Predictors of overall survival in advanced biliary tract cancer in the phase 3 TOPAZ-1 study

Sep 29, 2026

Predictors of overall survival were evaluated in participants with advanced biliary tract cancer treated in the phase 3 TOPAZ-1 study. The post-hoc analysis compared durvalumab plus gemcitabine and cisplatin versus placebo plus gemcitabine and cisplatin and focused on patient- and disease-related factors. It builds on prior findings that durvalumab improved overall survival versus standard-of-care combination therapy without added toxicity, then tests which factors may be prognostic or predictive for survival benefit under immune checkpoint inhibition.

LanguageEnglish

CategoryResearch & Report

FormatPDF

Pages5

Reading Time13 min

AccessFree to download

Introduction

Study background and rationale

Methods

Study design, eligibility, and analysis time points

Results

Evaluation of prognostic and predictive factors

Discussion

Interpretation in the context of immune checkpoint therapy

DOI: 10.1097/HC9.0000000000000868 RE SEARCH L E TTER Predictors of overall survival in advanced biliary tract cancer in the phase 3 TOPAZ-1 study Aiwu R. He 1 | Mohamed Bouattour2 | Vineet G. Gupta3 | Ludovic Evesque4 | David B. Zhen5 | Joon Oh Park6 | Aumkhae Sookprasert7 | Alejandro Salvatierra8 | Gina Vaccaro9 | Sang Cheul Oh10 | Sandra Anabel Ostoich11 | Taroh Satoh 12 | Aleksandra Kuzko13 | Magdalena Żotkiewicz13 | Nana Rokutanda 14 | Do-Youn Oh15 1Herbert Irving Comprehensive Cancer Center, New York-Presbyterian/Columbia University Irving Medical Center, New York, New York, USA 2Liver Cancer Unit, INSERM, U1149, AP-HP Hôpital Beaujon, Paris, France 3Medical Oncology, Fortis Hospital, Shalimar Bagh, New Delhi, India 4Department of Medical Oncology, Centre Antoine Lacassagne, Nice, France 5Division of Hematology Oncology, Department of Internal Medicine, University of Washington School of Medicine/Fred Hutch Cancer Center, Seattle, Washington, USA 6Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea 7Medical Oncology Unit, Department of Medicine, Faculty of Medicine, Khon Kaen University, Khon Kaen, Thailand 8Fundacion Ars Medica, San Salvador de Jujuy, Jujuy, Argentina 9Tennessee Oncology, Nashville, Tennessee, USA 10Division of Hematology and Oncology, Department of Internal Medicine, College of Medicine, Korea University, Seoul, Republic of Korea 11Department of Oncology, Hospital Provincial del Centenario, Rosario, Argentina 12Department of Gastroenterological Surgery, Osaka University, Suita, Osaka, Japan 13Late Oncology Statistics, Oncology Biometrics, AstraZeneca, Warsaw, Poland 14Oncology R&D, Late-Stage Development, AstraZeneca, Gaithersburg, Maryland, USA 15Division of Medical Oncology, Department of Internal Medicine, Seoul National University Hospital and Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea Correspondence Aiwu R. He, Herbert Irving Comprehensive Cancer Center, New York-Presbyterian/Columbia University Irving Medical Center, New York, New York, USA. Email: [arh2216@cumc.columbia.edu](arh2216@cumc.columbia.edu) Do-Youn Oh, Division of Medical Oncology, Department of Internal Medicine, Seoul National University Hospital and Cancer Research Institute, Seoul National University College of Medicine, Seoul, Republic of Korea. Email: [ohdoyoun@snu.ac.kr](ohdoyoun@snu.ac.kr) Abbreviations: aBTC, advanced biliary tract cancer; AIC, Akaike information criterion; CA 19-9, cancer antigen 19-9; CCS, complete case set; CEA, carcinoembryonic antigen; DCO, data cut-off; eCCA, extrahepatic cholangiocarcinoma; ECOG PS, Eastern Cooperative Oncology Group performance status; FAS, full analysis set; GemCis, gemcitabine and cisplatin; iCCA, intrahepatic cholangiocarcinoma; LDH, lactate dehydrogenase; NLR, neutrophil-to-lymphocyte ratio; OS, overall survival; PD-L1, programmed cell death ligand-1; SoC, standard of care. Keywords: cisplatin, durvalumab, gemcitabine, immunotherapy, phase 3 Aiwu R. He and Do-Youn Oh are joint lead/corresponding authors. Dr He will act as the primary corresponding author. Supplemental Digital Content is available for this article. Direct URL citations are provided in the HTML and PDF versions of this article on the journal’s website, www.hepcommjournal.com. -- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- ----- - This is an open access article distributed under the terms of the Creative Commons Attribution-Non Commercial-No Derivatives License 4.0 (CCBY-NC-ND), where it is permissible to download and share the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal. Copyright © 2025 The Author(s) . Published by Wolters Kluwer Health, Inc. on behalf of the Ameri

FAQs

What study design was used to assess survival predictors?

The analysis was a post-hoc evaluation within the phase 3 TOPAZ-1 trial, comparing durvalumab plus GemCis versus placebo plus GemCis in advanced biliary tract cancer.

Which outcomes and time points were used in the analysis?

Overall survival (OS) was the key endpoint, with the analysis anchored to a data cut-off dated February 25, 2022.

What types of factors were investigated for prognostic or predictive value?

The study compiled patient-related and disease-related variables, including pre-specified predictors from TOPAZ-1, and evaluated their ability to predict OS benefit under immune checkpoint inhibitor treatment.

Sep 29, 2026

Upload by mieayamfan