REGOBONE多队列研究探讨了瑞戈非尼对晚期骨肉瘤患者的疗效和安全性；该报告详细介绍了尤文肉瘤 (ES) 队列。尽管先前接受过标准治疗，但复发性 ES 仍出现进展的患者被随机 (2:1) 接受瑞戈非尼或安慰剂治疗。在集中确认病情进展后，服用安慰剂的患者可以交叉接受瑞格非尼治疗。主要终点是 8 周时的无进展率。单侧 α 为 0.05，功效为 80%，至少需要 14/24 的 8 周无进展患者才能成功。从 2014 年 9 月到 2019 年 11 月，共招募了 41 名患者。 36 名患者进行了疗效评估：23 名患者使用瑞戈非尼，13 名患者使用安慰剂。瑞格非尼组有 13 名患者（56%；单侧 95% CI [37.5%-[）] 在 8 周时无进展，而安慰剂组有 1 名患者（7.7%；95% CI [0.4%-[））。瑞戈非尼组的中位 PFS 为 11.4 周，安慰剂组为 3.9 周。十名安慰剂患者在病情进展后转而接受瑞戈非尼治疗。最常见的 ≥3 级瑞戈非尼相关不良事件是疼痛（22%）、乏力（17%）、血小板减少（13%）和腹泻（13%）。尽管在该随机队列中未达到统计学上的主要终点，但有证据表明，瑞戈非尼可能会适度延缓既往化疗失败后复发性 ES 的肿瘤进展。© 2023。作者，获得 Springer Nature Limited 的独家许可。

The REGOBONE multi-cohort study explored the efficacy and safety of regorafenib for patients with advanced bone sarcomas; this report details the Ewing sarcoma (ES) cohort.Patients with relapsed ES progressing despite prior standard therapy, were randomised (2:1) to receive regorafenib or placebo. Patients on placebo could crossover to receive regorafenib after centrally confirmed progression. The primary endpoint was the progression-free rate at 8 weeks. With one-sided α of 0.05, and 80% power, at least 14/24 progression-free patients at 8 weeks were needed for success.From September 2014 to November 2019, 41 patients were accrued. 36 patients were evaluable for efficacy: 23 on regorafenib and 13 on placebo. Thirteen patients (56%; one-sided 95% CI [37.5%-[)) were progression-free at 8 weeks on regorafenib vs. 1 (7.7%; 95% CI [0.4%-[) on placebo. Median PFS was 11.4 weeks on regorafenib, and 3.9 weeks on placebo. Ten placebo patients crossed over to receive regorafenib after progression. The most common grade ≥3 regorafenib-related adverse events were pain (22%), asthenia (17%), thrombocytopenia (13%) and diarrhoea (13%).Although the primary endpoint was not met statistically in this randomised cohort, there is evidence to suggest that regorafenib might modestly delay tumour progression in relapsed ES after failure of prior chemotherapy.© 2023. The Author(s), under exclusive licence to Springer Nature Limited.