Department of Medicine, Léon Bérard Center, Lyon, France.
Medical Oncology Unit, La Timone University Hospital, Marseille, France.
Curr Treat Options Oncol. 2022 Nov;23(11):1477-1502. doi: 10.1007/s11864-022-00990-0. Epub 2022 Sep 30.
Sarcomas are a rare group of tumors with many subtypes, conventionally classified into soft-tissue sarcomas and bone sarcomas. Chemotherapeutic regimens form the mainstay of systemic therapy but are not well defined beyond the first-line setting and clinical outcomes are variable. Tyrosine kinase inhibitors (TKIs), with a broad inhibition profile which have been shown to target tumor angiogenesis, have an established role in the treatment of sarcomas without characteristic driver alterations. One such TKI, regorafenib, has been evaluated in sarcomas and clinical data are discussed in this review. An overview of regorafenib data from five phase 2 and one phase 1b clinical trials in over 10 sarcoma subtypes (both soft-tissue and bone) in adult and pediatric patients is reviewed. Regorafenib demonstrated clinical benefit in patients with non-adipocytic soft-tissue sarcomas, osteosarcoma and Ewing sarcoma who had progressed on prior therapy. Patients with otherwise limited treatment options may therefore benefit from regorafenib therapy.
肉瘤是一组罕见的肿瘤，具有许多亚型，通常分为软组织肉瘤和骨肉瘤。化疗方案是系统治疗的主要方法，但在一线治疗以外的方案尚未明确，临床结果也各不相同。具有广泛抑制作用的酪氨酸激酶抑制剂（TKI）已被证实可靶向肿瘤血管生成，在无特征性驱动改变的肉瘤治疗中具有明确的作用。其中一种 TKI regorafenib 已在肉瘤中进行了评估，本综述讨论了相关临床数据。本文综述了五项 2 期和一项 1b 期临床试验的汇总数据，这些试验入组了超过 10 种肉瘤亚型（包括软组织和骨肉瘤）的成年和儿童患者。regorafenib 在前序治疗进展的非脂肪性软组织肉瘤、骨肉瘤和尤文肉瘤患者中显示出临床获益。因此，对于其他治疗选择有限的患者，regorafenib 治疗可能会受益。
