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Outcomes following FGFR inhibitor therapy in patients with cholangiocarcinoma: Multi-center single institution cohort experience
Background
Cholangiocarcinomas (CCA) are a group of heterogeneous tumors arising from the biliary system. Significant sequencing efforts have provided further insights on the molecular mechanisms of this disease including fibroblast growth factor receptor (FGFR) alterations, which occurs in approximately 15-20% of intrahepatic cholangiocarcinomas. There is lack of data on outcomes of patients following cessation of FGFR inhibitor (FGFRi) therapy. Herein, we described the patient characteristics and treatment outcomes among patients with cholangiocarcinoma harboring FGFR alterations treated with chemotherapy, another targeted therapy or a second FGFRi following treatment with a first FGFRi from a multi-center single institution experience.
Methods
We conducted a retrospective study of patients with pathologic confirmed diagnosis of cholangiocarcinoma treated at the Mayo Clinic Enterprise. The study was reviewed and approved by the institutional review board. The identified patients had FGFR alterations obtained from clinical genomic reports. The primary outcome was overall survival (OS) and progression free survival (PFS).
Results
Our group identified 88 advanced or metastatic cholangiocarcinoma patients, 28 males (31.8%) and 60 females (68.2%), harboring FGFR alterations who received FGFRi. Median progression free survival (PFS) on first FGFRi was 6.6 months for all patients (95% CI: 5.5 – 8.3). Following cessation of first FGFRi therapy 55% of patients received systemic therapy as next line: 67% chemotherapy or other targeted treatment and 33% received another FGFRi therapy. Median PFS for patients who received chemotherapy or other targeted agent was 2.1 months (95% CI: 1.6 – 5.7) and for patients who received a second FGFRi following first FGFRi therapy was 3.7 months (95% CI: 1.5 – not evaluable). 28% (N=25) of the patients received another FGFRi as any line after first FGFRi therapy and median PFS was 4.0 months.
Conclusions
This is a large multi-center single institution cohort study assessing the outcomes among patients with cholangiocarcinoma treated with a second FGFRi or chemotherapy after initial treatment with FGFR inhibitors. This data reflects the real-world experience at a tertiary cancer center. Following FGFRi treatment, almost half of the patients are able to receive next line of therapy. As more novel agents are being introduced and the optimal sequencing of FGFRi is under investigation, detailed understanding of outcomes following treatment with an FGFRi is essential.
Legal entity responsible for the study
The authors.
Funding
This work was partially supported by the National Institutes of Health Grants: P30CA15083 (Mayo Clinic Comprehensive Cancer Center grant).
Disclosures
T. Bekaii-Saab: Honoraria (self): Royalties: Uptodate; Advisory / Consultancy: Consulting (to institution): Ipsen, Arcus, Pfizer, Seattle Genetics, Bayer, Genentech, Incyte, Eisai and Merck. , Consulting (to self): Stemline, AbbVie, Boehringer Ingelheim, Janssen, Daichii Sankyo, Natera, TreosBio, Celularity, Exact Science, Sobi, Beigene, Kanaph, Astra Zeneca, Deciphera, MJH Life Sciences, Aptitude Health, Illumina and Foundation Medicine, IDMC/DSMB: Fibrogen, Suzhou Kintor, Astra Zeneca, Exelixis, Merck/Eisai, PanCan and 1Globe. ; Research grant / Funding (institution): Agios, Arys, Arcus, Atreca, Boston Biomedical, Bayer, Eisai, Celgene, Lilly, Ipsen, Clovis, Seattle Genetics, Genentech, Novartis, Mirati, Merus, Abgenomics, Incyte, Pfizer, BMS.; Licensing / Royalties: WO/2018/183488: HUMAN PD1 PEPTIDE VACCINES AND USES THEREOF – Licensed to Imugene, WO/2019/055687: METHODS AND COMPOSITIONS FOR THE TREATMENT OF CANCER CACHEXIA – Licensed to Recursion. All other authors have declared no conflicts of interest.
