Cancer Res Treat.  2020 Apr;52(2):594-603. 10.4143/crt.2019.493.

Efficacy and Safety of Pembrolizumab in Patients with RefractoryAdvanced Biliary Tract Cancer: Tumor Proportion Score as a PotentialBiomarker for Response

Affiliations
  • 1Departments of 1Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
  • 2Departments of Pathology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
  • 3Departments of Gastroenterology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
  • 4Departments of Surgery, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
  • 5Departments of Radiation Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea

Abstract

Purpose
The current standard chemotherapy for advanced biliary tract cancer (BTC) has limited benefit, and novel therapies need to be investigated.
Materials and Methods
In this prospective cohort study, programmed death ligand-1 (PD-L1)–positive BTC patients who progressed on first-line gemcitabine plus cisplatin were enrolled. Pembrolizumab 200 mg was administered intravenously every 3 weeks.
Results
Between May 2018 and February 2019, 40 patients were enrolled. Pembrolizumab was given as second-line (47.5%) or  third-line therapy (52.5%). The objective response rate was 10% and 12.5% by Response Evaluation Criteria in Solid Tumor (RECIST) v1.1 and immune-modified RECIST (imRECIST) and median duration of response was 6.3 months. Among patients with progressive disease as best response, one patient (1/20, 5.0%) achieved complete response subsequently. The median progression-free survival (PFS) and overall survival (OS) were 1.5 months (95% confidence interval [CI], 0.0 to 3.0) and 4.3 months (95% CI, 3.5 to 5.1), respectively, and objective response per imRECIST was significantly associated with PFS (p < 0.001) and OS (p=0.001). Tumor proportion score  50% was significantly associated with higher response rates including the response after pseudoprogression (vs. < 50%; 37.5% vs. 6.5%; p=0.049).
Conclusion
Pembrolizumab showed modest anti-tumor activity in heavily pretreated PD-L1–positive BTC patients. In patients who showed objective response, durable response could be achieved.

Keyword

Biliary tract neoplasms; Cholangiocarcinoma; Pembrolizumab; Immunotherapy
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