Protonotarios Nikos, Tsatsopoulou Adalena
Yannis Protonotarios Foundation, Medical Center of Naxos, Naxos 84300, Greece.
Orphanet J Rare Dis. 2006 Mar 13;1:4. doi: 10.1186/1750-1172-1-4.
Naxos disease is a recessively inherited condition with arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C) and a cutaneous phenotype, characterised by peculiar woolly hair and palmoplantar keratoderma. The disease was first described in families originating from the Greek island of Naxos. Moreover, affected families have been identified in other Aegean islands, Turkey, Israel and Saudi Arabia. A syndrome with the same cutaneous phenotype and predominantly left ventricular involvement has been described in families from India and Ecuador (Carvajal syndrome). Woolly hair appears from birth, palmoplantar keratoderma develop during the first year of life and cardiomyopathy is clinically manifested by adolescence with 100% penetrance. Patients present with syncope, sustained ventricular tachycardia or sudden death. Symptoms of right heart failure appear during the end stages of the disease. In the Carvajal variant the cardiomyopathy is clinically manifested during childhood leading more frequently to heart failure. Mutations in the genes encoding the desmosomal proteins plakoglobin and desmoplakin have been identified as the cause of Naxos disease. Defects in the linking sites of these proteins can interrupt the contiguous chain of cell adhesion, particularly under conditions of increased mechanical stress or stretch, leading to cell death, progressive loss of myocardium and fibro-fatty replacement. Implantation of an automatic cardioverter defibrillator is indicated for prevention of sudden cardiac death. Antiarrhythmic drugs are used for preventing recurrences of episodes of sustained ventricular tachycardia and classical pharmacological treatment for congestive heart failure, while heart transplantation is considered at the end stages.
纳克索斯病是一种隐性遗传疾病,伴有致心律失常性右室发育不良/心肌病(ARVD/C)和皮肤表型,其特征为特殊的羊毛状头发和掌跖角化病。该病最初在来自希腊纳克索斯岛的家族中被描述。此外,在其他爱琴海岛、土耳其、以色列和沙特阿拉伯也发现了患病家族。在来自印度和厄瓜多尔的家族中描述了一种具有相同皮肤表型且主要累及左心室的综合征(卡瓦哈尔综合征)。羊毛状头发出生时即出现,掌跖角化病在出生后第一年发展,心肌病在青春期临床表现出来,外显率为100%。患者表现为晕厥、持续性室性心动过速或猝死。右心衰竭症状在疾病末期出现。在卡瓦哈尔变异型中,心肌病在儿童期临床表现出来,更常导致心力衰竭。已确定编码桥粒蛋白盘状球蛋白和桥粒斑蛋白的基因突变是纳克索斯病的病因。这些蛋白质连接位点的缺陷可中断细胞粘附的连续链,特别是在机械应力或拉伸增加的情况下,导致细胞死亡、心肌逐渐丧失和纤维脂肪替代。植入自动心脏复律除颤器可预防心源性猝死。抗心律失常药物用于预防持续性室性心动过速发作的复发以及用于充血性心力衰竭的经典药物治疗,而在疾病末期考虑进行心脏移植。