PDF(Pubmed)
Abstract：
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.
摘要：
Osilodrostat是一种新型有效的口服类固醇生成抑制剂，具有非甾体化学结构，最近批准用于治疗内源性库欣综合征的成人患者，和库欣的疾病不能通过垂体手术治愈，或者垂体手术不是一种选择。Osilodrostat已在不同的多中心II期和III期临床研究中进行了评估，并显示出显著的效果，比如皮质醇分泌的显著减少，与体重的显着改善有关，血压,葡萄糖代谢，血脂谱,心理状况和生活质量。有利的安全状况，结合相关疗效，可以在库欣综合征治疗旅程的几个阶段使osilodrostat适合作为药物治疗：手术前，作为术前治疗,或者代替手术，在手术不是选择或拒绝的情况下，作为一线治疗；手术后，在持续性或复发性疾病的情况下，作为二线治疗；在垂体手术后的第二次手术或放疗作为桥接治疗后等待明确的疾病控制，作为三线治疗。需要进一步的真实世界临床经验数据来确认当前的知识。
