IRCCS Istituto delle Scienze Neurologiche di Bologna, Full Member of the ERN EpiCARE, Bologna, Italia.
Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italia.
Ann Clin Transl Neurol. 2021 Mar;8(3):704-710. doi: 10.1002/acn3.51259. Epub 2021 Jan 21.
The study aims to characterize the epilepsy phenotype of maternally inherited Leigh's syndrome (MILS) and neuropathy, ataxia, retinitis pigmentosa (NARP) due to mutations in the mitochondrial ATP6 gene and to correlate electroclinical features with mutant heteroplasmy load (HL). We investigated 17 individuals with different phenotype, from asymptomatic carriers to MILS: 11 carried the m.8993T> G mutation, 5 the m.8993T> C and one the novel, de novo m.8858G> A mutation. Seizures occurred in 37.5% of patients, EEG abnormalities in 73%. We ranked clinical and EEG abnormalities severity and performed quantitative EEG to estimate Abnormality Ratio (AR) and Spectral Relative Power (SRP). Spearman's rho and Kruskal-Wallis test were used for correlation with heteroplasmy load (HL). HL correlated with disease severity (Rho = 0.63, P = 0.012) and was significantly higher in patients with seizures or EEG abnormalities (P = 0.014). HL correlated with EEG severity score only for the m.8993T> G (Rho = 0.73, P = 0.040), showing a trend toward a positive correlation with AR and delta SPR, irrespective of the mutation.
本研究旨在对母系遗传 Leigh 综合征(MILS)和神经病、共济失调、视网膜色素变性(NARP)的癫痫表型进行特征描述,这些疾病是由线粒体 ATP6 基因突变引起的,并将电临床特征与突变异质体负荷(HL)相关联。我们调查了 17 名具有不同表型的个体,从无症状携带者到 MILS:11 名携带 m.8993T>G 突变,5 名携带 m.8993T>C 突变,1 名携带新的、从头发生的 m.8858G>A 突变。37.5%的患者发生癫痫,73%的患者脑电图异常。我们对临床和脑电图异常的严重程度进行了排序,并进行了定量脑电图以估计异常比(AR)和频谱相对功率(SRP)。采用 Spearman rho 和 Kruskal-Wallis 检验进行与异质体负荷(HL)的相关性分析。HL 与疾病严重程度相关(Rho=0.63,P=0.012),在有癫痫发作或脑电图异常的患者中显著更高(P=0.014)。HL 仅与 m.8993T>G 的脑电图严重程度评分相关(Rho=0.73,P=0.040),表明与 AR 和 delta SPR 呈正相关趋势,与突变无关。