Real-world Data Supports Findings from FIGHT-202 in Cholangiocarcinoma

Following treatment initiation, the real-world progression-free survival proportion was 95.8% (95% CI, 90.3%-98.2%) at 3 months and 71.5% (95% CI, 61.4%-79.4%) at 6 months.

Real-world data on the use of the FGFR inhibitor pemigatinib (Pemazyer) among a more diverse population of patients with cholangiocarcinoma (CCA) support its clinical benefit observed in the phase 2 FIGHT-202 (NCT02924376), according to recent findings from a real-world study published in The Oncologist.

The median real-world progression-free survival (rwPFS) observed in the study was 7.4 months (95% CI, 6.4-8.6), and the real-world overall response rate (rwORR) was 59.2% (95% CI, 50.0%-68.4%), with 5% of patients achieving a real-world complete response (CR) and 54.2% achieving a real-world partial response (PR). Following treatment initiation, the rwPFS proportion was 95.8% (95% CI, 90.3%-98.2%) at 3 months and 71.5% (95% CI, 61.4%-79.4%) at 6 months. The real-world overall survival proportion was 95.8% (95% CI, 90.3%-98.2%) at 3 months and 88.4% (95% CI, 80.3%-93.3%) at 6 months. Among those who achieved a CR or PR with pemigatinib, the median time to response was 3.5 months.

The retrospective, observational, physician-abstracted medical chart review study included 120 patients, with a median age of 65 years at the time of pemigatinib being prescribed. About half of the patient population (50.8%) were female, 55% were White, and 19.2% were Hispanic. At initial diagnosis, 70% of patients had intrahepatic cholangiocarcinoma. Most patients (90%) had metastatic disease at the time of pemigatinib being prescribed, and 78.3% of patients had an ECOG performance status of 0 or 1 at baseline.

The most frequently cited risk factors for cholangiocarcinoma were nonalcoholic fatty liver disease among 12.5% of patients, liver disease among 11.7%, primary sclerosing cholangitis among 3.3%, and hepatitis C among 3.3%. Metastases at baseline were most common in the liver (64.2%) and the lung (43.3%). The median follow-up time was 6.5 months following the prescribing of pemigatinib and 16.3 months following initial diagnosis of cholangiocarcinoma.

None of the patients who were diagnosed with advanced disease had received neoadjuvant systemic therapy or radiation. Prior surgery was reported in 12.5% of patients, and 3.3% had received adjuvant systemic therapy. Most patients (87.5%) initially received pemigatinib at a dose of 13.5 mg daily for 14 days, repeating every 21 days. Three patients initiated therapy with the 4.5-mg dose. During the 6.5-month median follow-up period, 2.5% of patients had a reduction in the frequency or dose of pemigatinib.

“One of the unexpected findings of this study was the relatively high percentage of patients with extrahepatic disease (distal or perihilar) at time of initial CCA diagnosis,” Kim Saverno, PhD, RPh, senior director of Health Economics and Outcomes Research at Incyte Corporation, and colleagues stated in the article. “While a previously reported US real-world analysis of patients indicated that 95% of patients with an FGFR2 fusion/rearrangement had intrahepatic disease, and 98% of patients in FIGHT-202 with FGFR2 fusion had intrahepatic disease, participating physicians in our real-world study reported that 70.0% of the patients who received pemigatinib had intrahepatic CCA at initial CCA diagnosis. Of the 102 patients in our study with FGFR2 fusions reported, 25.5% had extrahepatic CCA.”

All patients on the study were prescribed pemigatinib for unresectable, locally advanced, or metastatic cholangiocarcinoma and were at least 18 years of age. Pemigatinib was given as second-line therapy for 94.2% of patients on the study, and 5.8% received it in the third-line setting. FGFR2 testing was conducted in 111 (92.5%) patients, with all but one patient testing positive. The most common FGFR2 testing modality was next-generation sequencing, which was utilized for 95.5% of patients on the study. Fluorescence in situ hybridization testing was utilized for 2.7%, and 1.8% were tested via reverse transcription-polymerase chain reaction.

Reference

Saverno K, Zimmerman Savill KM, Brown-Bickerstaff C, et al. Real-world use of pemigatinib for the treatment of cholangiocarcinoma in the US. The Oncologist. oyae204. doi.10.1093/oncolo/oyae204