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丙酮酸代谢和三羧酸循环的缺陷。

Defects of pyruvate metabolism and the Krebs cycle.

作者信息

De Meirleir Linda

机构信息

Department of Pediatric Neurology, Free University of Brussels, Flemish speaking, Brussels, Belgium.

出版信息

J Child Neurol. 2002 Dec;17 Suppl 3:3S26-33; discussion 3S33-4.

Abstract

Seizures and metabolic disease are frequently associated, either indirectly as a consequence of the metabolically caused brain dysgenesis or directly by the metabolic derangement. This article describes defects in pyruvate metabolism (pyruvate carboxylase deficiency, pyruvate dehydrogenase deficiency) and Krebs cycle defects such as fumarase deficiency. Clinical characterizations and diagnostic strategies have been developed for each of these diseases. In contrast, very little is known about the specific epileptic features in these disorders. In females with a pyruvate dehydrogenase deficiency E1alpha owing to the mutation in the subunit E1alpha of the pyruvate dehydrogenase complex West's syndrome associated with large ventricles and corpus callosum agenesis on magnetic resonance imaging can be the main feature of the disease. In fumarase deficiency, prenatal brain dysgenesis is the most prominent feature of the disease. Diagnosis of these disorders requires measurements of lactate and pyruvate in plasma and cerebrospinal fluid, analysis of amino acids in plasma and organic acids in urine, and neuroradiologic investigations. Further biochemical and molecular analysis leads to a definitive diagnosis and opens the way to adequate treatment, genetic counseling, and prenatal diagnosis.

摘要

癫痫与代谢性疾病常常相关,这种关联要么是代谢导致脑发育异常的间接结果,要么是代谢紊乱的直接后果。本文描述了丙酮酸代谢缺陷(丙酮酸羧化酶缺乏症、丙酮酸脱氢酶缺乏症)以及三羧酸循环缺陷,如延胡索酸酶缺乏症。针对这些疾病中的每一种,都已制定了临床特征描述和诊断策略。相比之下,对于这些疾病中特定的癫痫特征却知之甚少。在因丙酮酸脱氢酶复合体E1α亚基突变导致丙酮酸脱氢酶E1α缺乏的女性患者中,磁共振成像显示与大脑室和胼胝体发育不全相关的韦斯特综合征可能是该疾病的主要特征。在延胡索酸酶缺乏症中,产前脑发育异常是该疾病最突出的特征。这些疾病的诊断需要检测血浆和脑脊液中的乳酸和丙酮酸,分析血浆中的氨基酸和尿液中的有机酸,以及进行神经放射学检查。进一步的生化和分子分析可得出明确诊断,并为适当的治疗、遗传咨询和产前诊断开辟道路。

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