调查人类附睾蛋白-4（HE4）血清作为子宫内膜癌和非典型子宫内膜增生（AEH）激素反应性的预测性生物标记物。前瞻性预后因素研究。连续受检的样本取自英格兰西北部的三级妇科肿瘤中心的女性。不适合或拒绝原发手术治疗的AEH或早期低级别子宫内膜癌患者。共76名女性，其中32名为AEH患者，44名为子宫内膜癌患者，接受左炔诺孕酮宫内节育系统（LNG-IUS）治疗12个月。进行子宫内膜活检和影像学评估治疗反应。采用化学发光免疫分析分析预处理血清HE4，并进行诊断准确性分析和 logistic 回归分析。12个月内的黄体酮反应，根据组织学和影像学检测结果而定义。最终队列的中位年龄和体重指数（BMI）分别为52岁（四分位距[IQR] 33-62岁）和46kg/m2（IQR 38-54 kg/m2），背景 HE4 血清在对照组和实验组之间有显著差异（119.2 pmol/L，IQR 94.0-208.4 pmol/L 对比 71.8 pmol/L，IQR 56.1-84.2 pmol/L，p <0.001）。年龄较大（比值比率[OR] 0.96，95％ CI 0.93-0.99，p = 0.02）、血清 HE4 水平（OR 0.97，95％ CI 0.96-0.99，p = 0.001）和子宫内膜癌组织学（OR 0.22，95％ CI 0.72-0.68，p = 0.009）与激素治疗反应的成功率呈负相关。调整年龄和组织学，血清 HE4 仍然与激素治疗失败独立相关（调整危险比0.97，95％ CI 0.96-0.99，p = 0.008）。血清 HE4 表现出作为子宫内膜癌和 AEH 激素治疗反应的预测性生物标记物的前景。© 2023 Wiley & Sons Ltd. 发表在《英国妇产科学杂志：国际妇产科杂志》上。

To investigate serum human epididymis-4 (HE4) as a predictive biomarker of intrauterine progestin response in endometrial cancer and atypical endometrial hyperplasia (AEH).Prospective prognostic factor study.Consecutive sample of women attending a tertiary gynaecological oncology centre in northwest England.Women with AEH or early-stage, low-grade endometrial cancer who were unfit for or declined primary surgical management.A total of 76 women, 32 with AEH and 44 with endometrial cancer, were treated with a levonorgestrel intrauterine system (LNG-IUS) for 12 months. Endometrial biopsies and imaging were performed to assess treatment response. Pretreatment serum HE4 was analysed by chemiluminescence immunoassay and diagnostic accuracy and logistic regression analyses were performed.Progestin response at 12 months defined by histology and imaging.The median age and body mass index (BMI) of the final cohort were 52 years (interquartile range [IQR] 33-62 years) and 46 kg/m2 (IQR 38-54 kg/m2 ), respectively. Baseline serum HE4 was significantly higher in non-responders than responders (119.2 pmol/L, IQR 94.0-208.4 pmol/L versus 71.8 pmol/L, IQR 56.1-84.2 pmol/L, p < 0.001). Older age (odds ratio [OR] 0.96, 95% CI 0.93-0.99, p = 0.02), baseline serum HE4 (OR 0.97, 95% CI 0.96-0.99, p = 0.001) and endometrial cancer histology (OR 0.22, 95% CI 0.72-0.68, p = 0.009) were associated with a lower likelihood of progestin treatment response. Serum HE4 remained independently associated with progestin treatment failure when adjusted for age and histology (adjusted hazard ratio 0.97, 95% CI 0.96-0.99, p = 0.008).Serum HE4 shows promise as a predictive biomarker of progestin treatment response in endometrial cancer and AEH.© 2023 The Authors. BJOG: An International Journal of Obstetrics and Gynaecology published by John Wiley & Sons Ltd.