Feasibility and benefits of second-line chemotherapy in advanced biliary tract cancer: a large retrospective study

Introduction: First-line chemotherapy (CT1) is effective in advanced biliary tract cancer (ABTC). The benefits of second-line chemotherapy (CT2) are unclear.

Methods: We retrospectively studied all patients starting at least one line of chemotherapy for ABTC at our institution between 1991 and 2011. We analysed patient and chemotherapy characteristics in order to: (1) characterise patients eligible for CT2; (2) evaluate the efficacy of CT2.

Results: Three hundred and seventy-eight received CT1 and 96 (25%) patients received CT2. Primary tumour location was the gallbladder (29%), intraphepatic (20%), perihilar (16%), distal common bile duct (19%) and ampulla of Vater (14%). Ninety percent had a baseline performance status (PS) of 0-1 prior to CT1. Females (p=0.03), ages ≤ 60 years (p=0.001) and patients with progression free survival (PFS) ≥ 6 months following CT1 (p=0.01) were more likely to be offered CT2. Objective response rates and stable disease with CT2 were 9% and 34%, respectively. Median PFS and median overall survival (OS) from the beginning of CT2 were 2.8 and 7.5 months, respectively. Prognostic factors impacting PFS with CT2 were the regimen type (doublet versus monotherapy, p=0.001) and PS<2 (p<0.0001).

Conclusions: Among patients with ABTC, 25% received CT2, typically younger patients and those with longer PFS following CT1. Disease control occurred in 43% of patients, and more often with a doublet than a single agent. However, clearly more effective therapies must be found.