1. Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study
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Guo‐Ming Shi, Xiao‐Yong Huang, Tian‐Fu Wen, Tian‐Qiang Song, Ming Kuang, Hai‐Bo Mou, Le‐Qun Bao, Hai‐Tao Zhao, Hong Zhao, Xie‐Lin Feng, Bi‐Xiang Zhang, Tao Peng, Yu‐Bao Zhang, Xiang‐Cheng Li, Hong‐Sheng Yu, Yu Cao, Lian‐Xin Liu, Ti Zhang, Wei‐Lin Wang, Jiang‐Hua Ran, Ying‐Bin Liu, Wei Gong, Ming‐Xia Chen, Lian Cao, Yang Luo, Yan Wang, Hui Zhou, Guo‐Huan Yang, Jia Fan, and Jian Zhou
- Subjects
antitumor activity ,cholangiocarcinoma ,FGFR2 fusions or rearrangements ,pemigatinib ,phase II ,Neoplasms. Tumors. Oncology. Including cancer and carcinogens ,RC254-282 - Abstract
Abstract Objective This study evaluated the antitumor activity and safety of pemigatinib in previously treated Chinese patients with advanced cholangiocarcinoma and fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements. Background Pemigatinib provided clinical benefits for previously treated patients with cholangiocarcinoma carrying FGFR2 fusions or rearrangements and was approved for this indication in multiple countries. Methods In this ongoing, multicenter, single‐arm, phase II study, adult patients with locally advanced or metastatic cholangiocarcinoma carrying centrally confirmed FGFR2 fusions or rearrangements who had progressed on ≥1 systemic therapy received 13.5 mg oral pemigatinib once daily (3‐week cycle; 2 weeks on, 1 week off) until disease progression, unacceptable toxicity, or consent withdrawal. The primary endpoint was objective response rate (ORR) assessed by an independent radiology review committee. Results As of January 29, 2021, 31 patients were enrolled. The median follow‐up was 5.1 months (range, 1.5–9.3). Among 30 patients with FGFR2 fusions or rearrangements evaluated for efficacy, 15 patients achieved partial response (ORR, 50.0%; 95% confidence interval [CI], 31.3–68.7); 15 achieved stable disease, contributing to a disease control rate of 100% (95% CI, 88.4–100). The median time to response was 1.4 months (95% CI, 1.3–1.4), the median duration of response was not reached, and the median progression‐free survival was 6.3 months (95% CI, 4.9–not estimable [NE]). Eight (25.8%) of 31 patients had ≥grade 3 treatment‐emergent adverse events. Hyperphosphatemia, hypophosphatasemia, nail toxicities, and ocular disorders were mostly
- Published
- 2023
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