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国际现实世界研究上腺库辛综合征中西洛德罗斯塔特的疗效和安全性
Marta Araujo-Castro1,2, Irina Bancos3, Mario Detomas4
1Endocrinology & Nutrition Department. Hospital Universitario Ramón y Cajal Madrid, Spain.
The Journal of clinical endocrinology and metabolism
|March 13, 2026
概括
在上腺库辛综合征 (CS) 中,奥西洛德罗斯塔特有效控制高皮质醇,改善血压和体重. 之前接受其他抑制剂治疗的患者对osilodrostat的反应率更高.
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.
- 在瘤学瘤学.
背景情况:
- 上腺库辛综合征 (CS) 是一种罕见的内分泌疾病,其特征是皮质醇的过度产生.
- 目前对上腺癌的治疗有局限性,需要新的治疗选择.
研究的目的:
- 评估奥西洛德罗斯塔特在患有上腺骨瘤的患者的疗效和安全性.
- 为了确定响应治疗的预测因子.
主要方法:
- 一项国际研究招募了28名上腺癌患者 (16名上腺皮癌,12名良性疾病).
- 在21名接受超过4周治疗的患者中评估了疗效,反应者定义为尿液自由皮质醇 (UFC) 减少>50%.
- 在整个研究过程中,对安全性和不良事件进行了监测.
主要成果:
- 超过4周的奥西洛德罗斯塔特治疗导致66.7%的应答率 (28.6%完全,38.1%部分).
- 在12周治疗的患者中,应答率增加到87.5%.
- 作为非第一线疗法 (OR 15.0,P=.010) 的奥西洛德罗斯塔特使用预测了反应,并显著降低了缩血压和体重.
结论:
- 在大多数上腺癌患者中,奥西洛德罗斯塔特有效控制高皮质醇症.
- 用奥西洛德罗斯塔特治疗对血压和体重产生积极影响.
- 以往使用类固醇生成抑制剂的治疗与对osilodrostat的反应更高的可能性有关.
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