Aurora Cardiovascular and Thoracic Services, Aurora Sinai/Aurora St. Luke's Medical Centers, University of Wisconsin School of Medicine and Public Health, Milwaukee, Wisconsin.
Aurora Cardiovascular and Thoracic Services, Aurora Sinai/Aurora St. Luke's Medical Centers, University of Wisconsin School of Medicine and Public Health, Milwaukee, Wisconsin.
Am J Cardiol. 2020 Nov 15;135:168-173. doi: 10.1016/j.amjcard.2020.08.027. Epub 2020 Aug 28.
Multiple lentigines syndrome is an autosomal dominant inherited condition with variable expressivity that is also known as LEOPARD syndrome. LEOPARD stands for lentigines, electrocardiographic conduction defects, ocular hypertelorism, pulmonary valve stenosis, abnormalities of genitalia, retardation of growth, and deafness. LEOPARD syndrome most frequently develops secondary to a missense mutation of protein-tyrosine phosphatase nonreceptor type 11 gene, which encodes tyrosine phosphatase. The missense mutation p.Tyr279Cys can either occur as a de novo mutation or affect multiple family members. Although hypertrophic cardiomyopathy is not part of the LEOPARD acronym, it is the most frequent cardiac anomaly observed in this syndrome. The recognition of increased left or right ventricular wall thickness in patients with LEOPARD syndrome may have significant impact on their clinical course similar to classic hypertrophic cardiomyopathy, which may require septal reduction procedures for relief of left or right ventricular outflow tract obstruction or implantable cardioverter-defibrillator placement for sudden cardiac death prevention. We describe a case series of a family with diffuse lentigines and hypertrophic cardiomyopathy in which the son carries the protein-tyrosine phosphatase nonreceptor type 11 (p.Tyr279Cys) gene mutation and both the son and daughter underwent left ventricular myectomy at an early age. In conclusion, our case series of a family with LEOPARD syndrome illustrates the importance of recognizing hypertrophic cardiomyopathy as part of this syndrome.
多发性黑子综合征是一种常染色体显性遗传疾病,具有可变的外显率,也被称为 LEOPARD 综合征。LEOPARD 代表黑子、心电图传导缺陷、眼距过宽、肺动脉瓣狭窄、生殖器异常、生长迟缓以及耳聋。LEOPARD 综合征最常继发于蛋白酪氨酸磷酸酶非受体型 11 基因的错义突变,该基因编码酪氨酸磷酸酶。错义突变 p.Tyr279Cys 既可以是新生突变,也可以影响多个家族成员。尽管肥厚型心肌病不是 LEOPARD 缩写的一部分,但它是该综合征中最常见的心脏异常。在 LEOPARD 综合征患者中,识别出左心室或右心室壁增厚可能对其临床病程有重大影响,类似于经典肥厚型心肌病,这可能需要进行室间隔切除术以缓解左心室或右心室流出道梗阻,或植入心脏复律除颤器以预防心源性猝死。我们描述了一个家族的病例系列,该家族有弥漫性黑子和肥厚型心肌病,其儿子携带蛋白酪氨酸磷酸酶非受体型 11 基因(p.Tyr279Cys)突变,儿子和女儿都在早期接受了左心室心肌切除术。总之,我们对一个 LEOPARD 综合征家族的病例系列研究说明了认识到肥厚型心肌病是该综合征的一部分的重要性。