Pemigatinib in previously treated Chinese patients with locally advanced or metastatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements: A phase II study.

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 <grade 3, except for 2 events ≥grade 3.

CONCLUSIONS:

The encouraging antitumor activity and favorable safety profile support the use of pemigatinib as a treatment in previously treated Chinese patients with cholangiocarcinoma and FGFR2 rearrangements.

• Authors: 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, Jian Zhou
• Journal: Cancer medicine (Cancer Med) Vol. 12 Issue 4 Pg. 4137-4146 (02 2023) ISSN: 2045-7634 [Electronic] United States
• PMID: 36127767 (Publication Type: Clinical Trial, Phase II, Multicenter Study, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2022 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.

Chemical References

• Antineoplastic Agents
• FGFR2 protein, human
• pemigatinib
• Receptor, Fibroblast Growth Factor, Type 2

Topics

• Adult
• Humans
• Antineoplastic Agents (adverse effects, therapeutic use)
• Bile Duct Neoplasms (drug therapy, genetics)
• Bile Ducts, Intrahepatic (pathology)
• Cholangiocarcinoma (drug therapy, genetics)
• East Asian People
• Receptor, Fibroblast Growth Factor, Type 2 (genetics)