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异戊酸血症:一例报告

Isovaleric Acidemia: A Case Report.

作者信息

Zegarra Buitron Elizabeth, Vidal Panduro Daniel A, Guillén Ramírez Nancy S, González Arteaga María

机构信息

Internal Medicine, School of Medicine, Universidad Peruana de Ciencias Aplicadas, Lima, PER.

Paediatrics and Child Health, Hospital Nacional Docente Madre Niño San Bartolomé, Lima, PER.

出版信息

Cureus. 2023 Nov 24;15(11):e49362. doi: 10.7759/cureus.49362. eCollection 2023 Nov.

Abstract

Isovaleric acidemia is an autosomal recessive disease of leucine metabolism. The clinical presentation is variable and three phenotypes are described, asymptomatic, acute neonatal, and chronic intermittent. Infections are the most important trigger for catabolic crises. Diagnosis is based on the detection of isovalerylglycine CoA in urine and elevated levels of isovaleryl (C5) carnitine in blood. Long-term treatment consists of prevention of catabolic state, dietary restriction, and supplementation with L-carnitine and/or L-glycine. We present the case of a three-year-old female patient with multiple episodes of decompensation since the age of two years. The episode in which she was diagnosed had encephalopathy, with no neurological sequelae. Currently, the patient continues with dietary restrictions and supplementation with good nutritional and growth results for her age.

摘要

异戊酸血症是一种常染色体隐性遗传的亮氨酸代谢疾病。临床表现多样,有三种表型被描述,即无症状型、急性新生儿型和慢性间歇性型。感染是分解代谢危机最重要的诱因。诊断基于尿液中异戊酰甘氨酸辅酶A的检测以及血液中异戊酰(C5)肉碱水平的升高。长期治疗包括预防分解代谢状态、饮食限制以及补充左旋肉碱和/或左旋甘氨酸。我们报告一例3岁女性患者,自2岁起就有多次失代偿发作。她被诊断出患病的那次发作伴有脑病,无神经后遗症。目前,该患者继续进行饮食限制并补充营养,营养状况和生长发育结果与其年龄相符。

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引用本文的文献

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