埃普隆特森对遗传性转基因氨基化症患者心脏结构和功能的影响
Ahmad Masri1, Mathew S Maurer2, Brian L Claggett3
1Oregon Health & Science University, Portland, Oregon.
Journal of cardiac failure
|December 8, 2023
概括
埃普隆特森在患有心肌病的遗传性转基因氨基粉症 (ATTRv) 患者中改善了心脏功能. 该研究显示,与安慰剂相比,射出分数和中风体积有所改善.
科学领域:
- 心脏病学 心脏病学
- 神经学 神经学
- 药理学 药理学是指药理学的学科.
背景情况:
- 遗传性转基因氨基粉症 (ATTRv) 可以影响心脏和神经.
- 埃普隆特森是一种潜在的ATTRv治疗方法.
研究的目的:
- 评估EPLONTERSEN在患有ATTRv多神经病变的患者的心脏影响.
- 评估eplontersen对心脏结构和功能的影响.
主要方法:
- 开放标签的NEURO-TTR转换试验包括144名ATTRv患者接受eplontersen.
- 与65周内NEURO-TTR试验中的历史安慰剂组进行比较.
- 对多个基线和人口统计因素进行调整的分析.
主要成果:
- 在心肌病小组中,eplontersen使左心室喷射率提高了4.3% (P=.049).
- 与安慰剂相比,Eplontersen增加了10.64毫升 (P=.002) 的中风体积.
- 其他心声回声学参数保持稳定.
结论:
- 在ATTRv心肌病患者中,Eplontersen表明心脏结构和功能稳定或改善.
- 在CARDIO-TTRansform试验中,对 transthyretin amyloid心肌病的eplontersen的进一步研究正在进行中.
更多相关视频
08:03Hybrid Cell Analysis System to Assess Structural and Contractile Changes of Human iPSC-Derived Cardiomyocytes for Preclinical Cardiac Risk Evaluation
Published on: October 20, 2022
1.8K
12:45Benefits of Cardiac Resynchronization Therapy in an Asynchronous Heart Failure Model Induced by Left Bundle Branch Ablation and Rapid Pacing
Published on: December 11, 2017
10.5K
相关概念视频
01:18Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
170
Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
170
01:26Heart Failure Drugs: Inhibitors of Renin-Angiotensin System
436
The activation of the sympathetic nervous system and the renin-angiotensin-aldosterone system (RAAS) contributes to cardiac remodeling, and inhibiting the RAAS is a pharmacological target in heart failure management. As a result, neurohumoral modulation is a crucial treatment principle for managing heart failure. This approach involves using medications like ACE inhibitors (ACEIs), angiotensin receptor blockers (ARBs), β-blockers, mineralocorticoid receptor antagonists (MRAs), and neutral...
436