确定口服黄体酮拮抗剂奥那司酮缓释剂（奥那司酮-XR）对复发性黄体酮受体（PR）阳性成人型颗粒细胞瘤（aGCT）、低级别浆液性卵巢癌（LGSOC）患者的安全性和有效性，或子宫内膜样子宫内膜癌 (EEC)。这项单机构 II 期研究纳入了 PR 阳性 aGCT、LGSOC 或 EEC 且接受过 ≥1 种既往化疗的患者。患者于2019年5月至2020年5月入组。通过免疫组织化学评估 PR 状态。符合条件的患者在入组后 3 年内收集的组织中 PR 表达≥1%。患者每天两次接受 50 毫克奥那司酮-XR，直至疾病进展或治疗停止。不良事件按照不良事件通用术语标准 5.0 版进行分级。主要终点是实体瘤 1.1 反应评估标准的总体反应率 (ORR)。次要终点是缓解持续时间、临床受益率 (CBR) 和安全性。 5 名 LGSOC 患者和 1 名 EEC 患者入组，但两个队列由于累积缓慢而提前结束。 14 名 aGCT 患者入组并完成了 1 期累积。没有观察到任何反应。 4 名 LGSOC 患者可进行评估，中位 PFS 为 4.4 个月（范围，1.8-NE），CBR 为 50%（范围，6.8%-93.2%）。所有 14 名 aGCT 患者均可进行评估，中位 PFS 为 2.8 个月（范围 1.6-4.9），6 个月 PFS 率为 21.4%（范围 5.2%-44.8%），12 个月 PFS 率为 14.3%（范围，2.3%-36.6%），CBR 为 35.7%（范围，12.8%-64.9%）。该研究未达到其主要终点。虽然奥那司酮-XR 在所有 3 个组中均具有良好的耐受性，但未观察到客观反应。版权所有 © 2024。由 Elsevier Inc. 出版。

To determine the safety and efficacy of the oral progesterone antagonist onapristone extended release (onapristone-XR) in patients with recurrent progesterone receptor (PR)-positive adult-type granulosa cell tumor (aGCT), low-grade serous ovarian cancer (LGSOC), or endometrioid endometrial cancer (EEC).This single-institution phase II study included patients with PR-positive aGCT, LGSOC, or EEC who received ≥1 prior line of chemotherapy. Patients were enrolled from 5/2019-5/2020. PR status was evaluated via immunohistochemistry. Eligible patients had PR expression ≥1% on tissue collected within 3 years of enrollment. Patients received 50 mg of onapristone-XR twice daily until disease progression or treatment discontinuation. Adverse events were graded by Common Terminology Criteria for Adverse Events version 5.0. The primary endpoint was overall response rate (ORR) by Response Evaluation Criteria in Solid Tumors 1.1. Secondary endpoints were response duration, clinical benefit rate (CBR), and safety.Five patients with LGSOC and 1 with EEC enrolled, but both cohorts closed early due to slow accrual. Fourteen patients with aGCT enrolled and completed stage 1 accrual. No responses were observed. Four patients with LGSOC were evaluable, with median PFS of 4.4 months (range, 1.8-NE) and CBR of 50% (range, 6.8%-93.2%). All 14 patients with aGCT were evaluable, with median PFS of 2.8 months (range, 1.6-4.9), 6-month PFS rate of 21.4% (range, 5.2%-44.8%), 12-month PFS rate of 14.3% (range, 2.3%-36.6%), and a CBR of 35.7% (range, 12.8%-64.9%).The study did not meet its primary endpoint. While onapristone-XR was well tolerated in all 3 arms, no objective responses were observed.Copyright © 2024. Published by Elsevier Inc.