Post-hoc Analysis of the ABC-01, -02, and -03 Trials in Patients With Advanced eCCA

This post-hoc analysis of the ABC-01, -02, and -03 clinical trials provides reference survival data for patients with advanced extrahepatic cholangiocarcinoma treated with first-line gemcitabine/cisplatin chemotherapy.

Earlier data showed that patients with intrahepatic cholangiocarcinoma (iCCA) have a better prognosis compared with other biliary tract cancers1; however, information is limited regarding outcomes for patients with extrahepatic cholangiocarcinoma (eCCA). At the 2023 ESMO Congress in Madrid, Spain, Angela Lamarca, MD, PhD, MSC, presented findings from a post-hoc analysis of reference survival data for patients with advanced eCCA treated with first-line gemcitabine/cisplatin (GemCis) chemotherapy within the prospective, randomized ABC-01, -02, and -03 studies.2

A total of 534 patients were enrolled in the ABC-01, -02, and -03 studies, and 179 (56.29%) had eCCA (107 distal CCA [dCCA], 72 hilar CCA [hCCA]).2 Of these patients, 117 were treated with GemCis and were eligible for analysis.2 Most patients (82 [70.1%]) had metastatic disease at the time of recruitment.2 Objective radiological response was achieved in 28 (23.93%) patients with eCCA: 19 (27.94%) with dCCA, and 9 (18.37%) with hCCA.2 The median progression-free survival (PFS) for patients with eCCA, dCCA, and hCCA was 8.37 months (95% confidence interval [CI], 7.36-9.33), 8.28 months (95% CI, 6.31-9.39), and 8.37 months (95% CI, 6.53-10.61), respectively; median overall survival (OS) was 12.76 months (95% CI, 10.44-16.12), 14.25 months (95% CI, 8.80-17.44), and 12.18 months (95% CI, 8.31-16.12), respectively.2 Patients with eCCA with locally advanced disease had a longer median PFS (13.1 months; 95% CI, 8.8-16.6) and OS (17.4 months; 95% CI, 14.3-21.1) compared with patients with advanced eCCA (PFS, 8.37 months [95% CI, 7.36-9.33]; OS, 12.76 months [95% CI, 10.44-16.12]), dCCA (PFS, 8.28 [95% CI, 6.31-9.39]; OS, 14.25 [95% CI, 8.80-17.44]), and hCCA (PFS, 8.37 [95% CI, 6.53-10.61]; OS, 12.18 [95% CI, 8.31-16.12]).2 Multivariable survival analysis for PFS confirmed worse PFS for patients with eCCA who had a performance status of 1/2 versus 0 (hazard ratio [HR], 13.24; 95% CI, 2.09-83.49) and high baseline CA 125 (HR, 1.0001; 95% CI, 1.0004-1.01; P=.002) when adjusted for other prognostic factors identified in the univariate analysis (stage, Ca 19-9).2 These remained significant when adjusted for site of eCCA.2

Lamarca and colleagues determined in this post-hoc analysis that patient outcomes were similar regardless of location of eCCA, but outcomes were worse than those reported historically for iCCA, specifically for OS.2 Survival in eCCA was strongly correlated with performance status and baseline tumor markers (CA 125).2 Future studies should focus on outcomes with first-line GemCis combined with immunotherapy.2


  1. Lamarca A, Ross P, Wasan HS, et al. Advanced intrahepatic cholangiocarcinoma: post hoc analysis of the ABC-01, -02, and -03 clinical trials. J Natl Cancer Inst. 2020;112(2):200-210.
  2. Lamarca A, Ross P, Wasan HS, et al. Advanced extrahepatic cholangiocarcinoma: post-hoc analysis of the ABC-01, -02 and -03 clinical trials. Presented at: ESMO Congress 2023, October 20-24, 2023; Madrid, Spain.

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