121P - PemiBil: Efficacy and safety of pemigatinib in advanced cholangiocarcinoma with FGFR2 fusions/rearrangements in real-world, results of multicentric French cohort from ACABI consortium

Background

Fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements (f/r) occur in up to 14% of patients with intrahepatic cholangiocarcinoma (iCCA). PEMIGATINIB, an oral FGFR 1-3 inhibitor, demonstrated significant efficacy in the phase II FIGHT-202 study with a response rate of 37% in heavily pretreated patients. Neither phase III nor phase IV data are currently available but due to the reported efficacy, PEMIGATINIB was FDA and EMA approved and is available in France since July 2020. Our study aimed to evaluate the efficacy and safety of pemigatinib and the disease profile of iCCA with FGFR2 fusions/rearrangements in an unselected population.

Methods

This national multicenter retrospective cohort collected data on demographics, therapeutic management, toxicity profile, objective response rate, progression-free survival and overall survival from French patients with advanced cholangiocarcinoma with FGFR2 f/r who received PEMIGATINIB since July 2020.

Results

From July 2020 to September 2022, 50 patients were included. At diagnosis the mean age was 57 years with 74% female, mainly ECOG 0-1 (82%). 58% had received two or more previous systemic therapies. The objective response rate was 45.3% (3 complete responses and 21 partial responses, n = 24). The median progression-free survival was 9 months (IIQ 6-14) and the median overall survival was 18 months (IIQ 12-NA). Neither clinical nor molecular characteristics were predictive factors for treatment response. The most common toxicities were hyperphosphatemia (58%), nail toxicity (58%) and fatigue (58%). 35% of patients developed grade 3 toxicity at the 13.5 mg daily dose. 57.5% (n = 19) of patients with progression were able to receive at least one subsequent line of treatment, mostly a new FGFR inhibitor (58%). The median overall survival from the locally advanced or metastatic stage was 37 months.

Conclusions

Our study confirms the efficacy of pemigatinib in an unselected population with high response rates and survivals similar to the FIGHT-202 data. It also confirms the good tolerability of pemigatinib and the better prognosis of iCCA with FGFR2 f/r.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

Dr Nadim Fares, MD PhD, Digestive Oncology Department, Centre Hospitalier Universitaire de Toulouse - Hopital Rangueil, Toulouse, France.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.