Purpose: Standard treatment for patients with unresectable locally advanced or metastatic soft-tissue sarcoma (LA/M STS) is chemotherapy based on anthracyclines, but patient tolerance of chemotherapy is limited. The present trial (NCT03792542) investigated the use of anlotinib as first-line treatment for patients with advanced STS, in particular liposarcoma (LPS). Patients and Methods: Eligible patients were previously untreated, pathologically confirmed, unresectable LA/M STS cases. Anlotinib was given orally at a dose of 12 mg once daily from day 1 to day 14 every 3 weeks until disease progression or intolerable adverse events (AEs) occurred. The primary endpoint was progression-free survival (PFS) and the secondary endpoints overall survival (OS), the objective response rate and the disease control rate (DCR). The safety profile was also evaluated. Results: Forty patients were enrolled from April 2019 to Jun 2022 and are included in the intention-to-treat analysis. The median PFS was 6.83 months [95% confidence interval (CI): 4.17–8.71] and the median OS 27.40 months (95% CI: 16.43–not evaluable); 1 patient reached partial response and 26 attained stable disease, with a DCR of 67.5% (27/40). Median PFS and OS times for LPS patients were 8.71 and 16.23 months, respectively. Ten (25.0%) patients had treatment-related AEs ≥ grade 3, with in particular a higher incidence of hypertension (15.0%) and proteinuria (7.5%). Conclusions: The findings suggest a potential benefit in employing front-line anlotinib to treat patients with STS, who are not eligible for cytotoxic chemotherapy. Of note, the clinical outcomes for the LPS subgroup of patients were encouraging.

中文翻译：

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安罗替尼一线治疗不适合化疗患者的软组织肉瘤：一项开放标签、单臂、2 期临床试验

目的：不可切除的局部晚期或转移性软组织肉瘤（LA/M STS）患者的标准治疗是基于蒽环类药物的化疗，但患者对化疗的耐受性有限。本试验 (NCT03792542) 研究了安罗替尼作为晚期 STS、特别是脂肪肉瘤 (LPS) 患者的一线治疗的用途。患者和方法：符合条件的患者是既往未经治疗、经病理证实、不可切除的 LA/M STS 病例。从第 1 天到第 14 天，每 3 周口服一次 12 mg 剂量的安罗替尼，每日一次，直至出现疾病进展或无法耐受的不良事件 (AE)。主要终点是无进展生存期（PFS），次要终点是总生存期（OS）、客观缓解率和疾病控制率（DCR）。还评估了安全性。结果：2019年4月至2022年6月期间入组了40名患者，并纳入意向治疗分析。中位 PFS 为 6.83 个月 [95% 置信区间 (CI)：4.17–8.71]，中位 OS 为 27.40 个月（95% CI：16.43 - 不可评估）；1 名患者达到部分缓解，26 名患者疾病稳定，DCR 为 67.5% (27/40)。LPS 患者的中位 PFS 和 OS 时间分别为 8.71 和 16.23 个月。10 名患者 (25.0%) 出现 3 级以上的治疗相关 AE，尤其是高血压 (15.0%) 和蛋白尿 (7.5%) 的发生率较高。结论：研究结果表明，使用一线安罗替尼治疗不适合接受细胞毒性化疗的 STS 患者具有潜在益处。值得注意的是，LPS 亚组患者的临床结果令人鼓舞。