BACKGROUND The VANISH trial (Valsartan for Attenuating Disease Evolution in Early Sarcomeric Hypertrophic Cardiomyopathy) targeted young sarcomeric gene mutation carriers with early-stage hypertrophic cardiomyopathy (HCM) to test whether valsartan can modify disease progression. We describe the baseline characteristics of the VANISH cohort and compare to previous trials evaluating angiotensin receptor blockers. METHODS Applying a randomized, double-blinded, placebo-controlled design, 178 participants with nonobstructive HCM (age, 23.3±10.1 years; 61% men) were randomized in the primary cohort and 34 (age, 16.5±4.9 years; 50% men) in the exploratory cohort of sarcomeric mutation carriers without left ventricular hypertrophy. RESULTS In the primary cohort, maximal left ventricular wall thickness was 17±4 mm for adults and Z score 7.0±4.5 for children. Nineteen percent had late gadolinium enhancement on cardiac magnetic resonance. Mean peak oxygen consumption was 33 mL/kg per minute, and 92% of participants were New York Heart Association functional class I. New York Heart Association class II was associated with older age, MYH7 variants, and more prominent imaging abnormalities. Six previous trials of angiotensin receptor blockers in HCM enrolled a median of 24 patients (range, 19-133) with mean age of 51.2 years; 42% of patients were in New York Heart Association class ≥II, and sarcomeric mutations were not required. CONCLUSIONS The VANISH cohort is much larger, younger, less heterogeneous, and has less advanced disease than prior angiotensin receptor blocker trials in HCM. Participants had relatively normal functional capacity and mild HCM features. New York Heart Association functional class II symptoms were associated with older age, more prominent imaging abnormalities, and MYH7 variants, suggesting both phenotype and genotype contribute to disease manifestations. CLINICAL TRIAL REGISTRATION URL: https://www.clinicaltrials.gov. Unique identifier: NCT01912534.

中文翻译：

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VANISH队列的基线特征。

背景VANISH试验（缬沙坦用于减轻早期肌节型肥大性心肌病的疾病发展）针对具有早期肥厚型心肌病（HCM）的年轻肌节基因突变携带者，以测试缬沙坦是否可以改变疾病进展。我们描述了VANISH队列的基线特征，并与以前评估血管紧张素受体阻滞剂的试验进行了比较。方法采用随机，双盲，安慰剂对照设计，将178例无阻塞性HCM参与者（年龄23.3±10.1岁；男性61％）随机分为原发性队列，将34名参与者（年龄16.5±4.9岁）随机性）在无左心室肥大的肌节突变携带者的探索性队列中。结果在主要队列中，成年人的最大左心室壁厚度为17±4 mm，Z评分为7.0±4。5个孩子。19％的g在心脏磁共振上具有晚期enhancement增强作用。平均峰值耗氧量为每分钟33 mL / kg，92％的参与者为纽约心脏协会I级功能。纽约心脏协会II级与年龄较大，MYH7变异以及更显着的影像异常有关。先前有6项HCM中血管紧张素受体阻滞剂试验纳入了24名患者（范围19-133），平均年龄51.2岁。42％的患者属于纽约心脏协会II级以上，并且不需要肌节突变。结论与HCM中以前的血管紧张素受体阻滞剂试验相比，VANISH队列更大，更年轻，异质性更低，疾病进展更短。参与者具有相对正常的功能能力和轻度的HCM特征。纽约心脏协会的功能性II类症状与年龄较大，影像异常突出和MYH7变异有关，表明表型和基因型均与疾病表现有关。临床试验注册网址：https：//www.clinicaltrials.gov。唯一标识符：NCT01912534。