Genetic alterations in the FGFR pathway are emerging as promising therapeutic targets in patients with cholangiocarcinoma (CCA). A retrospective chart review (or a medical record review) was performed in patients with CCA who were found to have an FGFR mutation during molecular profiling of the tumor as part of routine care for CCA.
Data on demographics, risk factors, pathology, molecular characteristics, systemic therapies, radiographic response, time receiving treatment, and overall survival were collected in a multicenter collaborative effort across 7 academic centers. The lead author was Lipika Goyal, MD, MPhil, Medical Oncologist, Tucker Gosnell Center for Gastrointestinal Cancers, Massachusetts General Hospital, Boston, and results were presented at the 2020 ASCO annual meeting.
A total of 135 patients with FGFR-altered CCA were included in the review. The median age at diagnosis was 57 years.
The median number of palliative systemic therapies (treatments to relieve symptoms) that patients received was 3, and 40 of 135 patients received liver-directed therapy. For the 55 patients with FGFR2 fusions who received chemotherapy with gemcitabine and cisplatin as first-line palliative systemic therapy, the average time receiving the treatment was 6.2 months. The average overall survival from time of initial diagnosis was 36.1 months in the patients with FGFR2 fusions.
Among the 92 patients with FGFR2 fusions, 70 patients received an FGFR inhibitor in a clinical trial; 12 were subsequently treated with a second FGFR inhibitor, and 58.3% stayed on the second FGFR inhibitor for 4 months or more. Overall, 16 patients with a BICC1 fusion partner had an overall response rate of 42.9% on FGFR-selective inhibitors compared with 30.8% in non-BICC1 fusion partners.
The authors observed that patients with CCA harboring FGFR mutations were found to have a high rate of normal CA19-9, a high rate of bone metastases (cancer spreading to the bone), and a short average time of using first-line palliative therapy with gemcitabine and cisplatin.
They also noted that additional comparative studies would be necessary to evaluate these findings.
