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Molecular profiling and treatment pattern differences between intrahepatic and extrahepatic cholangiocarcinoma
Kristen Spencer1, Leontios Pappas2, Islam Baiev2
1Department of Medicine, New York University Langone Health Perlmutter Cancer Center, New York University School of Medicine, New York, NY, USA.
Journal of the National Cancer Institute
|April 11, 2023
Summary
Extrahepatic cholangiocarcinoma (ECC) patients receive less molecular profiling and targeted therapy than intrahepatic cholangiocarcinoma (ICC) patients. Both subtypes have poor prognoses, highlighting the need for novel therapies and clinical trial access.
Area of Science:
- Oncology
- Gastroenterology
- Medical Research
Background:
- Intrahepatic cholangiocarcinoma (ICC) and extrahepatic cholangiocarcinoma (ECC) exhibit distinct treatment patterns.
- Limited comparative studies exist on molecular profiling and treatment disparities between ICC and ECC.
Purpose of the Study:
- To compare molecular profiling rates and treatment strategies for ICC and ECC.
- To investigate the utilization of adjuvant, liver-directed, targeted, and investigational therapies in ICC and ECC.
Main Methods:
- Multicenter retrospective study involving 847 patients with ICC or ECC.
- Data collected on risk factors, pathology, treatments, and survival outcomes.
- Comparative statistical analyses were performed.
Main Results:
- ECC patients were more likely to have early-stage disease, undergo resection, and receive adjuvant chemoradiation.
- ICC patients had higher rates of molecular profiling, liver-directed therapy, targeted therapy, and clinical trial enrollment.
- Advanced ECC patients had shorter median overall survival compared to advanced ICC patients.
Conclusions:
- Advanced ECC patients demonstrate low molecular profiling rates, potentially due to insufficient tissue, and limited use of targeted therapies and clinical trials.
- Despite higher rates in ICC, both cholangiocarcinoma subtypes have poor prognoses.
- There is a critical need for improved targeted therapies and expanded access to clinical trials for cholangiocarcinoma.

