Division of Cardiology, Department of Medicine, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA.
Sanger Heart and Vascular Institute, Atrium Health, Charlotte, North Carolina, USA.
Open Heart. 2021 Feb;8(1). doi: 10.1136/openhrt-2020-001560.
Despite advances in our understanding of the genetic causes of hypertrophic cardiomyopathy (HCM), a large portion of this patient population do not carry sarcomere gene mutations when screened. It remains largely unknown why patients without sarcomere mutations develop asymmetric myocardial hypertrophy.
We performed a retrospective analysis of probands with HCM who underwent genetic testing to determine if clinical phenotypes were different depending on sarcomere mutation status. A medical history, three generation family history and clinical phenotyping were performed on 127 probands with HCM. Genetic screening was performed using clinically available HCM genetic testing panels.
We found that probands with HCM with pathogenic sarcomere mutations were over three times more likely to have a family history of HCM (66% vs 17%, p<0.0001) and were diagnosed with HCM at a much younger age (32 vs 51 years old, p<0.0001). In contrast, probands with HCM without sarcomere mutations were significantly more obese (body surface area p=0.003, body mass index p=0.04 adjusted for age) and were more likely to present with left ventricular outflow tract obstruction (p=0.0483).
Patients with sarcomere mutation negative HCM present at an older age and are more obese compared with patients with sarcomere mutation positive HCM. The role of ageing and obesity in asymmetric myocardial hypertrophy warrants further investigation.
尽管我们对肥厚型心肌病(HCM)的遗传病因有了更多的了解,但当进行筛查时,仍有很大一部分患者没有携带肌节基因突变。目前尚不清楚为什么没有肌节突变的患者会发展为不对称性心肌肥厚。
我们对接受基因检测以确定是否存在肌节突变状态不同的临床表型的 HCM 先证者进行了回顾性分析。对 127 名 HCM 先证者进行了病史、三代家族史和临床表型检查。使用临床可用的 HCM 基因检测面板进行基因筛查。
我们发现,携带致病性肌节突变的 HCM 先证者发生 HCM 家族史的可能性高出三倍以上(66%比 17%,p<0.0001),且发病年龄更小(32 岁比 51 岁,p<0.0001)。相比之下,肌节基因突变阴性的 HCM 先证者明显更肥胖(体表面积 p=0.003,校正年龄后的体重指数 p=0.04),且更可能出现左心室流出道梗阻(p=0.0483)。
与肌节突变阳性的 HCM 患者相比,肌节突变阴性的 HCM 患者发病年龄更大,且更肥胖。年龄和肥胖在不对称性心肌肥厚中的作用值得进一步研究。