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KMID : 0939920200520020594
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2020 Volume.52 No. 2 p.594 ~ p.603
Efficacy and Safety of Pembrolizumab in Patients with Refractory Advanced Biliary Tract Cancer: Tumor Proportion Score as a Potential Biomarker for Response
Kang Jun-Ho

Jeong Jae-Ho
Hwang Hee-Sang
Lee Sang-Soo
Park Do-Hyun
Oh Dong-Wook
Song Tae-Jun
Kim Ki-Hun
Hwang Shin
Hwang Dae-Wook
Kim Song-Cheol
Park Jin-Hong
Hong Seung-Mo
Kim Kyu-Pyo
Ryoo Baek-Yeol
Yoo Chang-Hoon
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 ¡Ã 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 ¡Ã 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 cancer, Cholangiocarcinoma, Pembrolizumab, Immunotherapy
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