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中链酰基辅酶A脱氢酶缺乏症导致一名23个月大婴儿急性肝衰竭

Medium-Chain Acyl-CoA Dehydrogenase Deficiency Disorder as a Cause of Acute Liver Failure in a 23-Month-Old Baby.

作者信息

Gjeta Inva, Bakalli Ilirjana, Sala Durim, Celaj Ermela, Biqiku Marsela, Hoxha Vladimir, Velmishi Virtut, Kola Elmira

机构信息

Pediatric Intensive Care Unit, University Hospital Center "Mother Teresa", Tirana, Albania.

出版信息

J Med Cases. 2025 Mar;16(3):114-119. doi: 10.14740/jmc5093. Epub 2025 Feb 27.

Abstract

Fatty acid oxidation disorders are inborn metabolic defects caused by impaired beta-oxidation of fats within the mitochondria. This occurs due to a deficiency in the pathway of fatty acids into the mitochondria via carnitine. Although their incidence is not frequent, the clinical presence of this disorder often leads to morbidity and high mortality. Medium-chain acyl-CoA dehydrogenase (MCAD) deficiency is part of the large group of fatty acid oxidation disorders which has a high variability in clinical manifestations and in daily medical practice can be challenging to early and correctly diagnose. In this article, we present a 23-month-old boy with drowsiness, mild hypoglycemia, and rapid progression to acute liver failure as a consequence of this metabolic disorder. Once the diagnosis was confirmed, treatment was conducted following the guideline of hypoglycemia of the metabolic disorder of MCAD deficiency and its complications. The child was discharged in good condition and the follow-up after 6 months was successful. Further, we review the literature on this genetic condition and check on how they connect to our case. The article aims to focus on the early evaluation of the clinical signs that present from the underlying of this rare metabolic disorder and the importance of aggressive treatment to prevent complications that can be fatal for the patient.

摘要

脂肪酸氧化障碍是由线粒体内脂肪的β-氧化受损引起的先天性代谢缺陷。这是由于脂肪酸通过肉碱进入线粒体的途径存在缺陷所致。尽管其发病率不高,但这种疾病的临床表现常常导致发病和高死亡率。中链酰基辅酶A脱氢酶(MCAD)缺乏症是一大类脂肪酸氧化障碍的一部分,其临床表现具有高度变异性,在日常医疗实践中早期正确诊断具有挑战性。在本文中,我们介绍了一名23个月大的男孩,因这种代谢紊乱出现嗜睡、轻度低血糖,并迅速发展为急性肝衰竭。一旦确诊,按照MCAD缺乏症代谢紊乱及其并发症的低血糖治疗指南进行治疗。患儿康复出院,6个月后的随访很成功。此外,我们回顾了关于这种遗传疾病的文献,并研究它们与我们病例的关联。本文旨在关注这种罕见代谢紊乱潜在临床表现的早期评估,以及积极治疗对预防可能对患者致命的并发症的重要性。

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Clinical manifestations and management of fatty acid oxidation disorders.脂肪酸氧化障碍的临床表现与治疗。
Rev Endocr Metab Disord. 2020 Dec;21(4):479-493. doi: 10.1007/s11154-020-09568-3.

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