Paech Christian, Gebauer Roman Antonin, Karstedt Jens, Marschall Christoph, Bollmann Andreas, Husser Daniela
Department for Pediatric Cardiology, University of Leipzig-Heart Center, Strümpellstrasse 39, 04289, Leipzig, Germany,
Pediatr Cardiol. 2014 Dec;35(8):1437-41. doi: 10.1007/s00246-014-0950-2. Epub 2014 Jun 21.
Idiopathic ventricular fibrillation (IVF) is a rare genetically determined disease causing unexpected cardiac death in otherwise healthy individuals. This study identified two novel, functional heterozygous mutations in the ryanodine receptor 2 (RyR2) gene in a family with IVF. In the presented case all the patients received a thorough diagnostic workup to exclude structural heart disease. Blood was drawn from the patients, and genetic testing was performed including amplification and sequencing of splice locations in two exons of the RyR2 gene. The mutations were detected in five symptomatic family members. The genetic status of the five affected family members remains unclear. No clinically affected patient is without mutation. At this writing, one family member with confirmed mutation is asymptomatic. The differentiation between catecholaminergic polymorphic ventricular tachycardia (CPVT) and IVF remains a difficult issue, mainly based on clinical characteristics and gross genetic classification. In our case, the family history, exercise testing, and epinephrine stress testing do not suggest an association of arrhythmia and adrenergic triggers, which makes CPVT rather unlikely despite the fact that genetic testing showed RyR2 mutations. Currently, knowledge concerning the functional meaning of genetic mutations is growing. Future exploration of these functional aspects might give further impetus to allocation of these patients to a specific diagnosis.
特发性心室颤动(IVF)是一种罕见的由基因决定的疾病,可导致原本健康的个体意外心脏死亡。本研究在一个患有IVF的家族中,在兰尼碱受体2(RyR2)基因中鉴定出两个新的功能性杂合突变。在本病例中,所有患者均接受了全面的诊断检查以排除结构性心脏病。从患者身上采集血液,并进行基因检测,包括对RyR2基因两个外显子的剪接位点进行扩增和测序。在五名有症状的家庭成员中检测到了这些突变。五名受影响家庭成员的基因状态仍不清楚。没有临床症状的患者没有突变。在撰写本文时,一名确诊有突变的家庭成员没有症状。儿茶酚胺能多形性室性心动过速(CPVT)和IVF之间的鉴别仍然是一个难题,主要基于临床特征和大致的基因分类。在我们的病例中,家族史、运动试验和肾上腺素激发试验均未提示心律失常与肾上腺素能触发因素有关,尽管基因检测显示有RyR2突变,但CPVT的可能性较小。目前,关于基因突变功能意义的知识正在不断增加。对这些功能方面的未来探索可能会进一步推动将这些患者分配到特定诊断中。