对内源性库辛综合征 (PROMPT) 中 metyrapone 的前性研究
Lynnette K Nieman1, Marco Boscaro2, Carla Scaroni2
1National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Building 10, CRC, 1 East, Rm 1-3140, 10 Center Dr, MSC 1109, Bethesda, MD 20892-1109, United States.
European journal of endocrinology
|September 18, 2025
概括
梅西拉通过使许多患者的皮质醇水平正常化,有效治疗库辛综合征 (CS). 虽然恶心和疲劳等副作用很常见,但治疗通常会改善临床特征和生活质量.
科学领域:
- 内分泌学 在内分泌学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 库辛综合征 (Cushing's syndrome,简称CS) 是一种罕见的内分泌疾病,由长时间暴露于过多的皮质醇引起.
- 内源性CS的治疗选择包括手术,放射和药物治疗,特别是对于不适合手术或手术后无法控制的患者.
研究的目的:
- 评估 metyrapone 在治疗内源性库辛综合征的安全性和有效性.
- 评估 metyrapone 对尿液自由皮质醇 (UFC) 水平,临床特征和生活质量 (QoL) 的影响.
主要方法:
- 一项国际,前性,单臂,开放标签研究,涉及50名成年人内源性CS.
- 梅西拉治疗涉及12周的定位,然后可能进行24周的延长阶段,并定期评估安全性和有效性.
- 终点包括平均24小时UFC (mUFC) 的正常化,响应率,eucortisolemia的时间和QoL评估.
主要成果:
- 在12周内,46.9%的参与者达到mUFC正常化的主要终点,有效性维持到36周.
- 观察到高响应率 (基线mUFC≥50%降低):第12周80.9%,第24周77.5%,第36周71.4%.
- 大多数参与者 (94%) 经历了轻度至中度的不良事件 (恶心,疲劳,头痛);8人患有严重的不良事件,6人患有可逆性上腺功能衰竭.
结论:
- 梅西拉在内源性CS患者的皮质醇水平正常化方面表现出显著的有效性.
- 治疗通常是安全的,可控不良事件和改善临床特征和QOL.
- 梅西拉代表了内源性CS的有价值的治疗选择,特别是当手术不可行或不有效时.
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