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.
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。.
Keywords: Cushing’s disease; dopamine agonist; pharmacological treatment; somatostatin receptor ligand; steroidogenesis inhibitor.