Purpose: To assess efficacy and safety of apalutamide plus goserelin for androgen receptor (AR)-positive, unresectable or recurrent/metastatic salivary gland carcinoma (URM-SGC). Patients and Methods: This was an open-label, single-arm, multicenter phase II study for patients with AR-positive URM-SGC. The primary endpoint was the overall response rate (ORR) by an independent central radiology review (ICRR) in the first 24 response evaluable patients who had been observed at least 24 weeks from study initiation (primary RE patients). The efficacy was to be declared when at least 8 of the 24 primary RE patients responded. Results: 31 patients were enrolled. In the first 24 primary RE patients with a median follow-up of 7.4 months, confirmed ORR by ICRR was 25.0% (6/24 patients; 95%CI: 9.8%-46.7%; P =0.11 (one-sided)), which did not meet the predefined criteria of efficacy. Clinical benefit rate (ORR + rate of stable disease for at least 24 weeks) and median progression-free survival were 50.0% and 7.4 months, respectively. Both median duration of response and overall survival were not reached. Exploratory analyses showed a better ORR of 54.5% (6/11) in patients with AR-positivity ≥ 70% and no history of prior systemic therapy. Grade 3 or higher treatment-emergent adverse events were reported in 35.5% (11/31), which included skin rash, anemia, leukopenia, and cancer pain. Conclusions: Although this study did not meet the predefined efficacy criteria, apalutamide plus goserelin showed clinically meaningful efficacy in a subset of patients with AR-positive SGC and safety consistent with prior experience in prostate cancer.

中文翻译：

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阿帕鲁胺和戈舍瑞林治疗雄激素受体阳性唾液腺癌：2 期非随机临床试验，YATAGARASU


目的：评估阿帕鲁胺联合戈舍瑞林治疗雄激素受体（AR）阳性、不可切除或复发/转移性唾液腺癌（URM-SGC）的疗效和安全性。患者和方法：这是一项针对 AR 阳性 URM-SGC 患者的开放标签、单组、多中心 II 期研究。主要终点是独立中央放射学审查 (ICRR) 评估的前 24 名缓解可评估患者的总体缓解率 (ORR)，这些患者从研究开始起至少观察 24 周（主要 RE 患者）。当 24 名原发性 RE 患者中至少有 8 名有反应时，即可宣布疗效。结果：31 名患者入组。在中位随访时间为 7.4 个月的前 24 名原发性 RE 患者中，通过 ICRR 确认的 ORR 为 25.0%（6/24 名患者；95%CI：9.8%-46.7%；P =0.11（单侧）），不符合预定的功效标准。临床获益率（ORR + 至少 24 周疾病稳定率）和中位无进展生存期分别为 50.0% 和 7.4 个月。中位缓解持续时间和总生存期均未达到。探索性分析显示，AR 阳性≥ 70%且既往没有全身治疗史的患者的 ORR 更好，为 54.5% (6/11)。 35.5% (11/31) 报告了 3 级或以上治疗引起的不良事件，其中包括皮疹、贫血、白细胞减少和癌痛。结论：虽然这项研究没有达到预定的疗效标准，但阿帕鲁胺联合戈舍瑞林在 AR 阳性 SGC 患者的亚组中显示出具有临床意义的疗效，并且安全性与前列腺癌的既往经验一致。