Erdafitinib, an oral pan-FGFR inhibitor, demonstrated encouraging clinical activity and a manageable safety profile in Asian patients with FGFR
-aberrant advanced cholangiocarcinoma (CCA), according to preliminary results from a phase IIa trial presented at the 2018 ESMO Congress.
Investigators sought to evaluate the clinical efficacy and safety of erdafitinib in select patients with advanced CCA with FGFR
gene translocations or FGFR
gene mutations. To be eligible for enrollment, patients had to be 18 years of age or older, histologically or cytologically confirmed advanced CAA, an ECOG performance status of 0 or 1, and must have failed at least 1 prior line of therapy.
Those who had been treated with chemotherapy, targeted therapies, immunotherapy, or any investigational anticancer agent within the 2 weeks prior to the trial starting were ineligible to enroll, as were those with uncontrolled or history of cardiovascular disease, and those with persistent phosphate greater than the upper limit of normal.
Of the 193 patients who were molecularly screened, 29 patients had FGFR
alterations. Of those with alterations, 7 patients had FGFR2
fusions, 3 had FGFR2 mutations, and 2 had FGFR3
mutation; these 12 were given erdafitinib at a continuous dose of 8 mg once daily, with 1 patient uptitrated to receive a 9-mg dose. Eleven of the 12 patients were deemed evaluable for response. The median number of treatment cycles was 7.0 (range, 2-19), while the median treatment duration was 6.34 months (1.6-17.5).
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