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Axitinib for Gemcitabine-refractory Advanced Biliary Tract Cancer: Report of 5 Cases
Naohiro Okano1, Akiyoshi Kasuga2, Kirio Kawai2
1Department of Medical Oncology, Kyorin University Faculty of Medicine, Tokyo, Japan naohiro-okano@ks.kyorin-u.ac.jp.
Background/Aim:
Vascular endothelial growth factor receptor (VEGFR) has been identified as a treatment target for biliary tract cancer (BTC) and axitinib is a selective inhibitor of vascular endothelial growth factor receptor (VEGFR)-1/2/3. This study was conducted as a preliminary evaluation of the safety and efficacy of axitinib for patients with advanced BTC.
Patients And Methods:
Patients refractory to gemcitabine-based regimens were administered axitinib at the dose of 5 mg twice daily.
Results:
Five patients (3 male and 2 female) with a median age of 68 years were enrolled. Although 3 patients developed treatment-related grade 3/4 adverse events (AEs), none of these patients required discontinuation of the protocol treatment due to the AEs. Partial response (PR) was achieved in 1 patient, with a 67% reduction. The response was classified as stable disease (SD) in 3 patients and as progressive disease (PD) in 1 patient. Overall survival (OS) and progression-free survival (PFS) ranged from 2.0 to 19.9 months and 1.5 to 7.4 months, respectively.
Conclusion:
This preliminary study suggested that axitinib is well-tolerated and might exert promising activity in patients with BTC.
Insights
Axitinib, a vascular endothelial growth factor receptor (VEGFR) inhibitor, shows promise for advanced biliary tract cancer (BTC). This preliminary study found axitinib well-tolerated with potential efficacy in refractory BTC patients.
Area of Science:
- Oncology
- Pharmacology
Background:
- Biliary tract cancer (BTC) is a challenging malignancy with limited treatment options.
- Vascular Endothelial Growth Factor Receptor (VEGFR) signaling is implicated in BTC progression.
- Axitinib is a potent inhibitor targeting VEGFR-1/2/3, presenting a potential therapeutic strategy.
Observation:
- A pilot study evaluated axitinib in five patients with advanced BTC, refractory to gemcitabine-based chemotherapy.
- Axitinib was administered at 5 mg twice daily.
- Patients experienced treatment-related adverse events, but none necessitated treatment discontinuation.
Findings:
- One patient achieved partial response (67% reduction in tumor burden).
- Three patients demonstrated stable disease (SD).
- Overall survival (OS) ranged from 2.0 to 19.9 months, and progression-free survival (PFS) ranged from 1.5 to 7.4 months.
Implications:
- Axitinib demonstrates a favorable safety profile and potential anti-tumor activity in advanced BTC.
- Further investigation in larger clinical trials is warranted to confirm these preliminary findings.
- Targeting VEGFR with axitinib may offer a new therapeutic avenue for patients with refractory BTC.
