Thiene Gaetano, Calore Chiara, De Gaspari Monica, Basso Cristina
Department of Cardiac, Thoracic, Vascular Sciences and Public Health, University of Padua Medical School, 35121 Padova, Italy.
J Cardiovasc Dev Dis. 2024 Sep 28;11(10):300. doi: 10.3390/jcdd11100300.
The discovery of hypertrophic cardiomyopathy (HCM) dates back to 1958, when the pathologist Donald Teare of the St. George's Hospital in London performed autopsies in eight cases with asymmetric hypertrophy of the ventricular septum and bizarre disorganization (disarray) at histology, first interpreted as hamartoma. Seven had died suddenly. The cardiac specimens were cut along the long axis, similar to the 2D echo. In the same year, at the National Institute of Health U.S.A., Eugene Braunwald, a hemodynamist, and Andrew Glenn Morrow, a cardiac surgeon, clinically faced a patient with an apparently similar morbid entity, with a systolic murmur and subaortic valve gradient. "Discrete" subaortic stenosis was postulated. However, at surgery, Dr. Morrow observed only hypertrophy and performed myectomy to relieve the obstruction. This first Braunwald-Morrow patient underwent a successful cardiac transplant later at the disease end stage. The same Dr. Morrow was found to be affected by the familial HCM and died suddenly in 1992. The term "functional subaortic stenosis" was used in 1959 and "idiopathic hypertrophic subaortic stenosis" in 1960. Years before, in 1957, Lord Brock, a cardiac surgeon at the Guy's Hospital in London, during alleged aortic valve surgery in extracorporeal circulation, did not find any valvular or discrete subaortic stenoses. In 1980, John F. Goodwin of the Westminster Hospital in London, the head of an international WHO committee, put forward the first classification of heart muscle diseases, introducing the term cardiomyopathy (dilated, hypertrophic, and endomyocardial restrictive). In 1995, the WHO classification was revisited, with the addition of two new entities, namely arrhythmogenic and purely myocardial restrictive, the latter a paradox of a small heart accounting for severe congestive heart failure by ventricular diastolic impairment. A familial occurrence was noticed earlier in HCM and published by Teare and Goodwin in 1960. In 1989-1990, the same family underwent molecular genetics investigation by the Seidman team in Boston, and a missense mutation of the β-cardiac myosin heavy chain in chromosome 14 was found. Thus, 21 years elapsed from HCM gross discovery to molecular discoveries. The same original family was the source of both the gross and genetic explanations of HCM, which is now named sarcomere disease. Restrictive cardiomyopathy, characterized grossly without hypertrophy and histologically by myocardial disarray, was found to also have a sarcomeric genetic mutation, labeled "HCM without hypertrophy". Sarcomere missense mutations have also been reported in dilated cardiomyopathy (DCM) and non-compaction cardiomyopathy. Moreover, sarcomeric gene defects have been detected in some DNA non-coding regions of HCM patients. The same mutation in the family may express different phenotypes (HCM, DCM, and RCM). Large ischemic scars have been reported by pathologists and are nowadays easily detectable in vivo by cardiac magnetic resonance with gadolinium. The ischemic arrhythmic substrate enhances the risk of sudden death.
肥厚型心肌病(HCM)的发现可追溯到1958年,当时伦敦圣乔治医院的病理学家唐纳德·蒂尔对8例室间隔不对称肥厚且组织学上有奇异紊乱(排列紊乱)的病例进行了尸检,最初被解释为错构瘤。其中7例猝死。心脏标本沿长轴切开,类似于二维超声心动图。同年,在美国国立卫生研究院,血液动力学家尤金·布劳恩瓦尔德和心脏外科医生安德鲁·格伦·莫罗临床上遇到了一名患有明显类似病态实体的患者,有收缩期杂音和主动脉瓣下梯度。推测为“离散性”主动脉瓣下狭窄。然而,在手术中,莫罗医生仅观察到肥厚,并进行了心肌切除术以解除梗阻。这位首位布劳恩瓦尔德 - 莫罗患者在疾病终末期后来接受了成功的心移植手术。发现同一位莫罗医生患有家族性HCM,并于1992年猝死。1959年使用了“功能性主动脉瓣下狭窄”一词,1960年使用了“特发性肥厚性主动脉瓣下狭窄”一词。早在1957年,伦敦盖伊医院的心脏外科医生布罗克勋爵在所谓的体外循环主动脉瓣手术中,未发现任何瓣膜或离散性主动脉瓣下狭窄。1980年,伦敦威斯敏斯特医院的约翰·F·古德温,作为世界卫生组织一个国际委员会的负责人,提出了心肌疾病的首个分类,引入了心肌病(扩张型、肥厚型和心内膜限制性)这一术语。1995年,对世界卫生组织的分类进行了修订,增加了两个新的实体,即致心律失常性和单纯心肌限制性,后者是一种心脏小却因心室舒张功能障碍导致严重充血性心力衰竭的矛盾情况。HCM中更早注意到有家族性发病情况,并由蒂尔和古德温于1960年发表。1989 - 1990年,同一个家族在波士顿由塞德曼团队进行了分子遗传学研究,发现了14号染色体上β - 心脏肌球蛋白重链的一个错义突变。因此,从HCM的大体发现到分子发现历经了21年。同一个原始家族是HCM大体和遗传学解释的来源,HCM现在被称为肌节病。限制性心肌病,大体上无肥厚特征,组织学上有心肌排列紊乱,也被发现有肌节基因突变,标记为“无肥厚的HCM”。在扩张型心肌病(DCM)和非致密化心肌病中也报道了肌节错义突变。此外,在HCM患者的一些DNA非编码区域检测到了肌节基因缺陷。家族中的相同突变可能表现出不同的表型(HCM、DCM和RCM)。病理学家报告了大的缺血性瘢痕,如今通过钆增强心脏磁共振成像在体内很容易检测到。缺血性心律失常基质增加了猝死风险。