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.
Abstract
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.
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