摘要
库欣综合征(CS)是一种由肾上腺皮质分泌过多糖皮质激素引起的内分泌疾病,其诊断与治疗均具挑战性。诊断过程需结合实验室和影像学检查,进行筛查、定性及定位。治疗方式以手术为主,但药物治疗同样也占据重要地位。随着对CS发病机制的深入了解,发现了更多孤儿药研发的潜在靶点。本文总结了CS的诊疗现状,并对未来的研究方向做出了展望。
Cushing′s syndrome(CS),an endocrine disorder resulting from excessive glucocorticoid secretion by the adrenal cortex,poses significant challenges to both diagnosis and treatment.The diagnostic process involves comprehensive evaluation,combining laboratory tests and imaging studies for screening,qualification,and localization.Surgical intervention remains the primary treatment approach,although pharmacological therapy also plays a crucial role.With an increasing understanding of the pathogenesis of CS,more potential targets for orphan drug development have been discovered.This article summarizes the current status of diagnosis and treatment for CS and provides an outlook on future research directions.
作者
刘岑
赵紫楠
陈頔
胡欣
LIU Cen;ZHAO Zinan;CHEN Di;HU Xin(Department of Pharmacy,Beijing Hospital,National Center of Gerontology,Institute of Geriatric Medicine,Chinese Academy of Medical Sciences,Beijing Key Laboratory of Assessment of Clinical Drugs Risk and Individual Application(Beijing Hospital),Beijing 100730,China;The Key Laboratory of Geriatrics,National Health Commission,Beijing 100730,China)
出处
《罕见病研究》
2024年第2期187-194,共8页
Journal of Rare Diseases
关键词
库欣综合征
诊断
个体化治疗
孤儿药
奥唑司他
帕瑞肽
米非司酮
Cushing′s syndrome
diagnosis
individualized treatment
orphan drugs
osilodrostat
pasireotide
mifepristone