Durable response to vebreltinib in heavily pretreated MET-amplified intrahepatic cholangiocarcinoma: a case report.
5 affiliations
- 1Department of Oncology, Affiliated Hospital of Nanjing University of Chinese Medicine, Jiangsu Provincial Hospital of Chinese Medicine, Nanjing 210029, China.
- 2The First Clinical Medical College, Nanjing University of Chinese Medicine, Nanjing 210023, China.
- 3Jiangsu Collaborative Innovation Center of TCM Cancer Prevention and Treatment, Nanjing 210023, China.
- 4Geneseeq Research Institute, Nanjing Geneseeq Technology Inc, Nanjing 210032, China.
- 5Department of Pathology, Affiliated Hospital of Nanjing University of Chinese Medicine, Nanjing 210029, China.
Abstract
Background
MET amplification, although detected in only 2%-5% of intrahepatic cholangiocarcinomas (ICC), represents an actionable oncogenic driver. Treatment options after failure of first-line immunochemotherapy remain limited, and clinical data on vebreltinib, a novel highly selective MET inhibitor, in this molecular subset are scarce.Case presentation
A 61-year-old woman with metastatic ICC harboring high-level MET amplification (tissue gene copy number, 26.79) experienced disease progression on first-line durvalumab plus gemcitabine/cisplatin, second-line liposomal irinotecan/fluorouracil plus lenvatinib and durvalumab, radiotherapy, and transarterial chemoembolization. Following a brief period of vebreltinib (200 mg twice daily) plus durvalumab that was interrupted by grade 4 myelosuppression, the patient continued on vebreltinib monotherapy after hematologic recovery. Restaging computed tomography demonstrated a partial response (PR), with carbohydrate antigen 19-9 normalizing (a 99.2% reduction). This response lasted over 6 months until eventual disease progression.Conclusion
To our knowledge, this is the first report of vebreltinib monotherapy inducing a deep and durable partial response in heavily pretreated, MET-amplified ICC, with the response lasting over 6 months despite eventual disease progression. This case underscores the critical value of comprehensive molecular profiling after first-line treatment failure to identify actionable drivers such as MET amplification.
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BibTeX
@article{hang2026durable,
title = {{Durable response to vebreltinib in heavily pretreated MET-amplified intrahepatic cholangiocarcinoma: a case report.}},
author = {Yingying Hang and Lu Bai and Hsiquan Hsu and Ying Chen and Fufeng Wang and Wuyan Xu and Xia Zhen and Zihao Wang and Yuan Li and Jun Qian},
journal = {The oncologist},
year = {2026},
month = oct,
doi = {10.1093/oncolo/oyag409},
url = {https://europepmc.org/article/MED/42870395},
note = {CC BY},
}
RIS
TY - JOUR
TI - Durable response to vebreltinib in heavily pretreated MET-amplified intrahepatic cholangiocarcinoma: a case report.
AU - Yingying Hang
AU - Lu Bai
AU - Hsiquan Hsu
AU - Ying Chen
AU - Fufeng Wang
AU - Wuyan Xu
AU - Xia Zhen
AU - Zihao Wang
AU - Yuan Li
AU - Jun Qian
PY - 2026
DA - 2026/10/01
JO - The oncologist
DO - 10.1093/oncolo/oyag409
UR - https://europepmc.org/article/MED/42870395
AB - Background MET amplification, although detected in only 2%-5% of intrahepatic cholangiocarcinomas (ICC), represents an actionable oncogenic driver. Treatment options after failure of first-line immunochemotherapy remain limited, and clinical data on vebreltinib, a novel highly selective MET inhibitor, in this molecular subset are scarce.Case presentation A 61-year-old woman with metastatic ICC harboring high-level MET amplification (tissue gene copy number, 26.79) experienced disease progression on first-line durvalumab plus gemcitabine/cisplatin, second-line liposomal irinotecan/fluorouracil plus lenvatinib and durvalumab, radiotherapy, and transarterial chemoembolization. Following a brief period of vebreltinib (200 mg twice daily) plus durvalumab that was interrupted by grade 4 myelosuppression, the patient continued on vebreltinib monotherapy after hematologic recovery. Restaging computed tomography demonstrated a partial response (PR), with carbohydrate antigen 19-9 normalizing (a 99.2% reduction). This response lasted over 6 months until eventual disease progression.Conclusion To our knowledge, this is the first report of vebreltinib monotherapy inducing a deep and durable partial response in heavily pretreated, MET-amplified ICC, with the response lasting over 6 months despite eventual disease progression. This case underscores the critical value of comprehensive molecular profiling after first-line treatment failure to identify actionable drivers such as MET amplification.
SN - 1083-7159
SN - 1549-490X
ER -