Combination therapy with programmed cell death protein-1 (PD-1) inhibitors appears to be effective as a first-line treatment for advanced cholangiocarcinoma (CCA) with manageable toxicity, according to a retrospective real-world study published in International Immunopharmacology. These findings support the use of these therapies for the initial treatment of the disease.

PD-1 inhibitors are already being used as the first-line treatment for advanced CCA in the real world. However, their safety and effectiveness have not been clearly established.

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In the present study, researchers from China assessed the effect of PD-1 inhibitor therapy on survival in 54 patients with advanced CCA. All patients received first-line combination therapy with PD-1 inhibitors.

The researchers reported that the objective response rate of the patients was 16.7% and the disease control rate of the treatment was 79.6%.

The median progression-free survival of the patients was 6.6 months, while their overall survival was 13.9 months. 

The majority (88.9%) of patients had at least 1 adverse event following treatment, with grade 3 or higher adverse events recorded in 37% of all patients. The most common grade 3 or higher adverse events were neutropenia, anemia, and thrombocytopenia. 

A little over half of the patients (51.9%) developed at least 1 immune-related adverse event. The most common were rash, hypothyroidism, and pruritus. 

The researchers also reported that patients with a carcinoembryonic antigen (CEA) concentration of 5 ng/mL or less before treatment had longer progression-free and overall survival times than those with a CEA concentration higher than 5 ng/mL.

“Combination therapy with PD-1 inhibitors has demonstrated promising efficacy and manageable adverse events,” the researchers concluded. 

CCA is a group of rare malignant tumors originating from cells of the biliary tree. Serum carbohydrate antigen 19-9 and CEA are tumor markers that can help diagnose CCA.


Ye Z, Zhang Y, Chen J, Wang X, Hong Y, Zhao Q. First-line PD-1 inhibitors combination therapy for patients with advanced cholangiocarcinoma: a retrospective real-world study. Int Immunopharmacol. Published online May 26, 2023. doi:10.1016/j.intimp.2023.110344