Treatment Options for Metastatic Cholangiocarcinoma


Oncologist Dr. Milind Javel provides an overview of first- and subsequent-line treatments for patients with cholangiocarcinoma.


Milind Javle, MD: For patients who do not have actionable mutations, the first-line chemotherapy is still based on the ABC-02 trial, which is gemcitabine and cisplatin. While this is the standard of care, it’s relatively suboptimal. The response rate is in the 20% to 30% range, progression-free survival of 6 to 8 months, and overall survival of 11 months. Therefore, although it is a standard, this is a standard that we hope we can change with therapy. There have been many promising trials, including one recently conducted in the United States with gemcitabine, cisplatin, and Abraxane [paclitaxel] that we hope will change this paradigm.

In the second-line setting, the ABC-06 trial investigated the use of FOLFOX [folinic acid, fluorouracil, oxaliplatin], which is the standard chemotherapy used in colon cancer, and also used in cholangiocarcinoma. Again, it is very modest in terms of how effective it is, with a 5% response rate, progression-free survival of 4 months, and overall survival of only 6 months. Clearly, this is an area that needs further investigation, and cholangiocarcinoma represents an area of great unmet need.

Going back to our patient, she received appropriate first-line treatment, gemcitabine and cisplatin. She had a good ECOG [The Eastern Cooperative Oncology Group] performance status of 1. She handled the treatment, however not surprisingly, she experienced disease progression and the tumor stopped responding after a few months of therapy.

Transcript edited for clarity.


Case: A 61-Year-Old Woman with Metastatic Cholangiocarcinoma

May 2019

Initial presentation

  • A 61-year-old woman presents with jaundice and changes in stool and urine color.

Clinical workup

  • Enlarged liver is palpable on physical examination
  • Blood work reveals serum levels of CA 19-9 (1400 U/ml), bilirubin 2 mg/dL, ALT 550 U/L, AST 120U/L
  • Patient undergoes CT imaging and is found to have multiple liver masses, consistent with metastatic disease or intrahepatic cholangiocarcinoma (iCCA)
  • Histopathological examination identifies adenocarcinoma, CK7+, CK20-, HepPAR-, TTF- consistent with cholangiocarcinoma or upper GI Primary
  • Patient is diabetic and somewhat non-compliant with her diabetes medication
  • Her ECOG PS is 1

July 2019


  • Patient is treated with chemotherapy (gemcitabine + cisplatin) for 24 weeks.
  • Patient is monitored for disease progression every 2-3 months by CT imaging.

July 2020

  • Patient does well for 1 year after initiation of treatment but now has elevated CA 19-9 levels.
  • MRI scans show several liver and bone lesions but no signs of brain metastases.
  • Lab results are normal (absolute neutrophil count 4,000/mm3, platelets 150,000/ml, hemoglobin 12.1 g/dL
  • Patient undergoes NGS testing (Foundation Medicine, Inc.) and is found to have FGFR2-BICC1 gene fusion
  • Patient meets eligibility criteria for infigratinib phase 2 study and is enrolled in the trial and being treated with infigratinib
  • Patient does not show any signs of disease progression on MRI scans for six months, suggestive of stable disease and CA19-9 levels stay within normal limits.
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