Prognostic factors for progression-free and overall survival in advanced biliary tract cancer

John Bridgewater, A. Lopes, H. Wasan, D. Malka, L. Jensen, T. Okusaka, J. Knox, D. Wagner, D. Cunningham, J. Shannon, D. Goldstein, M. Moehler, T. Bekaii-Saab, M. G. McNamara, J. W. Valle

Research output: Contribution to journalArticlepeer-review

82 Scopus citations


Background: Biliary tract cancer is an uncommon cancer with a poor outcome. We assembled data from the National Cancer Research Institute (UK) ABC-02 study and 10 international studies to determine prognostic outcome characteristics for patients with advanced disease. Methods: Multivariable analyses of the final dataset from the ABC-02 study were carried out. All variables were simultaneously included in a Cox proportional hazards model, and backward elimination was used to produce the final model (using a significance level of 10%), in which the selected variables were associated independently with outcome. This score was validated externally by receiver operating curve (ROC) analysis using the independent international dataset. Results: A total of 410 patients were included from the ABC-02 study and 753 from the international dataset. An overall survival (OS) and progression-free survival (PFS) Cox model was derived from the ABC-02 study. White blood cells, haemoglobin, disease status, bilirubin, neutrophils, gender, and performance status were considered prognostic for survival (all with P < 0.10). Patients with metastatic disease hazard ratio (HR) 1.56 [95% confidence interval (CI) 1.20-2.02] and Eastern Cooperative Oncology Group performance status (ECOG PS) 2 had worse survival [HR 2.24 (95% CI 1.53- 3.28)]. In a dataset restricted to patients who received cisplatin and gemcitabine with ECOG PS 0 and 1, only haemoglobin, disease status, bilirubin, and neutrophils were associated with PFS and OS. ROC analysis suggested the models generated from the ABC-02 study had a limited prognostic value [6-month PFS: area under the curve (AUC) 62% (95% CI 57-68); 1-year OS: AUC 64% (95% CI 58-69)]. Conclusion: These data propose a set of prognostic criteria for outcome in advanced biliary tract cancer derived from the ABC-02 study that are validated in an international dataset. Although these findings establish the benchmark for the prognostic evaluation of patients with ABC and confirm the value of longheld clinical observations, the ability of the model to correctly predict prognosis is limited and needs to be improved through identification of additional clinical and molecular markers.

Original languageEnglish
Pages (from-to)134-140
Number of pages7
JournalAnnals of Oncology
Issue number1
StatePublished - 1 Jan 2016
Externally publishedYes

Bibliographical note

Publisher Copyright:
© The Author 2015.


ABC-02 was an investigator-initiated study sponsored by UCL Clinical Trials Unit, funded by Cancer Research UK with gemcitabine provided by Lilly Oncology (unrestricted grant). BT22 was an Eli Lilly Japan-sponsored trial; the ABC and TACtic trials were Australasian Gastro Intestinal Trials Group trials funded in part by untied grants from Eli Lilly and Amgen, respectively, and the AGITG; additional data collection for OS and PFS was investigator- initiated and supported by the Ministry of Health, Labour, and Welfare, Health Labour Sciences Research Grant (with data transfer to UCL-CTU under a study-specific agreement for the sole purposes of this meta-analysis). JB is partly supported by the UCLH/UCL Biomedical Research Centre. AL is supported by a CRUK grant C444/A15953 to the UCL CRUK trials centre.

FundersFunder number
Lilly Oncology
Eli Lilly Japan
Cancer Research UKC444/A15953
University College London
Ministeriet Sundhed Forebyggelse


    • ABC-02
    • Advanced disease
    • Biliary tract cancer
    • Cisplatin and gemcitabine
    • Performance status
    • Prognostic model


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