拉帕米辛和拉帕洛格用于结核性硬化综合体
Teguh Haryo Sasongko1,2, Kumaraswamy Kademane3, Stanley Chai Soon Hou4
1Department of Physiology, School of Medicine, International Medical University, Kuala Lumpur, Malaysia.
The Cochrane database of systematic reviews
|July 11, 2023
概括
这份综述发现,口服Everolimus有效治疗状硬化综合体 (TSC) 症状,如瘤和发作,而局部Rapamycin改善皮肤病变. 两种治疗都显示了增加的不良事件,需要调整剂量.
科学领域:
- 药理学和治疗学 药理学和治疗学
- 遗传学和疾病机制
- 临床试验和证据综合
背景情况:
- 结核性硬化综合体 (TSC) 是一种具有各种表现的遗传性疾病.
- 埃弗罗利斯 (一种拉帕洛格) 已被批准用于特定的TSC相关疾病,但需要其他表现的证据.
- 本系统性审查提供了对拉帕米辛和拉帕洛格在TSC中的疗效的最新分析.
研究的目的:
- 评估拉帕米辛和拉帕洛格在减少瘤大小和其他TSC症状方面的有效性.
- 评估拉帕米辛和拉帕洛格在TSC患者的安全性和不良影响.
- 综合来自各种TSC表现的随机对照试验 (RCT) 的证据.
主要方法:
- 随机对照试验 (RCT) 和准RCT的系统审查.
- 在主要数据库 (CENTRAL,MEDLINE) 和试验登记处进行搜索,截至2022年7月.
- 数据提取和偏差风险评估由两个评审人独立进行,以GRADE确定性评估.
主要成果:
- 口服Everolimus显著降低了血管瘤和SEGA瘤大小,减少了发作频率,改善了皮肤病变.
- 局部拉巴胺在治疗皮肤病变和面部血管纤维瘤方面表现出有效性,改善了患者的满意度.
- 虽然总不良事件 (AE) 没有显著差异,但口服和局部治疗都增加了需要剂量修改或取消的AE的风险.
结论:
- 口服Everolimus有助于治疗TSC的血管瘤,SEGA,发作和皮肤病变.
- 建议在TSC中治疗面部血管纤维瘤时使用局部拉巴胺素.
- 建议注意潜在的不良事件,特别是那些需要治疗调整的不良事件.
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