瑞戈非尼在晚期经治软组织肉瘤中的活性：一项单中心II期研究的结果
Activity of regorafenib in advanced pretreated soft tissue sarcoma: Results of a single-center phase II study.
作者信息
Marrari Andrea, Bertuzzi Alexia, Bozzarelli Silvia, Gennaro Nicolò, Giordano Laura, Quagliuolo Vittorio, De Sanctis Rita, Sala Simona, Balzarini Luca, Santoro Armando
机构信息
Medical Oncology and Hematology Unit.
Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), Italy.
出版信息
Medicine (Baltimore). 2020 Jun 26;99(26):e20719. doi: 10.1097/MD.0000000000020719.
BACKGROUND
Regorafenib, a multitargeted tyrosine kinase inhibitor, proved to be active in patients with soft tissue sarcomas (STS).
METHODS
We conducted an open-label, non-randomized, single-center phase II study in advanced pretreated STS patients. Patients received regorafenib 160 mg daily on days 1 enrule 21 of a 28-day cycle. The primary endpoint was the progression-free survival (PFS) at 8 weeks. Toxicity was registered.
RESULTS
Between April 2015 and November 2016, 21 patients were enrolled in the trial. A total of 13 out of 21 evaluable patients (61.9%) were progression-free at 8 weeks. Median PFS was 3.8 months (95% CI: 2.1-9.4). Median overall survival was 14.8 months (95% CI: 7.7-27.8). In the intention-to-treat population, we reported a PFS of 66.7% at 3 months (95% CI: 40.4-83.4) and 16.7% at 12 months (95% CI: 4.1-36.5). As per the RECIST criteria, the response rate was 4.7% (1 partial response out of 21 evaluable patients) with a clinical benefit rate of 61.9%; no complete response was observed. Treatment was well tolerated.
CONCLUSION
Regorafenib shows signs of clinical activity in patients with advanced STS.
CLINICAL TRIAL REGISTRATION
ClinicalTrials.gov NCT02307500.
背景
瑞戈非尼是一种多靶点酪氨酸激酶抑制剂，已被证明对软组织肉瘤（STS）患者有效。
方法
我们对晚期经预处理的STS患者进行了一项开放标签、非随机、单中心的II期研究。患者在28天周期的第1至21天每天接受160毫克瑞戈非尼治疗。主要终点是8周时的无进展生存期（PFS）。记录毒性反应。
结果
2015年4月至2016年11月期间，21例患者入组该试验。21例可评估患者中共有13例（61.9%）在8周时无进展。中位PFS为3.8个月（95%CI：2.1 - 9.4）。中位总生存期为14.8个月（95%CI：7.7 - 27.8）。在意向性治疗人群中，我们报告3个月时PFS为66.7%（95%CI：40.4 - 83.4），12个月时为16.7%（95%CI：4.1 - 36.5）。根据RECIST标准，缓解率为4.7%（21例可评估患者中有1例部分缓解），临床获益率为61.9%；未观察到完全缓解。治疗耐受性良好。
结论
瑞戈非尼在晚期STS患者中显示出临床活性迹象。
临床试验注册
ClinicalTrials.gov NCT02307500。
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