Dillon Brendan R, Agrawal Nidhi, Schwarz Yair, Dancel-Manning Kristen, Tabarin Antoine, Lacroix André, Hofland Leo J, Feelders Richard A
Holman Division of Endocrinology, Diabetes and Metabolism, Department of Medicine, New York University Langone Medical Center, New York, NY, USA.
Dina Recanati School of Medicine, Reichman University, Herzliyya, Israel.
Drugs. 2025 Sep 15. doi: 10.1007/s40265-025-02223-8.
First-line treatment of endogenous Cushing's syndrome (CS) is surgical removal of the tumor responsible for cortisol excess. However, medical therapy has an established role in treatment when patients are not surgical candidates or decline surgery, residual or recurrent disease is present and not amenable to repeat resection, and control of hypercortisolism is needed either preoperatively or while awaiting the effects of radiotherapy. The approach to medical therapy should be tailored based on the etiology, degree of hypercortisolism, and patient characteristics. Currently available medical therapy for all etiologies of CS either blocks adrenal production of cortisol or blocks its action at the level of the glucocorticoid receptor. Currently available medical therapy for Cushing's disease (CD) targets the adrenocorticotropic hormone-secreting pituitary tumor through activation of somatostatin and dopamine receptors, alkylating DNA damage, or immune system activation. More focused therapy with greater efficacy and fewer adverse effects is needed, particularly in the case of CD, with potential targets and drugs identified and in development.
内源性库欣综合征（CS）的一线治疗方法是手术切除导致皮质醇过量的肿瘤。然而，当患者不适合手术或拒绝手术、存在残留或复发性疾病且不适合再次切除，以及术前或等待放疗效果时需要控制高皮质醇血症时，药物治疗在治疗中具有既定作用。药物治疗方法应根据病因、高皮质醇血症程度和患者特征进行调整。目前可用于治疗所有CS病因的药物治疗，要么阻断肾上腺皮质醇的产生，要么在糖皮质激素受体水平阻断其作用。目前可用于治疗库欣病（CD）的药物治疗通过激活生长抑素和多巴胺受体、烷基化DNA损伤或激活免疫系统来靶向分泌促肾上腺皮质激素的垂体肿瘤。需要更有针对性、疗效更高且副作用更少的治疗方法，特别是在CD的情况下，已确定并正在研发潜在的靶点和药物。
