奥西洛司他用于治疗库欣综合征的新型强效 11-β-羟化酶抑制剂。

TouchREVIEWS in endocrinology Pub Date : 2024-04-01 Epub Date: 2023-12-11 DOI:10.17925/EE.2024.20.1.8
Rosario Pivonello, Chiara Simeoli, Nicola Di Paola, Angelica Larocca, Erminio Massimo Crescenzo, Annamaria Colao
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引用次数: 0

摘要

奥西洛德司他是一种新型强效口服类固醇生成抑制剂,具有非类固醇化学结构,最近被批准用于治疗内源性库欣综合征、垂体手术无法治愈或无法选择垂体手术的库欣病患者。奥西洛德司他已在不同的多中心 II 期和 III 期临床研究中进行了评估,结果表明其疗效显著,如皮质醇分泌显著减少,体重、血压、糖代谢、血脂、心理状态和生活质量均有明显改善。奥西洛德司他具有良好的安全性和相关疗效,可在库欣综合征治疗的多个阶段作为药物治疗:手术前,作为术前治疗,或在不能选择或拒绝手术的情况下替代手术,作为一线治疗;手术后,在疾病持续或复发的情况下,作为二线治疗;垂体手术后第二次手术或放疗后,作为等待疾病最终控制的过渡治疗,作为三线治疗。需要更多真实世界的临床经验数据来证实当前的知识。
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Osilodrostat: A Novel Potent Inhibitor of 11-Beta-Hydroxylase for the Treatment of Cushing's Syndrome.

Osilodrostat is a novel potent oral steroidogenesis inhibitor with a non-steroidal chemical structure, recently approved for the treatment of adult patients with endogenous Cushing's syndrome, and Cushing's disease not cured bytab pituitary surgery or in whom pituitary surgery is not an option. Osilodrostat has been evaluated in different multicentre phase II and III clinical studies, and has shown to have notable effects, such as significant reductions in cortisol secretion, associated with significant improvement in body weight, blood pressure, glucose metabolism, lipid profile, psychological status and quality of life. The favourable safety profile, combined with the relevant efficacy, could make osilodrostat suitable as medical treatment in several phases of the Cushing's syndrome treatment journey: before surgery, as preoperative treatment, or instead of surgery, in cases where surgery is not an option or refused, as first-line treatment; after surgery, in cases of persistent or recurrent disease, as second-line treatment; after second surgery or radiotherapy following pituitary surgery as bridging treatment waiting for the definitive disease control, as third-line treatment. Further real-world clinical experience data are needed to confirm the current knowledge.

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