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成功管理从异胎ACTH分泌的库希综合征在一个青少年与osilodrostat成功管理
Kathryn Blew1, David Van Mater2, Laura Page1
1Pediatric Endocrinology & Diabetes, Department of Pediatrics, Duke University, Durham, NC 27705, USA.
JCEM case reports
|November 1, 2023
概括
本案例研究详细介绍了成功的非标签使用的osilodrostat治疗库辛综合征在一个儿科患者与外阴ACTH分泌. 在这种罕见的儿科神经内分泌瘤病例中,治疗有效地使皮质醇水平正常化.
科学领域:
- 儿科内分泌学 儿科内分泌学
- 在瘤学瘤学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 一个罕见的儿科病人的病例呈现胰腺神经内分泌瘤 (世卫组织二级).
- 由于子宫外的ACTH分泌和寡基转移性肝病,该患者患上了耐火性高血压和库辛综合征.
- 之前的治疗包括手术和医疗管理 (基托可纳, metyrapone,lanreotide,cabergoline,spironolactone) 不足以控制库辛综合征.
研究的目的:
- 报告成功非标签使用的奥西洛德罗斯塔特在患有库辛综合征二次性异胎ACTH分泌的儿科患者中.
- 评估奥西洛德罗斯塔特在治疗耐火儿科库辛综合征中的疗效和安全性.
- 突出奥西洛德罗斯塔特作为一种潜在的治疗选择,用于儿科外阴ACTH综合征的病例.
主要方法:
- 一名患有胰腺神经内分泌瘤和库希综合征的儿科患者被用osilodrostat治疗.
- 根据尿液中自由皮质醇水平进行了osilodrostat剂量定位.
- 监测治疗结果,包括皮质醇正常化和不良反应.
主要成果:
- 奥西洛德罗斯塔特治疗导致尿液自由皮质醇水平的快速改善和正常化.
- 患者耐受性很好,没有报告任何不良反应.
- 这项研究表明,在儿科患者中,可以有效控制外阴ACTH诱导的库希综合征.
结论:
- 奥西洛德洛斯塔特是一种可行且有效的非标签治疗,用于治疗外宫ACTH分泌引起的儿科库希综合征.
- 这种病例扩大了罕见的儿科内分泌新发性综合征的治疗选择.
- 进一步研究在儿科群体中使用osilodrostat是有必要的.
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