OBJECTIVES: We report patient-reported outcomes (PROs) results from the pivotal phase 2 trial FOENIX-CCA2 (NCT02052778). FOENIX-CCA2 evaluated futibatinib, an irreversible FGFR1–4 inhibitor, among intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 fusions/rearrangements. Futibatinib demonstrated a confirmed objective response rate of 41.7%; adverse events were manageable. PROs were a prespecified secondary objective of the study.

METHODS: Patients had locally advanced/metastatic unresectable iCCA with FGFR2 fusions/rearrangements, ≥1 prior line of therapy (including gemcitabine/cisplatin) and ECOG performance status 0-1. Patients received continuous dosing with oral futibatinib 20 mg/day for 21 days per cycle. PRO measures included the EORTC-QLQ-C30, EQ visual analogue scale (VAS), and EQ-5D-3L. PROs were collected at baseline, cycles 2 and 4, every 3 cycles thereafter, and at end of treatment. PRO data were evaluated up to cycle 13 (PRO primary assessment timepoint), the last visit for which >50% of the PRO population had recorded PRO scores.

RESULTS: As of the primary cut-off date (October 1, 2020), 92 of 103 patients were evaluable for PROs, with 48 having PRO data at cycle 13. Median age was 58 years, 56.5% were female. Mean (SD) baseline EORTC QLQ-C30 global health status score was 70.1 (19.4). Mean scores were maintained to cycle 13 (~9.0 months on-treatment), with changes in scores at each cycle of <2.0 points. Similarly, mean EQ VAS scores (mean [SD] at baseline, 71.7 [20.3]) were maintained to cycle 13 and were within the population normative range. On individual EQ-5D-3L health dimensions (mobility, self-care, usual activities, pain, anxiety/depression), status from baseline to cycle 13 showed that 72–94% of patients remained the same or improved on futibatinib treatment.

CONCLUSIONS: Data from the pivotal phase 2 trial of futibatinib among patients with advanced iCCA demonstrated maintenance or improvement of PRO scores through cycle 13.