Complete response to pembrolizumab in advanced hepatocellular carcinoma with microsatellite instability

Ando Yuwa

Yamauchi Masami

Suehiro Yosuke

Yamaoka Kenji

Kosaka Yumi

Fuji Yasutomo

Uchikawa Shinsuke

Kodama Kenichiro

Morio Kei

Fujino Hatsue

Nakahara Takashi

Ono Atsushi

Murakami Eisuke

Kawaoka Tomokazu

Takahashi Shoichi

Tsuge Masataka

Hiramatsu Akira

Imamura Michio

Chayama Kazuaki

Aikata Hiroshi

Clinical Journal of Gastroenterology

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.

Hepatocellular carcinoma

Mismatch repair deficiency

MSI-high

Immune checkpoint blockade

PD-L1

eng

Springer

This is a post-peer-review, pre-copyedit version of an article published in Clinical Journal of Gastroenterology. The final authenticated version is available online at: https://doi.org/10.1007/s12328-020-01099-3

This is not the published version. Please cite only the published version. この論文は出版社版ではありません。引用の際には出版社版をご確認、ご利用ください。

[ISSN] 1865-7257

[ISSN] 1865-7265

[DOI] 10.1007/s12328-020-01099-3

[PMID] 32020539

[DOI] https://doi.org/10.1007/s12328-020-01099-3