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Evolving Story of Clinical Trials in Hypertrophic Cardiomyopathy
Circulation: Heart Failure ( IF 7.8 ) Pub Date : 2018-01-01 , DOI: 10.1161/circheartfailure.117.004572
Perry M. Elliott 1
Affiliation  

See Article by Olivotto et al

Our greatest glory is not in never falling, but in rising every time we fall.

—Attributed to Confucius

Thousands of papers on hypertrophic cardiomyopathy have been published since Donald Teare’s landmark description of the disease in 1958, but in spite of considerable advances in its management, clinicians still struggle with the treatment of symptoms in people afflicted by the condition. Patients with hypertrophic cardiomyopathy often complain of exertional chest pain, dyspnea, and fatigue.1 The underlying mechanisms are complex and vary between patients; in some, heart failure symptoms are caused by diastolic dysfunction with preserved ejection fraction and in others by systolic left ventricular dysfunction or left ventricular outflow tract obstruction (with or without mitral insufficiency). Atrial fibrillation is frequent in both scenarios and often exacerbates symptoms.

When caused by dynamic left ventricular outflow tract obstruction, exertional symptoms can be managed effectively with drugs or interventions, such as surgical septal myectomy and alcohol septal ablation. However, treatment options in nonobstructive patients are usually much less successful and are often associated with side effects. In this issue of Circulation: Heart Failure, Olivotto et al2 report a multicenter, double-blind, phase 2 study of ranolazine—an inhibitor of the cardiac late sodium current (INaL)—in 80 adult patients with …



中文翻译:

肥厚型心肌病临床试验的发展故事

参见Olivotto等的文章

我们最大的荣耀不是永不落下,而是每次跌倒时都在升起。

-归功于孔子

自从1958年唐纳德·泰尔(Donald Teare)对这种疾病进行具有里程碑意义的描述以来,已经发表了成千上万篇有关肥厚型心肌病的论文,但是尽管在疾病的管理方面取得了长足的进步,临床医生仍在为患有这种疾病的人的症状治疗方面挣扎。肥厚型心肌病患者经常抱怨劳累性胸痛,呼吸困难和疲劳。1潜在的机制很复杂,并且因患者而异;在某些情况下,心力衰竭症状是由舒张功能不全,射血分数保留而引起的,而在另一些情况下,则是由于收缩期的左心功能不全或左心室流出道梗阻(有或没有二尖瓣关闭不全)引起的。在这两种情况下,心房颤动都很频繁,并且常常加剧症状。

由动态左心室流出道梗阻引起的劳累性症状可以通过药物或干预措施来有效地加以管理,例如手术中隔肌切除术和酒精中隔消融术。但是,非阻塞性​​患者的治疗选择通常不太成功,并且常常与副作用相关。在本期《循环:心力衰竭》中,Olivotto等[ 2]报告了雷诺嗪(一种心脏晚期钠电流(I NaL)的抑制剂)在80例成年患者中的多中心,双盲,2期研究。

更新日期:2018-01-17
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