Background/Objectives:The effects of combining chemotherapy with hormonal therapy based on hormone receptor (HR) expression in epithelial ovarian, fallopian tube, or primary peritoneal (EOC) remain unclear. This study evaluated the efficacy and safety of physician-chosen chemotherapy combined with hormonal therapy in patients with heavily pretreated advanced EOC, stratified by HR expression.Methods:This phase II, multicenter, pilot study included patients with heavily pretreated advanced EOC, allocated to estrogen receptor (ER)-dominant or progesterone receptor (PR)-dominant arms. Patients in the ER-dominant arm received tamoxifen plus physician-selected chemotherapy, while those in the PR-dominant arm received megestrol acetate (MA) plus chemotherapy. The primary outcome was the best objective response rate (ORR) for six months, assessed using an optimal two-stage Simon design.Results:Among 33 ER-dominant patients with high-grade serous carcinoma (HGSC), the six-month best ORR was 27.3% (3% complete response, 24.2% partial response). The six-month ORR and clinical benefit rate (CBR) were 18.8% and 37.5%, respectively, with 62.5% experiencing progressive disease (PD). Among three PR-dominant patients (two clear cell carcinoma and one HGSC), the six-month best ORR was 0%. The six-month ORR and CBR were also 0%, and all experienced PD within six months. No unacceptable toxicity related to tamoxifen or MA was encountered.Conclusions:In heavily pretreated advanced HGSC patients with ER-dominant expression, chemotherapy combined with tamoxifen showed encouraging clinical activity with favorable safety. While limited by the study design, these findings suggest a potential role for tailored hormonal therapy combined with chemotherapy based on HR expression in heavily pretreated advanced EOC. Clinical Trial Registration: KCT0004571
背景/目的：基于激素受体（HR）表达，将化疗与激素治疗联合应用于上皮性卵巢癌、输卵管癌或原发性腹膜癌（EOC）的效果尚不明确。本研究评估了在HR表达分层下，医生选择的化疗联合激素治疗在经多线治疗的晚期EOC患者中的疗效与安全性。 方法：这项II期、多中心、探索性研究纳入了经多线治疗的晚期EOC患者，并将其分配至雌激素受体（ER）主导组或孕激素受体（PR）主导组。ER主导组患者接受他莫昔芬联合医生选择的化疗，而PR主导组患者则接受醋酸甲地孕酮（MA）联合化疗。主要终点为六个月内的最佳客观缓解率（ORR），采用优化的两阶段Simon设计进行评估。 结果：在33例ER主导的高级别浆液性癌（HGSC）患者中，六个月最佳ORR为27.3%（完全缓解3%，部分缓解24.2%）。六个月ORR和临床获益率（CBR）分别为18.8%和37.5%，62.5%的患者出现疾病进展（PD）。在三例PR主导患者（两例透明细胞癌，一例HGSC）中，六个月最佳ORR为0%。六个月ORR和CBR同样为0%，且所有患者均在六个月内出现PD。未观察到与他莫昔芬或MA相关的不可接受毒性。 结论：在ER主导表达的经多线治疗晚期HGSC患者中，化疗联合他莫昔芬显示出令人鼓舞的临床活性和良好的安全性。尽管受研究设计所限，这些发现提示基于HR表达，在经多线治疗的晚期EOC患者中采用个体化激素治疗联合化疗可能具有潜在作用。临床试验注册号：KCT0004571。
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