极低剂量依维莫司治疗可减少结节性硬化症的心脏肿瘤。

IF 0.7 4区医学 Q4 CARDIAC & CARDIOVASCULAR SYSTEMS

Cardiology in the Young Pub Date : 2025-08-01 Epub Date: 2025-08-20 DOI:10.1017/S1047951125101650

Stefan Rupp, Christian Jux, David Backhoff

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引用次数: 0

摘要

结节性硬化症是一种累及多个器官的综合征。心脏表现包括横纹肌瘤，可导致心内血流阻塞。在本病例中，迄今为止记录的依维莫司最低血药浓度为2-3 ng/ml，导致肿瘤消退。因临床原因反复停用和重新使用依维莫司作为依维莫司治疗结节性硬化症的概念证明。

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Very low-dose Everolimus therapy diminishes cardiac tumours in tuberous sclerosis complex disease.

Tuberous sclerosis complex is syndrome that affects several organs. Cardiac manifestations include rhabdomyoma, which could lead to intracardiac obstruction of blood flow. In the present case, the so far lowest documented Everolimus blood level of 2-3 ng/ml led to tumour regression. Repeated Everolimus stopping and restarting for clinical reasons serves as a proof-of-concept for Everolimus therapy in tuberous sclerosis complex.

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来源期刊

Cardiology in the Young 医学-小儿科

CiteScore

1.70

自引率

10.00%

发文量

715

审稿时长

4-8 weeks

期刊介绍： Cardiology in the Young is devoted to cardiovascular issues affecting the young, and the older patient suffering the sequels of congenital heart disease, or other cardiac diseases acquired in childhood. The journal serves the interests of all professionals concerned with these topics. By design, the journal is international and multidisciplinary in its approach, and members of the editorial board take an active role in the its mission, helping to make it the essential journal in paediatric cardiology. All aspects of paediatric cardiology are covered within the journal. The content includes original articles, brief reports, editorials, reviews, and papers devoted to continuing professional development.