Clinical cancer research : an official journal of the American Association for Cancer Research · 2026
Erdafitinib in Patients with FGFR-Altered Advanced or Metastatic Cholangiocarcinoma.
СтатьяКлиническое исследование II фазыМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Pant Shubham, Park Joon Oh, Su Wu-Chou, Loriot Yohann, Carranza Omar, Corassa Marcelo, Doi Toshihiko, Qin Shukui, Tabernero Josep, Prenen Hans, Folprecht Gunnar, Winter Helen, Dal Molin Graziela Z, Shen Lin, Qian Jiaqi, Liao Huimin, Thomas Shibu, Sweiti Hussein, Triantos Spyros, Feng Yin-Hsun
Аннотация
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 leading to treatment discontinuation.
CONCLUSIONS: Pooled analyses confirm the robust efficacy of erdafitinib in a diverse population of pretreated patients with advanced/metastatic CCA harboring prespecified FGFR alterations. These findings are consistent with previously observed efficacy of FGFR-targeted agents in patients with CCA.
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 leading to treatment discontinuation.
CONCLUSIONS: Pooled analyses confirm the robust efficacy of erdafitinib in a diverse population of pretreated patients with advanced/metastatic CCA harboring prespecified FGFR alterations. These findings are consistent with previously observed efficacy of FGFR-targeted agents in patients with CCA.