Peer-Reviewed Publication
Nat Med2026September 22, 2026Journal Article

Trastuzumab plus nivolumab with gemcitabine and cisplatin as first-line therapy for HER2-positive unresectable biliary tract cancer: a phase 1b/2 trial.

Choong-Kun Lee1,2, Taek Chung3, Il Hwan Kim4, Hong Jae Chon5, Jin Won Kim6, Se Jun Park7, Woo Kyun Bae8, Junghun Kang9, Changhoon Yoo10, Dong-Hoe Koo11, Sora Kang12, Minsuk Kwon13, Ju Won Kim14, Beodeul Kang5, Sejung Park15, Woochan Hwang16, Chang Ho Ahn16, Chang Gon Kim17, Hye Jin Choi17
1Division of Medical Oncology, Yonsei Cancer Center, Yonsei University College of Medicine, Seoul, Republic of Korea. cklee512@yuhs.ac.
2Song-Dang Institute for Cancer Research, Yonsei University College of Medicine, Seoul, Republic of Korea. cklee512@yuhs.ac.
3Department of Pathology, Yonsei University College of Medicine, Seoul, Republic of Korea.
4Division of Oncology, Department of Internal Medicine, Inje University College of Medicine, Haeundae Paik Hospital, Busan, Republic of Korea.
5Medical Oncology, Department of Internal Medicine, CHA Bundang Medical Center, CHA University, Seongnam, Republic of Korea.
6Department of Internal Medicine, Seoul National University Bundang Hospital, Seoul National University College of Medicine, Seongnam, Republic of Korea.
7Division of Medical Oncology, Department of Internal Medicine, The Catholic University of Korea, Seoul St. Mary's Hospital, Seoul, Republic of Korea.
8Department of Hematology-Oncology, Chonnam National University Medical School and Hwasun Hospital, Hwasun, Republic of Korea.
9Department of Internal Medicine, Gyeongsang National University, Jinju, Republic of Korea.
10Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
11Division of Hematology/Oncology, Department of Internal Medicine, Kangbuk Samsung Hospital, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
12Division of Hemato-oncology, Department of Internal Medicine, Chungnam National University Hospital, Daejeon, Republic of Korea.
13Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
14Division of Hemato-oncology, Department of Internal Medicine, Korea University Anam Hospital, Korea University College of Medicine, Seoul, Republic of Korea.
15Song-Dang Institute for Cancer Research, Yonsei University College of Medicine, Seoul, Republic of Korea.
16Lunit, Seoul, Republic of Korea.
17Division of Medical Oncology, Yonsei Cancer Center, Yonsei University College of Medicine, Seoul, Republic of Korea.

Abstract

Biliary tract cancers (BTCs) are associated with poor outcomes despite recent incorporation of immune checkpoint inhibitors into first-line chemotherapy. Human epidermal growth factor receptor 2 (HER2) overexpression or amplification defines a therapeutically targetable subset of BTC but its integration into first-line chemoimmunotherapy has not been prospectively evaluated. This multi-institution…

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