Published in:
01-10-2020 | Hepatocellular Carcinoma | Case Report
Complete response to pembrolizumab in advanced hepatocellular carcinoma with microsatellite instability
Authors:
Yuwa Ando, Masami Yamauchi, Yosuke Suehiro, Kenji Yamaoka, Yumi Kosaka, Yasutomo Fuji, Shinsuke Uchikawa, Kenichiro Kodama, Kei Morio, Hatsue Fujino, Takashi Nakahara, Atsushi Ono, Eisuke Murakami, Tomokazu Kawaoka, Shoichi Takahashi, Masataka Tsuge, Akira Hiramatsu, Michio Imamura, Kazuaki Chayama, Hiroshi Aikata
Published in:
Clinical Journal of Gastroenterology
|
Issue 5/2020
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Abstract
Hepatocellular carcinoma (HCC) has limited systemic treatment options and a poor prognosis. The immune checkpoint inhibitor pembrolizumab was recently approved for the treatment of solid tumors with microsatellite instability (MSI). However, its clinical utility for the management of HCC remains to be clarified. Here, we present a case of unresectable HCC with MSI that showed an impressive response to pembrolizumab treatment. A 64-year-old man with chronic HCV infection was diagnosed with a large HCC. His severe liver dysfunction and poor performance status prevented any treatment option other than sorafenib. However, sorafenib failed after a few days due to the rapid progression of the tumor. Based on the finding of MSI in a biopsy specimen, immunotherapy using pembrolizumab was initiated. A dramatic improvement in his general condition and a reduction in tumor size were observed after the initiation of pembrolizumab treatment. Among a cohort of 50 consecutive patients with advanced HCC who were refractory to standard systemic therapy, MSI was found only in the present case. Immune checkpoint blockade therapy induced prominent anti-tumor effects in HCC with MSI. Screening for defects in DNA mismatch repair function may be warranted in HCC patients despite the low frequency of MSI.