Abstract 149P
Background
Cholangiocarcinoma has a poor prognosis and more than 60% of patients are ineligible for curative resection at diagnosis. Although gemcitabine plus cisplatin and an immune checkpoint inhibitor is the standard first-line palliative chemotherapy and FOLFOX is the second-line option, the efficacy remains limited and requires improvement. The FGFR2 fusion or rearrangement is a promising therapeutic target in intrahepatic (ICC) and perihilar cholangiocarcinoma (PCC), leading to novel therapeutic approaches. In Japan, 14% of resectable and 5-7% of advanced ICC are FGFR2 fusion or rearrangement. The FGFR2 fusion or rearrangement positivity and characteristics of the patients in the Asian region have not been well reported. Clarifying the frequency of the FGFR2 fusion or rearrangement will support the development of FGFR inhibitors in the Asian region.
Methods
The study was a 14-site prospective observational study conducted as part of the MASTER KEY Asia study under the ATLAS project in four countries. The ATLAS project is the Cancer Research Network in Asia of NCC Japan, and one of its missions is to stimulate cancer clinical trials. The primary endpoint was the FGFR2 fusion or rearrangement positivity rate in advanced ICC and PCC by FISH. In addition, all patients underwent a comprehensive genomic profiling using the TOP2 panel.
Results
113 patients were enrolled: 59 from Thailand, 28 from Malaysia, 17 from Taiwan, and 9 from Vietnam. Ninety-one ICC and 18 PCC patients were tested, while four patients were ineligible. The median age was 65 (29 to 80), and 54% (57) were male. Nineteen (18%) were recurrent cases. FISH was performed in 106 patients (90 with ICC and 16 with PCC). Four patients with ICC were FGFR2 rearrangement by FISH. The positivity rate of the FGFR2 fusion gene in advanced ICC was 4.4%, but there were no FGFR2 fusion-positive cases in PCC. Using the RNA-based NGS panel (TOP2), FGFR2 fusion was detected in all patients with positive FGFR2 rearrangement identified by FISH. Younger age (≤50 years) was significantly associated with the presence of FGFR2 fusion (p <0.01).
Conclusions
The positivity of FGFR2 fusion was slightly lower in the Asian region. However, consistent with previous reports, it was more common in younger ICC patients.
Clinical trial identification
NCT05217407.
Editorial acknowledgement
During the preparation of this work, the authors used DeepL in order to English editing. After using this tool/service, the authors reviewed and edited the content as needed and take full responsibility for the content of the publication. In addition, a review of the abstracts is being conducted by Eisai Co.
Legal entity responsible for the study
The first author, Yuta Maruki, is responsible for this study.
Funding
Eisai Co., Ltd.
Disclosure
All authors have declared no conflicts of interest.