Front Horm Res. 2010;38:165-173. doi: 10.1159/000318507. Epub 2010 Jul 5.
The goals of ideal medical therapy for Cushing's disease should be to target the aetiology of the disorder, and thus surgery is the current 'gold standard' treatment. However, no effective drug that directly and effectively targets the adrenocorticotropin-secreting pituitary adenoma has been found to date, and treatments to control the hypercortisolaemic state by adrenal-based therapy are frequently used. Inhibitors of adrenal steroidogenesis, adrenolytic agents, compounds with neuromodulatory properties, and ligands of different nuclear hormone receptors involved in hypothalamo-pituitary regulation currently used have been reviewed. Ketoconazole and metyrapone can control hypercortisolaemicstates, as well as mitotane in selective cases, depending on their side effects and frequent monitoring. The somatostatin analogue pasireotide and the dopamine agonist cabergoline, as well as their combination, show some therapeutic promise, while retinoic acid analogues should be further investigated in the pituitary-targeted medical therapy of Cushing's disease. Since a percentage of patients treated with surgery are not cured, or improve and subsequently relapse, there is an urgent need for effective medical therapies for this disorder. At present, only cabergoline and pasireotide are under active investigation, while adrenal steroidogenesis inhibitors are still the mainstay treatments for the control of the hypercortisolaemic state.
库欣病的理想医学治疗目标应该针对疾病的病因，因此手术是目前的“金标准”治疗方法。然而，迄今为止尚未发现能够直接有效针对促肾上腺皮质激素分泌垂体腺瘤的有效药物，因此经常使用基于肾上腺的治疗来控制皮质醇过多状态。目前已对抑制肾上腺类固醇生成、肾上腺溶解剂、具有神经调节特性的化合物以及涉及下丘脑-垂体调节的不同核激素受体的配体进行了综述。酮康唑和米托坦可控制皮质醇过多状态，在某些选择性病例中也可使用美替拉酮，但取决于其副作用和频繁监测。生长抑素类似物帕瑞肽和多巴胺激动剂卡麦角林及其联合用药显示出一定的治疗前景，而视黄酸类似物应在库欣病的垂体靶向医学治疗中进一步研究。由于接受手术治疗的患者中有一定比例未治愈或改善后复发，因此迫切需要针对这种疾病的有效医学治疗方法。目前，只有卡麦角林和帕瑞肽正在积极研究中，而肾上腺类固醇生成抑制剂仍然是控制皮质醇过多状态的主要治疗方法。
