对BCG不响应的NMIBC基因介导疗法的系统性审查:新兴证据和未来的前景
Constance Bertrand1, Idir Ouzaid1, Felix Guerrero-Ramos2,3,4
1Department of Urology, Bichat Claude-Bernard Hospital, Assistance Publique-Hôpitaux de Paris Nord, University Paris Cité, Paris, France.
World journal of urology
|December 15, 2025
概括
基因疗法为BCG不响应的非肌肉侵入性膀癌 (NMIBC) 提供了一个有前途的膀节省选择,显示出高响应率和有利的安全性.
科学领域:
- 在瘤学瘤学.
- 泌尿器科 泌尿器科 泌尿器科 泌尿器科
- 基因治疗 基因治疗
背景情况:
- 对BCG无反应的非肌肉侵入性膀癌 (NMIBC) 是一个重大的临床挑战.
- 对于患有高进展风险的患者,存在有限的膀节省选择.
研究的目的:
- 系统地审查BCG不响应的NMIBC基因疗法的瘤学证据,安全性和未来前景.
- 评估基因介导疗法作为一个节约膀的策略.
主要方法:
- 在Medline和谷歌学者的系统文献综述.
- 选了3157个记录,包括13个相关引用 (8个已发表,5个未发表).
主要成果:
- 完全响应率在3个月后从53.4%到82.9%,在12个月后从24.4%到57.1%不等.
- 响应的平均持续时间为9.7至28个月. 总体不良事件为47%-91%,其中4%-14%为等级≥3.3.
- 预计FDA对Nadofaragene firadenovec的批准将产生真实世界的数据,尽管患者的选择和成本仍然是挑战.
结论:
- 基因疗法对BCG不响应的NMIBC显示出显著的疗效和有利的安全概况.
- 新兴的基因疗法代表了新且有前途的膀保护方法.
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