Guignat Laurence, Bertherat Jerome
Department of Endocrinology and National Reference Center for Rare Adrenal Diseases, Hôpital Cochin, Assistance Publique Hôpitaux de Paris, Paris, France.
Genomics and Signaling of Endocrine Tumors, Institut Cochin, INSERM U1016, CNRS UMR 8104, Université Paris Cité, Paris, France.
Endocrinol Metab (Seoul). 2025 Feb;40(1):26-38. doi: 10.3803/EnM.2024.501. Epub 2025 Jan 13.
Endogenous Cushing's syndrome (CS) refers to the manifestations of chronic cortisol excess. This rare disease is associated with multiple comorbidities, impaired quality of life, and increased mortality. The management of CS remains challenging. Regardless of the underlying cause, surgical resection of the tumor is typically the first-line and preferred treatment. However, when surgery is not feasible or has been unsuccessful, medical therapies may be employed to control CS. The therapeutic strategy should be individualized based on the recommendations of a multidisciplinary team of experts and the patient's preferences, informed by detailed information on the available options. All medications require careful monitoring, along with adequate instructions for patients and caregivers. The aim of this mini-review is to provide an overview of the main medical therapies currently used to treat CS, including their efficacy, safety, and management. Despite the availability of new drugs in recent years, the need remains for more effective specific targeted pharmacological therapies.
内源性库欣综合征(CS)是指慢性皮质醇过量的表现。这种罕见疾病与多种合并症、生活质量受损及死亡率增加相关。CS的治疗仍然具有挑战性。无论潜在病因如何,肿瘤的手术切除通常是一线且首选的治疗方法。然而,当手术不可行或未成功时,可采用药物治疗来控制CS。治疗策略应根据多学科专家团队的建议和患者的偏好进行个体化制定,并参考有关可用选项的详细信息。所有药物都需要仔细监测,同时要为患者和护理人员提供充分的指导。本综述的目的是概述目前用于治疗CS的主要药物治疗方法,包括其疗效、安全性和管理。尽管近年来有新药问世,但仍需要更有效的特异性靶向药物治疗。