An open-label single-arm phase II study of regorafenib for the treatment of angiosarcoma
An open-label single-arm phase II study of regorafenib for the treatment of angiosarcoma
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DOI：
10.1016/j.ejca.2021.06.027
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发表时间：
2021-07-17
影响因子：
8.4
通讯作者：
Van Tine, Brian A.
中科院分区：
文献类型：
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作者：
Agulnik, Mark;Schulte, Brian;Van Tine, Brian A.
目的：血管肉瘤是一组多种多样的侵袭性高级别血管肿瘤，治疗选择有限。我们试图确定小分子多激酶抑制剂瑞格非尼治疗转移性或局部晚期不能切除的血管肉瘤的安全性和有效性。患者和方法：在这项单臂、多中心、开放标签的II期临床试验中，31名患者被纳入，在28天的周期中，每天服用瑞格非尼160 mg PO，共21天。这项研究的主要终点是4个月的无进展生存期。次要终点包括总体存活率、应答率和安全性。东部合作肿瘤学小组(ECOG)评分为0-1，预期寿命至少4个月的患者(>；18岁)在至少一种但不超过4种既往治疗路线上取得进展的患者符合条件。结果：可评价疗效的23例患者中，完全缓解2例(8.7%)，部分缓解2例(8.7%)，总有效率为17.4%。中位PFS为5.5个月，其中12/23例(52.2%)PFS>4个月。可评估毒性的患者中有10/31(32.3%)有3级或更高的不良事件。结论：瑞格非尼是治疗难治性、转移性和不可切除的血管肉瘤的一种安全、有效的治疗方法。不良事件的发生率与以前对其他肿瘤类型的瑞格拉非尼的研究相当。单药瑞格非尼可考虑用于治疗转移性或不能切除的AS患者。2021爱思唯尔有限公司，版权所有。
Purpose: Angiosarcomas represents a diverse group of aggressive high-grade vascular tumours with limited therapeutic options. We sought to determine the safety and efficacy of regorafenib, a small-molecule multikinase inhibitor, in the treatment of metastatic or locally advanced unresectable angiosarcoma. Patients and methods: In this single-arm multicentre, open-label phase II clinical trial, 31 patients were enrolled and received regorafenib 160 mg PO daily for 21 days of a 28-day cycle. The primary endpoint for the study was progression-free survival at 4 months. Secondary endpoints included overall survival, response rate, and safety. Patients (>18 years) with an Eastern Cooperative Oncology Group (ECOG) score of 0-1, a life expectancy of at least 4 months who had progressed on at least one but no more than 4 prior lines of therapy were eligible. Results: Of the 23 patients evaluable for efficacy, 2 had a complete response (8.7%), and 2 had a partial response (8.7%), for a total overall response rate of 17.4%. Median PFS was 5.5 months, and 12/23 patients (52.2%) had a PFS of greater than 4 months. 10/31 (32.3%) patients evaluable for toxicity had a grade 3 or higher adverse events. Conclusions: Regorafenib is a safe and active treatment for refractory metastatic and unresectable angiosarcoma. Rates of adverse events were comparable to prior studies of regorafenib for other tumour types. Regorafenib, the single agent, could be considered as therapy for patients with metastatic or unresectable AS. 2021 Elsevier Ltd. All rights reserved.