A phase 2 trial of regorafenib as a single agent in patients with chemotherapy refractory advanced and metastatic biliary adenocarcinoma/cholangiocarcinoma.
A phase 2 trial of regorafenib as a single agent in patients with chemotherapy refractory advanced and metastatic biliary adenocarcinoma/cholangiocarcinoma.
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瑞戈非尼作为单药治疗化疗难治性晚期和转移性胆管腺癌/胆管癌患者的 2 期试验。
DOI：
10.1200/jco.2017.35.15_suppl.4081
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发表时间：
2017
期刊：
影响因子：
--
通讯作者：
H. Zeh
中科院分区：
文献类型：
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作者：
Weijing Sun;D. Normolle;N. Bahary;J. Ohr;B. Lembersky;K. Patel;Anuj K. Patel;James J. Lee;E. Chu;Natalie Streeter;Summer Drummond;John P. Morcos;M. Sulecki;D. Geller;J. Marsh;A. Tsung;H. Zeh
4081背景：胆管腺癌/胆管癌是一种罕见但侵袭性强的肿瘤。大多数患者存在不可切除或转移性疾病，5年生存率约为5%。没有二线治疗方案证明对这种疾病有临床获益。瑞戈非尼是一种口服多激酶抑制剂，具有强效抗肿瘤活性。这项单臂II期研究评价了瑞戈非尼单药治疗全身化疗失败的晚期或转移性胆管癌/胆管癌患者的疗效和安全性。研究方法：ECOG PS 0- 1且肝脏、肾脏和骨髓功能良好的患者每天口服一次瑞戈非尼，28天为一个周期，给药21天，停药7天。前3例患者的初始剂量为160 mg。毒性评估后，后续患者的剂量降至120 mg。主要终点为PFS，零假设为2.0个月，中位PFS ≥3.5个月作为研究药物活性的证据（α = 0.10，80%把握度）。中学...
4081Background: Biliary adenocarcinoma/cholangiocarcinoma is a rare but aggressive neoplasm. Most patients present with unresectable or metastatic disease with 5-year survival rate ~5%. No second-line regimen has demonstrated clinical benefit in this disease. Regorafenib is an oral multi-kinase inhibitor with potent antitumor activity. This single arm phase II study evaluates the efficacy and safety of regorafenib as a single agent in advanced or metastatic biliary carcinoma/cholangiocarcinoma pts who failed systemic chemotherapy. Methods: Patients with ECOG PS 0-1and adequate liver, kidney and bone marrow function were given regorafenib orally once daily, 21 days on and 7 days off in a 28-day cycle. The initial dose of 160 mg was given to the first 3 patients. After toxicity assessment, the dose was reduced to 120 mg for the subsequent pts. The primary endpoint is PFS with the null hypotheses of 2.0 months, and median PFS ≥3.5 months as evidence of the study drug activity (α = 0.10, 80% power). Secondary...