KfH Nierenzentrum München - West and Nephrologisches Zentrum, Klinikum der Universität München, Munich, Germany.
Wien Klin Wochenschr. 2010 Jun;122(11-12):363-5. doi: 10.1007/s00508-010-1388-z. Epub 2010 Jun 17.
Mitochondrial diseases are a heterogeneous group of syndromes caused by genetic defects in mitochondrial DNA (mtDNA) or nuclear-encoded mitochondrial genes. They present with a wide range of clinical phenotypes. Myopathy may be the sole or main sign, or merely an incidental finding occurring in the late course of a multisystemic illness. Although mitochondrial disorders are increasingly being recognized, confirming a specific diagnosis remains a great challenge due to the clinical heterogeneity of the diseases. Several well-defined clinical syndromes associated with specific mutations have been described. Thus, suspicion of mitochondrial diseases among patients with multiple, seemingly unrelated neuromuscular and multisystem disorders should be confirmed by the finding of deleterious mutations in genes involving mitochondrial biogenesis and function. This case report describes a 58-year-old patient with a 40-year course of a multisystemic illness representing the diagnostic challenges of mitochondrial disease.
线粒体疾病是一组由线粒体 DNA(mtDNA)或核编码线粒体基因的遗传缺陷引起的异质性综合征。它们表现出广泛的临床表型。肌病可能是唯一或主要的表现,或者仅仅是多系统疾病后期发生的偶然发现。尽管线粒体疾病的认识日益增加,但由于疾病的临床表现异质性,确定特定的诊断仍然是一个巨大的挑战。已经描述了几种与特定突变相关的明确临床综合征。因此,对于患有多种看似无关的神经肌肉和多系统疾病的患者,如果发现涉及线粒体生物发生和功能的基因中有有害突变,应怀疑线粒体疾病。本病例报告描述了一名 58 岁患者,其患有 40 年的多系统疾病,代表了线粒体疾病的诊断挑战。