当前位置: X-MOL 学术Best Pract. Res. Clin. Endocrinol. Metab. › 论文详情
Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)
Updates in Adrenal Steroidogenesis Inhibitors for Cushing’s syndrome – a Practical Guide
Best Practice & Research Clinical Endocrinology & Metabolism ( IF 6.1 ) Pub Date : 2021-02-06 , DOI: 10.1016/j.beem.2021.101490
Elena V Varlamov 1 , Ashley J Han 2 , Maria Fleseriu 1
Affiliation  

Medical therapy is essential in the management of patients with Cushing’s syndrome (CS) when curative surgery has failed, surgery is not feasible, when awaiting radiation effect, and in recurrent cases of CS. Steroidogenesis inhibitors have a rapid onset of action and are effective in reducing hypercortisolism, however, adverse effects, including adrenal insufficiency require very close patient monitoring. Osilodrostat is the only steroidogenesis inhibitor to have been assessed in prospective randomized controlled trials and approved for Cushing’s disease (CD) by the US Food and Drug Administration and for CS by the European Medical Agency (EMA). Osilodrostat has been shown to be highly effective at maintaining normal urinary free cortisol in patients with CD. Drugs such as metyrapone, ketoconazole (both EMA approved), and etomidate lack prospective evaluation(s). There is, however, considerable clinical experience and retrospective data that show a very wide efficacy range in treating patients with CS. In the absence of head-to-head comparative clinical trials, therapy choice is determined by the specific clinical setting, risk of adverse events, cost, availability, and other factors. In this review practical points to help clinicians who are managing patients with CS being treated with steroidogenesis inhibitors are presented.



中文翻译:

库欣氏综合征肾上腺类固醇生成抑制剂的更新-实用指南

当根治性手术失败,手术不可行,等待放疗时以及复发的CS患者中,药物治疗对于库欣综合征(CS)的治疗至关重要。类固醇生成抑制剂起效迅速,对减少皮质醇过多有效,但是,包括肾上腺功能不全在内的不良反应需要非常密切的患者监测。Osilodrostat是唯一在前瞻性随机对照试验中评估的类固醇生成抑制剂,并已获得美国食品和药物管理局(US Food and Drug Administration)批准用于库欣病(CD)和被欧洲医学机构(EMA)批准用于CS。已经证明,osilodrostat在维持CD患者的正常尿液游离皮质醇方面非常有效。甲吡酮,酮康唑(均获EMA批准),和依托咪酯缺乏前瞻性评估。但是,有大量的临床经验和回顾性数据显示,治疗CS患者的疗效范围非常广泛。在没有进行头对头比较临床试验的情况下,治疗的选择取决于具体的临床背景,不良事件的风险,成本,可用性和其他因素。在这篇综述中,提出了一些实用的要点,以帮助正在使用类固醇生成抑制剂治疗CS患者的临床医生。和其他因素。在这篇综述中,提出了一些实用的要点,以帮助正在使用类固醇生成抑制剂治疗CS患者的临床医生。和其他因素。在这篇综述中,提出了一些实用的要点,以帮助正在使用类固醇生成抑制剂治疗CS患者的临床医生。

更新日期:2021-02-07
down
wechat
bug