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Cholangiocarcinoma News

Retrospective Analysis of Post Second-Line Chemotherapy Outcomes with Advanced or Metastatic CCA and FGFR2 Fusions

Web Exclusives — May 28, 2020

Cholangiocarcinoma (CCA) is the most common biliary tract malignancy, with an estimated incidence of 8000 to 10,000 patients annually in the United States. Chemotherapy is the most common second-line treatment with response rates of <10% and median progression-free survival (PFS) of approximately 3 to 4 months, including FOLFOX in the ABC-06 trial.1 Fibroblast growth factor receptor 2 (FGFR2) fusions occur in 13% to 17% of CCA, and multiple targeted agents are in development for patients with FGFR2 fusions. To date, the outcome of patients with CCA and FGFR2 fusions receiving standard second-line chemotherapy is unknown.

Patients with advanced CCA and FGFR2 fusions or translocations after prior treatment with gemcitabine-based chemotherapy were enrolled in a single-arm phase 2 study (NCT02150967) and received the FGFR1-3 selective tyrosine kinase inhibitor infigratinib (previously BGJ398) 125 mg orally once daily on days 1 to 21, with cycles repeated every 28 days until unacceptable toxicity, disease progression, investigator discretion, or withdrawal of consent.2 A retrospective analysis of a subset of patients who received infigratinib as third- or later-line treatment was performed. Investigator-assessed PFS and best overall response (per RECIST 1.1) following second-line chemotherapy (pre-infigratinib) and third-line or later-line infigratinib were calculated.

A total of 71 patients with FGFR2 fusions enrolled at the time of analysis, and 37 (52%) patients were included in the retrospective analysis. Median PFS with standard second-line chemotherapy was 4.63 months compared with 6.8 months for third- and later-line infigratinib. Best overall response for second-line chemotherapy was 5.4% compared with 21.6% for third- and later-line infigratinib. The authors concluded that in patients with CCA and FGFR2 fusions, median PFS and overall response rate was better for third- and later-line treatment with infigratinib versus standard second-line chemotherapy. number NCT02150967.


  1. Lamarca A, et al. Ann Oncol. 2014;25:2328-2338.
  2. Javle M, et al. ASCO 2020. Abstract 4591.

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