Division of Hematology/Oncology, Northwestern University, Feinberg School of Medicine, NMH/Arkes Family Pavilion Suite 850, 676 N Saint Clair, Chicago, IL, USA.
Division of Hematology/Oncology, Mayo Clinic in Florida, Jacksonville, FL, USA.
Target Oncol. 2018 Aug;13(4):417-422. doi: 10.1007/s11523-018-0575-0.
Sarcomas encompass a group of rare solid tumors responsible for approximately 1% of all cancer-related deaths in the United States each year. Subtypes include, but are not limited to, soft tissue sarcomas (STS) such as leiomyosarcoma, liposarcoma, pleomorphic sarcoma, and gastrointestinal stromal tumor (GIST). Treatment options for patients with STS vary depending on, among other factors, histological subtype. Data from a mix of phase 2 and phase 3 trials have suggested that the orally available multikinase inhibitor regorafenib may have efficacy in patients with STS who have progressed on previous lines of systemic therapy. Some clinical benefit of regorafenib has been shown in patients with leiomyosarcoma, synovial sarcoma, GIST, Ewing's sarcoma, and other sarcoma subtypes, suggesting a broad spectrum of potential activity in this population. Studies have also shown that the safety profile of regorafenib is acceptable in these patients, with adverse events that can be managed through dose reductions and/or interruptions as well as other supportive measures.
肉瘤是一组罕见的实体肿瘤，每年导致美国约 1%的癌症相关死亡。亚型包括但不限于软组织肉瘤（STS），如平滑肌肉瘤、脂肪肉瘤、多形性肉瘤和胃肠道间质瘤（GIST）。STS 患者的治疗选择取决于多种因素，包括组织学亚型。来自 2 期和 3 期试验的混合数据表明，口服多激酶抑制剂瑞戈非尼可能对先前接受过系统治疗的 STS 患者有效。在患有平滑肌肉瘤、滑膜肉瘤、GIST、尤文肉瘤和其他肉瘤亚型的患者中已显示出瑞戈非尼的临床获益，表明该药在该人群中有广泛的潜在活性。研究还表明，瑞戈非尼在这些患者中的安全性是可以接受的，不良反应可以通过减少剂量和/或中断以及其他支持性措施来管理。
