Second-line systemic treatment for advanced cholangiocarcinoma

Jane E. Rogers, Lindsey Law, Van D. Nguyen, Wei Qiao, Milind M. Javle, Ahmed Kaseb, Rachna T. Shroff

Research output: Contribution to journalArticlepeer-review

36 Scopus citations


Background: Gemcitabine plus platinum (GEM-P) combination chemotherapy is standard treatment for first-line advanced cholangiocarcinoma (aCC). GEM-P first-line therapy reports a progression-free survival (PFS) of 8 months and overall survival (OS) of 11.7 months. Treatment in the second-line setting is less clear. Five-year survival for aCC remains dismal at 5-10%. The purpose of this study was to describe the outcomes with second-line systemic treatment at our institution. Methods: This study was a single institution retrospective chart review of aCC patients who initiated second-line systemic treatment during 1/1/2009 to 12/31/2012. The primary objective was to evaluate PFS with second-line systemic treatment. Secondary objectives were OS and disease control rate. Second-line systemic regimens were classified into four treatment groups: GEM-P, gemcitabine + fluoropyrimidine (GEM-FU), other FU combination (FU-combo), and others. Results: Fifty-six patients were included and the majority had intrahepatic aCC. A total of 80% received first-line gemcitabine-based therapy. Second-line therapy consisted of GEM-P (19.6%), GEM-FU (28.6%), FU-combo (37.5%), and others (14.3%). Median PFS was 2.7-month (95% CI, 2.3-3.8 months) with a median OS of 13.8 months (95% CI, 12-19.3 months) and a disease control rate of 50%. No significant difference in survival was identified between the four treatment groups. Conclusions: This study revealed a 2.7-month PFS, 50% disease control rate, and potential survival benefit with second-line treatment. Options for second-line systemic therapy include GEM-FU, FU-combo, GEM-P if not given in the first-line setting. Targeted therapy with erlotinib or bevacizumab could be considered in addition to chemotherapy.

Original languageEnglish (US)
Pages (from-to)408-413
Number of pages6
JournalJournal of Gastrointestinal Oncology
Issue number6
StatePublished - 2014
Externally publishedYes


  • Biotherapy
  • Chemotherapy
  • Cholangiocarcinoma
  • Metastatic
  • Second-line

ASJC Scopus subject areas

  • Oncology
  • Gastroenterology


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