Related Experiment Video
Updated: Dec 7, 2025
Patient-derived Heterogeneous Xenograft Model of Pancreatic Cancer Using Zebrafish Larvae as Hosts for Comparative Drug Assessment
Published on: April 30, 2019
Multicenter Phase II Trial of Axitinib Monotherapy for Gemcitabine-Based Chemotherapy Refractory Advanced Biliary
Naohiro Okano1, Junji Furuse1, Makoto Ueno2
1Department of Medical Oncology, Kyorin University Faculty of Medicine, Tokyo, Japan.
Lessons Learned:
Axitinib exhibited marginal activity against gemcitabine-refractory unselected biliary tract cancer. Pretreated soluble vascular endothelial growth factor receptor-2 may be a useful biomarker for axitinib treatment outcome. Ascites should be carefully monitored in patients receiving anti-vascular endothelial growth factor receptor therapy including axitinib in advanced biliary tract cancer.
Background:
There are no clear options for second-line treatment in patients with gemcitabine (GEM)-refractory biliary tract cancer (BTC). We conducted a multicenter, single-arm, phase II trial to confirm the efficacy and safety of axitinib, a potent selective inhibitor of vascular endothelial growth factor receptor (VEGFR)-1/2/3, in patients with GEM-refractory BTC.
Methods:
Patients refractory or intolerant to GEM-based chemotherapy were enrolled. Axitinib was administered orally at an initial dose of 5 mg twice daily. The primary endpoint was progression-free survival (PFS), and the threshold and expected values were set at 2 and 3 months, respectively. The target sample size was 32 patients.
Results:
Nineteen patients were enrolled. The trial was interrupted for a total of 13 months for the evaluation of adverse events. Thirteen patients were previously treated with ≥2 regimens. The median PFS was 2.8 months (95% confidence interval [CI]: 2.1-4.1). The median overall survival was 5.8 months (95% CI: 3.3-9.7). The response rate was 5.3% (95% CI: 0.0-15.3). Grade 3 ascites occurred in two patients. Baseline soluble VEGFR-2 levels were significantly associated with PFS.
Conclusion:
Axitinib exhibited marginal activity against GEM-refractory BTC. Ascites should be carefully monitored in axitinib-treated patients with advanced BTC.
Insights
Axitinib showed limited effectiveness in biliary tract cancer resistant to gemcitabine. Soluble VEGFR-2 levels may predict treatment success, and ascites requires careful monitoring during axitinib therapy.
Area of Science:
- Oncology
- Medical Research
Background:
- Biliary tract cancer (BTC) lacks effective second-line treatment options after gemcitabine (GEM) failure.
- Axitinib, a selective vascular endothelial growth factor receptor (VEGFR) inhibitor, was investigated for GEM-refractory BTC.
Purpose of the Study:
- To evaluate the efficacy and safety of axitinib in patients with gemcitabine-refractory biliary tract cancer.
- To identify potential predictive biomarkers for axitinib treatment response.
Main Methods:
- A multicenter, single-arm, phase II clinical trial was conducted.
- Patients received oral axitinib at 5 mg twice daily.
- Progression-free survival (PFS) was the primary endpoint.
Main Results:
- The median PFS was 2.8 months, and median overall survival was 5.8 months.
- A response rate of 5.3% was observed.
- Elevated baseline soluble VEGFR-2 levels correlated significantly with improved PFS.
Conclusions:
- Axitinib demonstrated marginal activity in gemcitabine-refractory BTC.
- Soluble VEGFR-2 may serve as a predictive biomarker for axitinib efficacy.
- Close monitoring for ascites is crucial in advanced BTC patients treated with axitinib.
More Related Videos
03:07Single-Port Robotic-assisted Transaxillary Breast-conserving Surgery: A Prospective, Single-arm, Non-randomized Phase IIa Clinical Trial
Published on: August 19, 2025
04:04Endobronchial Ultrasound-guided Intratumoral Injection of Cisplatin for the Treatment of Isolated Mediastinal Recurrence of Lung Cancer
Published on: February 12, 2017
