine/cisplatin. The results of the TOPAZ-1 and KEYNOTE-966 trials have now introduced immune checkpoint inhibitors (ICIs) into first-line therapy. Methods Between July 2022 and March 2024, we retrospectively analyzed patients with advanced BTC who were treated with gemcitabine/cisplatin and durvalumab (GCD) at collaborating German university hospitals, tertiary hospitals, and outpatient oncology practices. Results A total of 90 patients were enrolled. The median overall survival (mOS) was 16 months, and the median progression-free survival (mPFS) was 5 months. The overall response rate (ORR) was 11.1%, and the disease control rate (DCR) was 41.1%. A perihilar primary tumor was significantly associated with better mPFS, while age group between 70 and 75 years and performance status of ECOG 2 at treatment initiation were significantly associated with poorer mOS. Adverse events (AEs) occurred in a total of 64% of patients. The most common grade 1 and grade 2 AEs included anemia (23%), thrombocytopenia (16%), neutropenia (10%), nausea (14%), and fatigue (16%). Grade 3 and grade 4 AEs included anemia (10%), thrombocytopenia (5%), and neutropenia (11%). Only one case of immune-mediated hypothyroidism (imAE) was documented. Conclusion Our real-world data support previously reported findings and further validate ICI based therapy as the standard of care for patients with advanced BTCs.https://doi.org/10.1007/s00432-025-06239-1CholangiocarcinomaBiliary tract cancerLiver cancerImmune checkpoint inhibitorReal-world data
spellingShingle Florian Gerhardt
Christian Müller
Marino Venerito
Jack Chater
Raphael Mohr
Mara Egerer
Udo Lindig
Aaron Schindler
Sebastian Ebel
Janett Fischer
Maik Schwarz
Sonja Gehring
Thomas Berg
Florian van Bömmel
Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population
Journal of Cancer Research and Clinical Oncology
Cholangiocarcinoma
Biliary tract cancer
Liver cancer
Immune checkpoint inhibitor
Real-world data
title Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population
title_full Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population
title_fullStr Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population
title_full_unstemmed Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population
title_short Treatment with gemcitabine/cisplatin and durvalumab for advanced biliary tract cancer – real-world data from a multicenter German patient population
title_sort treatment with gemcitabine cisplatin and durvalumab for advanced biliary tract cancer real world data from a multicenter german patient population
topic Cholangiocarcinoma
Biliary tract cancer
Liver cancer
Immune checkpoint inhibitor
Real-world data
url https://doi.org/10.1007/s00432-025-06239-1
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