Masanori Tsujie1, Tomohisa Iwai2, Shoji Kubo3, Takashi Ura4, Etsuro Hatano5, Daisuke Sakai6, Yutaka Takeda7, Masaki Kaibori8, Tomoe Kobayashi9, Akio Katanuma10, Yu Katayose11, Koji Fukase12. 1. Department of Surgery, Osaka Rosai Hospital, Osaka, Japan. 2. Department of Gastroenterology, Kitasato University Hospital, Sagamihara, Japan. 3. Department of Hepato-Biliary-Pancreatic Surgery, Osaka City University Hospital, Osaka. 4. Department of Clinical Oncology, National Hospital Organization Kyoto Medical Center, Kyoto, Japan. 5. Department of Gastroenterological Surgery, Hyogo College of Medicine, Nishinomiya, Japan. 6. Department of Gastroenterological Surgery, Osaka University Hospital, Suita, Japan. 7. Department of Gastroenterological Surgery, Kansai Rosai Hospital, Amagasaki, Japan. 8. Department of Surgery, Kansai Medical University, Osaka, Japan. 9. Department of Gastroenterology and Hematology, Tomakomai City Hospital, Tomakomai, Japan. 10. Center for Gastroenterology, Teine-Keijinkai Hospital, Sapporo, Japan. 11. Department of Hepato-Biliary Pancreatic Surgery, Tohoku Medical and Pharmaceutical University Hospital, Sendai, Japan. 12. Department of Surgery, Japanese Red Cross Ishinomaki Hospital, Ishinomaki, Japan.
Abstract
OBJECTIVE: Fibroblast growth factor receptor gene alterations have emerged as promising drug targets for intrahepatic cholangiocarcinoma, a rare cancer that has a poor prognosis. This study evaluated the frequency of fibroblast growth factor receptor 2 fusions in clinical specimens from Japanese patients with iCCA. METHODS: This study enrolled 116 patients who had histologically or cytologically confirmed adenocarcinoma and been diagnosed as relapsing after resection or with unresectable intrahepatic cholangiocarcinoma. We evaluated the frequency of fibroblast growth factor receptor 2 fusions-positive cells in their specimens using break-apart fluorescent in situ hybridization 'for 114 patients who met the study protocol'. RESULTS: Of a total of 114 cases, six (5.3%) were identified as fibroblast growth factor receptor 2 fusions-positive with a high frequency (87% or more) of fibroblast growth factor receptor 2 fusions-positive tumour cells whereas the remainder, with the exception of three cases with indeterminate results, were identified as fibroblast growth factor receptor 2 fusions-negative. The patients' baseline characteristics as well as their objective response rates, disease control rates, times to progression, and times to treatment failure with previous or ongoing first-line chemotherapy did not have any obvious relationship to the proportion of fibroblast growth factor receptor 2 fusions-positive case. CONCLUSIONS: Further detailed elucidation of fibroblast growth factor receptor 2 fusion status is expected to contribute to the development of promising therapeutic options for patients suffering from recurrent or unresectable intrahepatic cholangiocarcinoma.
OBJECTIVE: Fibroblast growth factor receptor gene alterations have emerged as promising drug targets for intrahepatic cholangiocarcinoma, a rare cancer that has a poor prognosis. This study evaluated the frequency of fibroblast growth factor receptor 2 fusions in clinical specimens from Japanese patients with iCCA. METHODS: This study enrolled 116 patients who had histologically or cytologically confirmed adenocarcinoma and been diagnosed as relapsing after resection or with unresectable intrahepatic cholangiocarcinoma. We evaluated the frequency of fibroblast growth factor receptor 2 fusions-positive cells in their specimens using break-apart fluorescent in situ hybridization 'for 114 patients who met the study protocol'. RESULTS: Of a total of 114 cases, six (5.3%) were identified as fibroblast growth factor receptor 2 fusions-positive with a high frequency (87% or more) of fibroblast growth factor receptor 2 fusions-positive tumour cells whereas the remainder, with the exception of three cases with indeterminate results, were identified as fibroblast growth factor receptor 2 fusions-negative. The patients' baseline characteristics as well as their objective response rates, disease control rates, times to progression, and times to treatment failure with previous or ongoing first-line chemotherapy did not have any obvious relationship to the proportion of fibroblast growth factor receptor 2 fusions-positive case. CONCLUSIONS: Further detailed elucidation of fibroblast growth factor receptor 2 fusion status is expected to contribute to the development of promising therapeutic options for patients suffering from recurrent or unresectable intrahepatic cholangiocarcinoma.