Bian JL, Wang MM, Tong EJ, Sun J, Li M, Miao ZB, Li YL, Zhu BH, Xu JJ. Benefit of everolimus in treatment of an intrahepatic cholangiocarcinoma patient with a PIK3CA mutation. World J Gastroenterol 2017; 23(23): 4311-4316 [PMID: 28694672 DOI: 10.3748/wjg.v23.i23.4311]
Corresponding Author of This Article
Dr. Ji-Lai Bian, Department of Medical Oncology, The Fifth People’s Hospital of Dalian, Dalian 116021, Liaoning Province, China. bianjilai123@126.com
Research Domain of This Article
Oncology
Article-Type of This Article
Case Report
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Ji-Lai Bian, Mei-Mei Wang, En-Juan Tong, Jing Sun, Ming Li, Department of Medical Oncology, The Fifth People’s Hospital of Dalian, Dalian 116021, Liaoning Province, China
Jia-Jia Xu, Institute of Precision Medicine, 3D Medicines Inc, Shanghai 201114, China
Author contributions: Bian JL and Wang MM followed the patient; Miao ZB, Li YL, Zhu BH and Xu JJ provided genetic analysis for the variants tested in the patient; Tong EJ, Sun J and Li M searched related articles; Bian JL and Li YL wrote the paper; all authors have read and approved the final manuscript.
Conflict-of-interest statement: There are no potential conflicts of interest relevant to this article.
Open-Access: This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Correspondence to: Dr. Ji-Lai Bian, Department of Medical Oncology, The Fifth People’s Hospital of Dalian, Dalian 116021, Liaoning Province, China. bianjilai123@126.com
Received: February 26, 2017 Peer-review started: February 27, 2017 First decision: April 10, 2017 Revised: April 22, 2017 Accepted: May 19, 2017 Article in press: May 19, 2017 Published online: June 21, 2017
Abstract
Intrahepatic cholangiocarcinoma (ICC) is a relatively rare form of liver cancer with a poor prognosis. The therapeutic options for patients with advanced ICC are limited and usually ineffective. There is currently no approved targeted therapy for ICC, although accumulating evidence supports inhibition of the PI3K/Akt/mTOR signaling pathway as a promising therapeutic strategy in the treatment of ICC. Here, we report a patient with stage IV ICC harboring a PIK3CA mutation who responded well to the mTOR inhibitor everolimus. Computed tomography and magnetic resonance imaging demonstrated shrinkage of the tumor and maintenance of a partial response for 6.5 mo after everolimus treatment as the best response. To the best of our knowledge, this is the first clinical case report in the literature of clinical benefit from everolimus treatment in an ICC patient with PIK3CA mutation.
Core tip: We report a stage IV intrahepatic cholangiocarcinoma (ICC) patient harboring a PIK3CA mutation who responded well to the mTOR inhibitor everolimus. Computed tomography and magnetic resonance imaging demonstrated shrinkage of the tumor and the maintenance of partial response for 6.5 mo after everolimus treatment as the best response. To the best of our knowledge, this is the first clinical case report in the literature of an ICC patient with PIK3CA mutation deriving benefit from everolimus treatment.
