Pemigatinib in Chinese patients with advanced/metastatic or surgically unresectable cholangiocarcinoma including FGFR2 fusion or rearrangement: Updated data from an open-label, single-arm, multicenter phase II study (CIBI375A201 study).

作者：Guoming Shi, Xiaoyong Huang, Tianfu Wen, Tianqiang Song, Ming Kuang, Haibo Mou, Lequn Bao, Haitao Zhao, Hong Zhao, Xielin Feng, Bixiang Zhang, Tao Peng, Yubao Zhang, Xiangcheng Li, Hongsheng Yu, Yu Cao, Yang Luo, Mingxia Chen, Jia Fan, Jian Zhou · 发表于：Journal of Clinical Oncology · 年份：2022 · DOI：10.1200/jco.2022.40.16_suppl.e16183 · 被引用次数：6 · 研究领域：Cholangiocarcinoma and Gallbladder Cancer Studies、Fibroblast Growth Factor Research、Viral-associated cancers and disorders

e16183 Background: Pemigatinib is a selective FGFR inhibitor that showed effectiveness and tolerability in patients with cholangiocarcinoma with FGFR2 fusion or rearrangement. However, pemigatinib has not been investigated in Chinese population with cholangiocarcinoma (CCA). Initial efficacy and safety data were previously presented (G-M. Shi et al. ESMO 2021; NCT04256980). Here, we report updated follow-up results from the phase II study. Methods: Patients aged 18 years or older with recurrent or metastatic CCA that failed at least one prior systemic therapy were enrolled. In Part 1, 3 subjects were enrolled regardless of the FGFR2 status and were treated at 9 mg pemigatinib. The other 31 subjects with documented FGFR2 fusion or rearrangement were enrolled in Part 2 and received 13.5 mg pemigatinib. From 2/26/2020 to 12/20/2021, all the subjects in both parts were orally given pemigatinib QD on a 2 weeks on/1 week off schedule until disease progression, unacceptable toxicity, withdrawal of consent, or physician decision. The primary efficacy end point was ORR assessed by independent radiological review committee (IRRC) per RECIST V1.1 in 31 patients enrolled in Part 2. Results: As of the data cutoff date (Dec 20 th , 2021), with a median follow up of 12.6 (11.8, 14.2) months, among 30 efficacy evaluable subjects with 1 participant excluded due to inadequate FGFR2 aberrant frequency, the updated confirmed objective response rate (ORR) was 60% (95% CI: 42.5%, 77.5%) per RECIST...