Lim Kenneth, Steele David, Fenves Andrew, Thadhani Ravi, Heher Eliot, Karaa Amel
Division of Nephrology, and.
Department of Genetics and Pediatrics, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Clin Nephrol Case Stud. 2017 Mar 3;5:20-25. doi: 10.5414/CNCS109083. eCollection 2017.
Primary mitochondrial diseases (MD) are complex, heterogeneous inherited diseases caused by mutations in either the mitochondrial or nuclear DNA. Glomerular diseases in MD have been reported with tRNA mutation m.3243A>G causing a syndrome of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS). We describe here a case of focal segmental glomerulosclerosis (FSGS) associated with a new tRNA mutation site. A 34-year-old man with a history of living related kidney transplantation, diabetes, hearing loss, and developmental delay presented to the outpatient clinic with complaints of new behavioral difficulties, worsening symptoms, and brain involvement on imaging. Physical examination was remarkable for difficulty hearing, a pattern of dysarthric speech, and cerebellar gait. Laboratory investigations including lactate levels were unremarkable. Based on this set of clinical circumstances, concern for an underlying genetic abnormality was raised. Multiple metabolic tests were unremarkable. Whole exome sequencing revealed a mitochondrial MT-TW tRNA change at position m.5538G>A. Genotype-phenotype correlations are consistent with this tRNA mutation as a cause of his symptoms. To the best of our knowledge, this is the first case describing FSGS-associated MD caused by an m.5538 G>A mutation. Consideration of an underlying MD should be made in patients presenting with deafness, neurologic changes, diabetes, and renal failure.
原发性线粒体疾病(MD)是由线粒体或核DNA突变引起的复杂、异质性遗传性疾病。MD中的肾小球疾病已有报道,其中tRNA突变m.3243A>G可导致线粒体脑肌病、乳酸酸中毒和卒中样发作综合征(MELAS)。我们在此描述一例与新的tRNA突变位点相关的局灶节段性肾小球硬化(FSGS)病例。一名34岁男性,有活体亲属肾移植史、糖尿病、听力丧失和发育迟缓,因出现新的行为困难、症状加重以及影像学检查发现脑部受累而到门诊就诊。体格检查发现听力困难、构音障碍和小脑性步态。包括乳酸水平在内的实验室检查无异常。基于这一系列临床情况,人们对潜在的基因异常产生了担忧。多项代谢检查无异常。全外显子测序显示线粒体MT-TW tRNA在m.5538G>A位置发生改变。基因型-表型相关性与该tRNA突变导致其症状一致。据我们所知,这是第一例描述由m.5538 G>A突变引起的FSGS相关MD的病例。对于出现耳聋、神经病变、糖尿病和肾衰竭的患者,应考虑潜在的MD。