库欣病的药物治疗进展  

Advances in pharmacological treatment of Cushing’s disease

作　　者：柴金宝 唐宇[1] 刘宇珂 谭惠文[1] 李建薇[1] CHAI Jinbao;TANG Yu;LIU Yuke;TAN Huiwen;LI Jianwei(Department of Endocrinology&Metabolism,West China Hospital,Sichuan University,Chengdu 610041;Department of Endocrinology&Metabolism,Meishan Hospital,West China Hospital,Sichuan University,Meishan Sichuan 620000,China)

机构地区：[1]四川大学华西医院内分泌代谢科,成都610041 [2]四川大学华西医院眉山医院内分泌代谢科,四川眉山620000

出　　处：《中南大学学报（医学版）》2024年第7期1023-1033,共11页Journal of Central South University ：Medical Science

基　　金：四川省科技计划重点研发项目(2023SFY0039)。

摘　　要：库欣病是一种以高皮质醇血症为特点的罕见内分泌疾病。长期高皮质醇水平可引发心血管、糖代谢、骨代谢等功能障碍或并发症,严重影响患者的生活质量甚至危及其生命安全。库欣病首选手术治疗,但在诊断延迟、存在手术禁忌证或手术治疗效果欠佳时,往往需要内科药物治疗。目前常用的药物包括3类:1)作用于垂体生长抑素受体和/或多巴胺受体,抑制垂体促肾上腺皮质激素合成的药物(如帕瑞肽、卡麦角林);2)作用于肾上腺皮质相关受体,抑制类固醇合成的药物(如酮康唑、美替拉酮、米托坦及奥西卓司他等);3)作用于糖皮质激素受体的药物(如米非司酮)。对库欣病药物的研究从未止步,目前seliciclib、CRN04894、fimepinostat、vorinostat、SPI-62等新药处于Ⅱ期临床试验中。期待新型药物的开发能对库欣病患者实现更精准和更有效的治疗。Cushing’s disease is a rare endocrine disorder characterized by hypercortisolism.Chronic elevated cortisol levels can lead to dysfunction or complications in multiple organs of systems,including cardiovascular,glucose,and bone metabolism,severely impacting patients’quality of life and posing life-threatening risks.Surgery is the first-line treatment for Cushing’s disease.However,in cases of delayed diagnosis,surgical contraindications,or suboptimal surgical outcomes,pharmacological therapy is often required.Currently,three categories of medications are commonly used:1)Drugs targeting somatostatin and/or dopamine receptors to inhibit pituitary adrenocorticotropic hormone(ACTH)synthesis(e.g.,pasireotide,cabergoline);2)drugs targeting adrenal cortex-related receptors to suppress steroid synthesis(e.g.,ketoconazole,metyrapone,mitotane,and osilodrostat);3)drugs acting on glucocorticoid receptors(e.g.,mifepristone).Research on pharmacological treatments for Cushing’s disease is ongoing,with new drugs such as seliciclib,CRN04894,fimepinostat,vorinostat,and SPI-62 currently in phase Ⅱ clinical trial.The development of novel drugs holds promise for more precise and effective treatment for Cushing’s disease patients.

关 键 词：库欣病 药物治疗 生长抑素受体配体 多巴胺激动剂 类固醇合成抑制剂 

分 类 号：R586[医药卫生—内分泌]