title: Second-line systemic treatment for advanced cholangiocarcinoma.
abstract: 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.
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Jane E
authors.initials: JE
authors.lastname: Rogers
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Lindsey
authors.initials: L
authors.lastname: Law
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Van D
authors.initials: VD
authors.lastname: Nguyen
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Wei
authors.initials: W
authors.lastname: Qiao
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Milind M
authors.initials: MM
authors.lastname: Javle
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Ahmed
authors.initials: A
authors.lastname: Kaseb
authors.affiliation: 1 Pharmacy Clinical Programs, 2 Department of Gastrointestinal Medical Oncology, 3 Department of Biostatistics, UT MD Anderson Cancer Center, Houston, TX 77030, USA.
authors.forename: Rachna T
authors.initials: RT
authors.lastname: Shroff
keywords: Cholangiocarcinoma; biotherapy; chemotherapy; metastatic; second-line