Abstract
BackgroundCholangiocarcinoma, or bile duct cancer, is a gastrointestinal cancer with limited therapeutic options and a poor outcome. Studies have revealed that some major driver genes are associated with cholangiocarcinoma, but no targeted therapies have been approved. Immune checkpoint inhibitors, which are represented by inhibitors of programmed cell death 1 (PD-1)/programmed death-ligand 1 (PD-L1), have emerged as a potential therapy for multiple types of solid cancers.Case presentationA 53-year-old female presented with postoperative recurrence of PD-L1-positive intrahepatic cholangiocarcinoma with a high tumour mutational burden. This patient exhibited a marked response to the combination of anti-PD-1 immunotherapy and chemotherapy.ConclusionsAs far as we know, this is the first case report on the success of the combination of immunotherapy and chemotherapy for advanced cholangiocarcinoma with PD-L1 positivity and a high tumour mutational burden.
Highlights
Cholangiocarcinoma, or bile duct cancer, is a gastrointestinal cancer with limited therapeutic options and a poor outcome
No targeted agents have been approved for advanced cholangiocarcinoma, and only limited chemotherapeutic options are recommended by the NCCN for patients who are not candidates for surgery [3]
Compared with the overall survival of patients who are treated with small molecule targeted drugs, conventional chemotherapy drugs and programmed cell death 1 (PD-1)/programmed death-ligand 1 (PD-L1) inhibitors can lead to significant improvements in OS and progression-free survival (PFS) in some cancers [9,10,11,12]
Summary
According to the 2010 WHO guidelines, cholangiocarcinoma is stratified anatomically into intrahepatic cholangiocarcinoma (iCCA) and extrahepatic cholangiocarcinoma (ECCA) [1], both of which have a poor prognosis. The FDA has approved pembrolizumab as a first-line therapy for advanced non-small cell lung cancers (NSCLCs) that have high PD-L1 expression and no driver mutations (tumour proportion score (TPS) ≥ 50%) [16]. We reported a patient who was diagnosed with an intrahepatic cholangiocarcinoma with a high tumour mutational burden (TMB) and high expression of PD-L1 (TPS reached 80%). This patient achieved complete remission (CR) and experienced fewer side effects after treatment with pembrolizumab and chemotherapy. An immunohistochemical analysis revealed the following: AFP(−), CA-125(−), CD10(−), CD34(blood vessel+), CKpan(+), CK7(−), CK19(+), CK20(−), HCV(−), HBcAg(−), HBsAg(liver+) Ki-67(50%+), P53(90%+), TTF-1(−), vimentin(+), WT1(−), and Gly3(−) These results suggested a diagnosis of stage IIIB iCCA (pT2N1M0). From July 2017 to May 2018, when was the patient was last followed-up, the cancer was in complete remission (CR) (Fig. 2e and j), and the toxicity associated with immunotherapy was not obvious
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