Cadoni Gabriella, Primiano Guido, Picciotti Pasqualina M, Calandrelli Rosalinda, Galli Jacopo, Servidei Serenella, Conti Guido
Dipartimento di Neuroscienze, Organi di Senso e Torace, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, 00168 Rome, Italy.
Dipartimento di Testa-Collo e Organi di Senso, Catholic University of the Sacred Heart, 00168 Rome, Italy.
J Pers Med. 2023 Aug 29;13(9):1329. doi: 10.3390/jpm13091329.
Mitochondrial diseases (MDs) are heterogeneous genetic disorders characterized by mitochondrial DNA (mtDNA) defects, involving tissues highly dependent on oxidative metabolism: the inner ear, brain, eye, skeletal muscle, and heart. We describe adult patients with genetically defined MDs, characterizing hearing function and neuroimaging results. We enrolled 34 patients (mean age: 50.02 ± 15 years, range: 18-75 years; 20 females and 14 males) classified in four groups: MELAS, MIDD, PEO, and Encephalopathy/Polyneuropathy. Audiological evaluations included psychoacoustical tests (pure-tone and speech audiometry), electrophysiological tests (Auditory Brainstem Responses, ABRs), and Impedenzometry. Neuroimaging evaluations considered global MRI abnormalities or structural brain changes. In total, 19/34 patients carried the m.3243A > G mutation (6 affected by MELAS, 12 affected by MIDD, and 1 affected by PEO); 11 had an mtDNA deletion (all affected by PEO); 3 had nuclear genes associated with MDs (POLG1 and OPA1); and 1 patient had an mtDNA deletion without an identified nuclear gene defect (affected by PEO). Sensory neural, bilateral, and symmetrical hearing loss was present in 25 patients (73.5%) to different degrees: 9 mild, 9 moderate, 5 severe, and 2 profound. The severe/profound and mild hearing losses were associated with pantonal and high-frequency audiograms, respectively. Instead, moderate hearing losses were associated with both high-frequency (five cases) and pantonal (five cases) audiogram shapes. In addition, 21/25 patients showed a cochlear site of lesion (84%), and 4/25 (16%) showed a retrocochlear site. We found global MRI abnormalities or structural brain changes in 26/30 subjects (86.6%): 21 had white matter abnormalities, 15 had cortical atrophy, 10 had subcortical atrophy, 8 had basal nuclei involvement or cerebellar atrophy, 4 had stroke-like lesions or laminar necrosis, and 1 had cysts or vacuolated lesions. We concluded that genetic alterations are associated with different clinical presentations for both auditory function and neuroradiological findings. There is no fixed relationship between genotype and phenotype for the clinical conditions analyzed.
线粒体疾病(MDs)是一类异质性遗传疾病,其特征为线粒体DNA(mtDNA)缺陷,累及高度依赖氧化代谢的组织:内耳、脑、眼、骨骼肌和心脏。我们描述了具有明确遗传特征的成年MDs患者,对其听力功能和神经影像学结果进行了特征分析。我们纳入了34例患者(平均年龄:50.02±15岁,范围:18 - 75岁;女性20例,男性14例),分为四组:线粒体脑肌病伴乳酸血症和卒中样发作(MELAS)、母系遗传性糖尿病伴耳聋(MIDD)、进行性眼外肌麻痹(PEO)和脑病/多发性神经病。听力学评估包括心理声学测试(纯音和言语测听)、电生理测试(听觉脑干反应,ABR)和阻抗测听法。神经影像学评估考虑了整体MRI异常或脑部结构变化。总共,34例患者中有19例携带m.3243A > G突变(6例为MELAS,12例为MIDD,1例为PEO);11例存在mtDNA缺失(均为PEO);3例有与MDs相关的核基因(聚合酶γ1基因(POLG1)和视神经萎缩蛋白1基因(OPA1));1例患者存在mtDNA缺失但未发现核基因缺陷(为PEO)。25例患者(73.5%)存在不同程度的感音神经性、双侧对称性听力损失:9例轻度,9例中度,5例重度,2例极重度。重度/极重度和轻度听力损失分别与全频和高频听力图相关。相反,中度听力损失与高频(5例)和全频(5例)听力图形状均相关。此外,25例患者中有21例(84%)显示病变位于耳蜗,4例(16%)显示病变位于蜗后。我们发现30例受试者中有26例(86.6%)存在整体MRI异常或脑部结构变化:21例有白质异常,15例有皮质萎缩现象,10例有皮质下萎缩,8例有基底核受累或小脑萎缩,4例有卒中样病变或层状坏死,1例有囊肿或空泡样病变。我们得出结论,基因改变与听觉功能和神经放射学检查结果的不同临床表现相关。在所分析的临床病症中,基因型和表型之间不存在固定关系。