Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer

Abstract Background and Aim The success rate of microsatellite instability (MSI) examination in biliary tract cancer (BTC) and the treatment outcomes of pembrolizumab in patients with MSI‐high (MSI‐H) BTC have not been fully investigated. We examined the success rate of MSI examination and the rate...

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Main Authors: Yugo Kai, Kenji Ikezawa, Ryoji Takada, Kazuma Daiku, Shingo Maeda, Yutaro Abe, Takuo Yamai, Nobuyasu Fukutake, Tasuku Nakabori, Hiroyuki Uehara, Shigenori Nagata, Hiroshi Wada, Kazuyoshi Ohkawa
Format: Article
Language:English
Published: Wiley 2021-06-01
Series:JGH Open
Subjects:
Online Access:https://doi.org/10.1002/jgh3.12576
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author Yugo Kai
Kenji Ikezawa
Ryoji Takada
Kazuma Daiku
Shingo Maeda
Yutaro Abe
Takuo Yamai
Nobuyasu Fukutake
Tasuku Nakabori
Hiroyuki Uehara
Shigenori Nagata
Hiroshi Wada
Kazuyoshi Ohkawa
author_facet Yugo Kai
Kenji Ikezawa
Ryoji Takada
Kazuma Daiku
Shingo Maeda
Yutaro Abe
Takuo Yamai
Nobuyasu Fukutake
Tasuku Nakabori
Hiroyuki Uehara
Shigenori Nagata
Hiroshi Wada
Kazuyoshi Ohkawa
author_sort Yugo Kai
collection DOAJ
description Abstract Background and Aim The success rate of microsatellite instability (MSI) examination in biliary tract cancer (BTC) and the treatment outcomes of pembrolizumab in patients with MSI‐high (MSI‐H) BTC have not been fully investigated. We examined the success rate of MSI examination and the rate of MSI‐H status in patients with BTC as well as the treatment outcomes of patients with MSI‐H status who underwent pembrolizumab treatment. Methods We retrospectively reviewed 60 consecutive patients with unresectable or postoperative recurrent BTC who underwent MSI examination in a Japanese cancer referral center between January 2019 and September 2020. Results The study included 24 intrahepatic cholangiocarcinomas, 12 hilar cholangiocarcinomas, 4 distal cholangiocarcinomas, 16 gallbladder carcinomas, and 4 ampullary carcinomas. The methods of cancer tissue sampling were percutaneous liver tumor biopsy in 26 cases, surgery in 15 cases, endoscopic ultrasound fine‐needle aspiration in 12 cases, transpapillary bile duct biopsy in 5 cases, and others in 2 cases. The success rate of MSI examination was 98.3% (59 of 60). MSI examination failed in only one case using a surgical specimen due to time‐dependent degradation of DNA. The frequency of MSI‐H BTC was 3.3% (2 of 60 cases). One patient with MSI‐H intrahepatic cholangiocarcinoma achieved a complete response with pembrolizumab treatment. Conclusions MSI examinations in BTC were successful in almost all cases, regardless of tissue sampling methods. We experienced a case in which pembrolizumab resulted in a complete response to MSI‐H BTC. Since pembrolizumab for MSI‐H BTC could prolong survival time, MSI examination should be performed proactively to increase treatment options.
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spelling doaj.art-0c08d7c21cff40d8b7e1aa6b24ce1d912022-12-21T22:16:08ZengWileyJGH Open2397-90702021-06-015671271610.1002/jgh3.12576Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancerYugo Kai0Kenji Ikezawa1Ryoji Takada2Kazuma Daiku3Shingo Maeda4Yutaro Abe5Takuo Yamai6Nobuyasu Fukutake7Tasuku Nakabori8Hiroyuki Uehara9Shigenori Nagata10Hiroshi Wada11Kazuyoshi Ohkawa12Department of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanDepartment of Diagnostic Pathology and Cytology Osaka International Cancer Institute Osaka JapanDepartment of Surgery Osaka International Cancer Institute Osaka JapanDepartment of Hepatobiliary and Pancreatic Oncology Osaka International Cancer Institute Osaka JapanAbstract Background and Aim The success rate of microsatellite instability (MSI) examination in biliary tract cancer (BTC) and the treatment outcomes of pembrolizumab in patients with MSI‐high (MSI‐H) BTC have not been fully investigated. We examined the success rate of MSI examination and the rate of MSI‐H status in patients with BTC as well as the treatment outcomes of patients with MSI‐H status who underwent pembrolizumab treatment. Methods We retrospectively reviewed 60 consecutive patients with unresectable or postoperative recurrent BTC who underwent MSI examination in a Japanese cancer referral center between January 2019 and September 2020. Results The study included 24 intrahepatic cholangiocarcinomas, 12 hilar cholangiocarcinomas, 4 distal cholangiocarcinomas, 16 gallbladder carcinomas, and 4 ampullary carcinomas. The methods of cancer tissue sampling were percutaneous liver tumor biopsy in 26 cases, surgery in 15 cases, endoscopic ultrasound fine‐needle aspiration in 12 cases, transpapillary bile duct biopsy in 5 cases, and others in 2 cases. The success rate of MSI examination was 98.3% (59 of 60). MSI examination failed in only one case using a surgical specimen due to time‐dependent degradation of DNA. The frequency of MSI‐H BTC was 3.3% (2 of 60 cases). One patient with MSI‐H intrahepatic cholangiocarcinoma achieved a complete response with pembrolizumab treatment. Conclusions MSI examinations in BTC were successful in almost all cases, regardless of tissue sampling methods. We experienced a case in which pembrolizumab resulted in a complete response to MSI‐H BTC. Since pembrolizumab for MSI‐H BTC could prolong survival time, MSI examination should be performed proactively to increase treatment options.https://doi.org/10.1002/jgh3.12576biliary tract neoplasmscholangiocarcinomaendoscopic ultrasound‐guided fine‐needle aspirationmicrosatellite instabilitypembrolizumab
spellingShingle Yugo Kai
Kenji Ikezawa
Ryoji Takada
Kazuma Daiku
Shingo Maeda
Yutaro Abe
Takuo Yamai
Nobuyasu Fukutake
Tasuku Nakabori
Hiroyuki Uehara
Shigenori Nagata
Hiroshi Wada
Kazuyoshi Ohkawa
Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
JGH Open
biliary tract neoplasms
cholangiocarcinoma
endoscopic ultrasound‐guided fine‐needle aspiration
microsatellite instability
pembrolizumab
title Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
title_full Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
title_fullStr Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
title_full_unstemmed Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
title_short Success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
title_sort success rate of microsatellite instability examination and complete response with pembrolizumab in biliary tract cancer
topic biliary tract neoplasms
cholangiocarcinoma
endoscopic ultrasound‐guided fine‐needle aspiration
microsatellite instability
pembrolizumab
url https://doi.org/10.1002/jgh3.12576
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