Arrhythmias, Inherited Cardiac Diseases and Sudden Death Unit, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950, Barcelona, Catalonia, Spain.
Malalties Cardiovasculars en El Desenvolupament, Institut de Recerca Sant Joan de Déu (IRSJD), Arrítmies Pediàtriques, Cardiologia Genètica I Mort Sobtada, Esplugues de Llobregat, 08950, Barcelona, Spain.
Eur J Pediatr. 2024 Nov;183(11):4635-4640. doi: 10.1007/s00431-024-05757-3. Epub 2024 Sep 23.
Cardiac channelopathies are a group of inherited syndromes that can cause malignant arrhythmias and sudden cardiac death, particularly in the pediatric population. Today, a 12-lead electrocardiogram is the most effective tool to diagnose these diseases. Incomplete penetrance and variable expressivity are hallmarks of these syndromes. Some of these malignant entities may remain hidden and only a trigger such as exercise, emotions or fever can unmask the electrical pattern to diagnose the disease. Sudden cardiac death may be the first manifestation of any of these syndromes. The use of complementary tests that allow early diagnosis is strongly recommended, among which we find: pharmacological provocations, exercise tests, and genetic analysis. Genetic testing makes it possible to unravel the origin of the disease, and also identify family members who carry the harmful genetic defect and are therefore at risk. One of the main challenges in this area is the large number of genetic variants of uncertain significance, which prevent effective translation into clinical practice. Early identification of the pediatric population at risk and adequate risk stratification are crucial to adopting personalized preventive measures that reduce the risk of lethal episodes in this population. What is Known: • In the pediatric population, malignant arrhythmias leading to sudden cardiac death are mainly caused by inherited syndromes. • A conclusive genetic diagnosis unravels the origin of the syndrome and allows cascade screening to identify relatives carrying the genetic alteration. What is New: • The use of sequencing technologies allows a broad genetic analysis, helping to unravel new genetic alterations causing inherited arrhythmogenic syndromes. • A periodic reanalysis of genetic variants that currently have an ambiguous role will help discern those that are truly pathogenic.
心脏通道病是一组遗传性综合征,可导致恶性心律失常和心脏性猝死,尤其是在儿科人群中。如今,12 导联心电图是诊断这些疾病最有效的工具。不完全外显率和可变表达是这些综合征的特征。这些恶性实体中的一些可能仍然隐藏着,只有运动、情绪或发热等诱因才能揭示电模式,从而诊断疾病。心脏性猝死可能是这些综合征的首次表现。强烈建议使用允许早期诊断的补充测试,其中包括:药理学激发、运动试验和基因分析。基因测试可以揭示疾病的起源,还可以识别携带有害遗传缺陷的家庭成员,因此他们处于危险之中。该领域的主要挑战之一是大量意义不明的遗传变异,这使得它们无法有效地转化为临床实践。早期识别高危儿科人群并进行充分的风险分层对于采取个性化预防措施以降低该人群致命事件的风险至关重要。已知内容:
在儿科人群中,导致心脏性猝死的恶性心律失常主要由遗传性综合征引起。
明确的基因诊断揭示了综合征的起源,并允许级联筛查以识别携带遗传改变的亲属。
测序技术的使用允许进行广泛的基因分析,有助于揭示导致遗传性心律失常综合征的新遗传改变。
定期重新分析目前具有模糊作用的遗传变异将有助于辨别那些真正具有致病性的变异。