扩展MPOWERED试验的开放标签:长期有效性和安全性数据,用于巨的口服片
Maria Fleseriu1, Mark Molitch2, Alexander Dreval3
1Pituitary Center, Oregon Health & Science University, Portland, OR 97239, USA.
The Journal of clinical endocrinology and metabolism
|June 15, 2023
概括
长期使用口服八胺囊 (OOC) 在壮病患者中维持了治疗反应和安全性. 回到OOC的过渡改善了患者的便利性和症状控制,证明了其长期的有效性.
科学领域:
- 内分泌学 在内分泌学.
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 巨症是一种罕见的内分泌疾病,由多余的生长激素引起.
- 可注射的体静止素受体配体 (iSRLs) 是标准的治疗方法,但口服八胺囊 (OOC) 提供了一个替代方案.
- 该MPOWERED试验调查了OOC.的长期疗效和安全性.
研究的目的:
- 评估OOC在壮病患者的长期疗效和安全性.
- 评估患者在OOC和iSRLs之间过渡后报告的结果.
- 确认OOC在开放式扩展阶段的持续反应和安全性.
主要方法:
- 这项研究包括了完成MPOWERED核心试验并接受OOC.的患者.
- 一个独特的设计允许通过OOC和iSRLs之间的过渡进行患者内部比较.
- 每年监测生物化学反应 (IGF-I水平) 和安全性.
主要成果:
- 在整个延长阶段,高响应率保持在高水平:第1年为89.7%,第2年为87.8%,第3年为93.5%.
- 没有发现新的安全问题.
- 从iSRLs过渡到OOC的患者报告了改善的便利性和症状控制.
结论:
- 口服八胺囊 (OOC) 提供长期维护疗效和持续的安全性概况在壮症.
- 将患者转移到OOC可以改善治疗方便性和症状管理.
- 患者报告的结果支持OOC作为有效的长期治疗选择.
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