Cytotoxic and noncytotoxic chemotherapy do not seem to be significantly different in terms of overall and progression-free survival, overall response rate, and disease control in patients with combined hepatocellular cholangiocarcinoma. This is according to a new study published in the journal ESMO Open.

The authors also reported that “immunotherapy may be effective in a subset of patients.”

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There is currently no consensus about the optimal systemic treatment regimen for patients with combined hepatocellular cholangiocarcinoma, the researchers noted.

In the present study, a team of researchers, led by Matthias Pinter, MD, PhD, from the Medical University of Vienna in Austria, described the clinical characteristics and outcomes of patients with combined hepatocellular cholangiocarcinoma. The investigators especially focused on patients who were receiving palliative systemic therapy, including immune checkpoint inhibitors.

The study team retrospectively analyzed 101 patients with histologically proven combined hepatocellular cholangiocarcinoma who received either cytotoxic chemotherapy, noncytotoxic chemotherapy, or palliative systemic therapy at 4 institutions between April 2003 and June 2022.

The results showed no significant difference between patients treated with cytotoxic chemotherapy and noncytotoxic chemotherapy in overall response rate, disease control rate, and median progression-free survival. However, there was a trend toward longer median overall survival in patients treated with cytotoxic chemotherapy at 15.5 months compared to those treated with noncytotoxic chemotherapy. 

In multivariable analysis, however, the type of first-line regimen was not associated with overall survival. 

The overall survival rate in patients treated with immune checkpoint inhibitors was 29%.

“Prospective studies are needed to identify optimal systemic treatment regimens in [combined hepatocellular cholangiocarcinoma],” the authors concluded.

Cholangiocarcinoma is a heterogeneous group of rare malignant tumors that affect the liver and originate from the biliary tree’s cells.


Pomej K, Balcar L, Shmanko K, et al. Clinical characteristics and outcome of patients with combined hepatocellular-cholangiocarcinoma-a European multicenter cohort. ESMO Open. Published online February 6, 2023. doi:10.1016/j.esmoop.2023