Erdafitinib in Patients with FGFR- Altered Advanced or Metastatic Cholangiocarcinoma

作者：S. Pant, Joon Oh Park, Wu-Chou Su, Yohann Loriot, Omar Carranza, Marcelo Corassa, Toshihiko Doi, Shukui Qin, Josep Tabernero, Hans Prenen, G. Folprecht, Helen Winter, Graziela Zibetti Dal Molin, Lin Shen, Jiaqi Qian, Huimin Liao, Shibu Thomas, Hussein Sweiti, Spyros Triantos, YIN-HSUN FENG · 发表于：Clinical Cancer Research · 年份：2025 · DOI：10.1158/1078-0432.ccr-25-2264 · 被引用次数：3 · 研究领域：Fibroblast Growth Factor Research、Cholangiocarcinoma and Gallbladder Cancer Studies、Bladder and Urothelial Cancer Treatments

PURPOSE: Up to 20% of patients with cholangiocarcinoma (CCA) harbor FGFR gene aberrations. Erdafitinib is approved for pretreated, locally advanced/metastatic urothelial carcinoma with susceptible FGFR3 alterations. The present study evaluated the efficacy and safety of erdafitinib using a pooled analysis of patients with CCA from the RAGNAR and LUC2001 studies. EXPERIMENTAL DESIGN: In RAGNAR (phase II, global, tumor-agnostic study) and LUC2001 (an open-label, multicenter, phase IIa study in Asian patients), patients with advanced solid tumors after ≥1 prior lines of therapy received once daily oral erdafitinib (8 mg/day with an option for pharmacodynamically guided up-titration to 9 mg). Patients were pooled for efficacy [objective response rate (ORR) per blinded independent review committee, duration of response (DOR), progression-free survival (PFS), and overall survival (OS)] and safety analyses. RESULTS: At a median efficacy follow-up of 14.7 months in 78 erdafitinib-treated patients (RAGNAR: n = 66; LUC2001: n = 12), ORR was 55% [95% confidence interval (CI), 43.4-66.4]. The median time to response was 1.7 months; the median DOR, PFS, and OS were 6.9 (95% CI, 4.37-8.61), 8.5 (95% CI, 6.83-9.72), and 18.1 (95% CI, 13.40-24.28) months, respectively. The most common treatment-emergent adverse events (TEAE) were hyperphosphatemia (83%), stomatitis (72%), diarrhea (68%), dry mouth (51%), and palmar-plantar erythrodysesthesia (51%); 42% had serious TEAEs, and 12% had TEAEs le...