Erdafitinib in Asian patients with advanced solid tumors: an open-label, single-arm, phase IIa trial

作者：Joon Oh Park, Yin‐Hsun Feng, Wu‐Chou Su, Do‐Youn Oh, Bhumsuk Keam, Lin Shen, Sang-We Kim, Xiufeng Liu, Huimin Liao, Min Qing, Chong Zhang, Jiaqi Qian, Xiaodan Tang, Liming Peng, Spyros Triantos, Hussein Sweiti · 发表于：BMC Cancer · 年份：2024 · DOI：10.1186/s12885-024-12584-0 · 被引用次数：16 · 研究领域：Fibroblast Growth Factor Research、Cholangiocarcinoma and Gallbladder Cancer Studies、Lung Cancer Treatments and Mutations

BACKGROUND: FGFR genomic aberrations occur in approximately 5-10% of human cancers. Erdafitinib has previously demonstrated efficacy and safety in FGFR-altered advanced solid tumors, such as gliomas, thoracic, gastrointestinal, gynecological, and other rare cancers. However, its efficacy and safety in Asian patients remain largely unknown. We conducted a multicenter, open-label, single-arm phase IIa study of erdafitinib to evaluate its efficacy in Asian patients with FGFR-altered advanced cholangiocarcinoma, non-small cell lung cancer (NSCLC), and esophageal cancer. METHODS: Patients with pathologically/cytologically confirmed, advanced, or refractory tumors who met molecular and study eligibility criteria received oral erdafitinib 8 mg once daily with an option for pharmacodynamically guided up-titration to 9 mg on a 28-day cycle, except for four NSCLC patients who received erdafitinib 10 mg (7 days on/7 days off) as they were recruited before the protocol amendment. The primary endpoint was investigator-assessed objective response rate per RECIST v1.1. Secondary endpoints included progression-free survival, duration of response, disease control rate, overall survival, safety, and pharmacokinetics. RESULTS: Thirty-five patients (cholangiocarcinoma: 22; NSCLC: 12; esophageal cancer: 1) were enrolled. At data cutoff (November 19, 2021), the objective response rate for patients with cholangiocarcinoma was 40.9% (95% CI, 20.7-63.6); the median progression-free survival was 5.6 m...