Multicenter Phase II Trial of Axitinib Monotherapy for Gemcitabine-Based Chemotherapy Refractory Advanced Biliary Tract Cancer (AX-BC Study)

The oncologist

article

Naohiro Okano¹  Yousuke Nakai²  Satoshi Kobayashi⁴  Manabu Morimoto⁴  Hiroko Hosoi⁵  Satoko Maeno¹  Fumio Nagashima¹  Masafumi Ikeda⁶  Takuji Okusaka⁵  Junji Furuse¹  Makoto Ueno⁴  Chigusa Morizane⁵  Takeharu Yamanaka⁷  Hidenori Ojima⁸  Masato Ozaka⁹  Mitsuhito Sasaki⁶  Naminatsu Takahara² 

[1] Department of Medical Oncology, Kyorin University Faculty of Medicine;Department of Gastroenterology, The University of Tokyo;Department of Endoscopy and Endoscopic Surgery, The University of Tokyo;Department of Gastroenterology, Hepatobiliary and Pancreatic Medical Oncology Division, Kanagawa Cancer Center;Department of Hepatobiliary and Pancreatic Oncology, National Cancer Center Hospital;Department of Hepatobiliary and Pancreatic Oncology, National Cancer Center Hospital East;Department of Biostatistics, Yokohama City University School of Medicine;Department of Pathology, Keio University School of Medicine;Department of Hepatobiliary and Pancreatic Oncology, The Cancer Institute of JFCR

关键词: Axitinib;    Gemcitabine;    Biliary tract cancer;    Chemotherapy refractory;   

DOI  :  10.1002/onco.13547

学科分类：地质学

来源: AlphaMed Press Incorporated

【 摘 要 】

Background. There are no clear options for second-line treatment in patients with gemcitabine (GEM)-refractory biliary tract cancer (BTC). We conducted a multicenter, singlearm, 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 GEMrefractory 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 GEMrefractory BTC. Ascites should be carefully monitored in axitinibtreated patients with advanced BTC.

【 授权许可】

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