Division of Endocrinology, Department of Internal Medicine, Erasmus Medical Center, Rotterdam, The Netherlands.
Endocr Relat Cancer. 2012 Nov 9;19(6):R205-23. doi: 10.1530/ERC-12-0191. Print 2012 Dec.
Cushing's syndrome (CS) is a severe endocrine disorder characterized by chronic cortisol excess due to an ACTH-secreting pituitary adenoma, ectopic ACTH production, or a cortisol-producing adrenal neoplasia. Regardless of the underlying cause, untreated CS is associated with considerable morbidity and mortality. Surgery is the primary therapy for all causes of CS, but surgical failure and ineligibility of the patient to undergo surgery necessitate alternative treatment modalities. The role of medical therapy in CS has been limited because of lack of efficacy or intolerability. In recent years, however, new targets for medical therapy have been identified, both at the level of the pituitary gland (e.g. somatostatin, dopamine, and epidermal growth factor receptors) and the adrenal gland (ectopically expressed receptors in ACTH-independent macronodular adrenal hyperplasia). In this review, results of preclinical and clinical studies with drugs that exert their action through these molecular targets, as well as already established medical treatment options, will be discussed.
库欣综合征(CS)是一种严重的内分泌疾病,其特征为慢性皮质醇过多,病因可能为 ACTH 分泌性垂体腺瘤、异位 ACTH 产生或皮质醇产生的肾上腺肿瘤。无论病因如何,未经治疗的 CS 与相当大的发病率和死亡率相关。手术是治疗 CS 的所有病因的主要方法,但手术失败和患者不适宜手术需要替代治疗方法。由于疗效或不耐受性,医学治疗在 CS 中的作用受到限制。然而,近年来,已经确定了新的医学治疗靶点,包括垂体(例如生长抑素、多巴胺和表皮生长因子受体)和肾上腺(ACTH 非依赖性大结节性肾上腺增生中外源表达的受体)水平。在这篇综述中,将讨论通过这些分子靶点发挥作用的药物以及已确立的医学治疗选择的临床前和临床研究结果。