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一名患有1型瓜氨酸血症的青少年患者出现非典型表现的高氨血症性脑病。

Hyperammonemic Encephalopathy in an Adolescent Patient of Citrullinemia Type 1 With an Atypical Presentation.

作者信息

Ruxmohan Samir, Quinonez Jonathan, Choudhari Jinal, Poudel Sujan, Pandav Krunal

机构信息

Department of Neurology, Larkin Community Hospital, South Miami, USA.

Orthopedics, California Institute of Behavioral Neurosciences & Psychology, Fairfield, USA.

出版信息

Cureus. 2021 May 19;13(5):e15109. doi: 10.7759/cureus.15109.

Abstract

Citrullinemia refers to a family of autosomal recessive disorders involving the urea cycle. Three forms exist, which have different implications. Type I citrullinemia exists in both mild and severe forms. It arises due to mutations with argininosuccinate synthase leading to accumulation of ammonia and producing symptoms of lethargy, poor feeding, and seizures. Type II citrullinemia occurs due to citrin mutations involved in the urea cycle transport or during neonatal cholestasis. Management of both conditions requires low-protein diets along with arginine, sodium benzoate, and sodium phenylacetate. While traditional treatment shows improved outcomes, modifications may be necessary depending on a patient's presentation. We present a unique case of a 19-year-old wheelchair-bound female with a past medical history of heterozygous type I citrullinemia, seizures, and chronic encephalopathy presented to a local children's hospital for evaluation of altered mental status with a lethargic mental state. She was initially found to have an ammonia level of 329 µmol/L and choledocholithiasis on admission. Dietary modification with intravenous dextrose and intralipids with oral lactulose saw improvement in her labs. However, her ammonia level increased to 381 µmol/L despite such interventions. Intensive care was required to normalize her serum ammonia level and clear her for a magnetic resonance cholangiopancreatography (MRCP). We present a unique case of heterozygous type I citrullinemia with some overlap with type II citrullinemia features. Further studies are needed to understand better the observed unique presentation and long-term clinical implications associated with the disease.

摘要

瓜氨酸血症是指一组涉及尿素循环的常染色体隐性遗传疾病。它有三种形式,各有不同影响。I型瓜氨酸血症有轻度和重度两种形式。它是由精氨琥珀酸合成酶突变引起的,导致氨积累并产生嗜睡、喂养困难和癫痫发作等症状。II型瓜氨酸血症是由于参与尿素循环转运的citrin突变或新生儿胆汁淤积期间发生的。这两种情况的治疗都需要低蛋白饮食,同时补充精氨酸、苯甲酸钠和苯乙酸钠。虽然传统治疗显示出较好的效果,但可能需要根据患者的表现进行调整。我们报告了一例独特病例,一名19岁的女性,因患有杂合子I型瓜氨酸血症、癫痫和慢性脑病,需要轮椅辅助行动,她因精神状态改变、嗜睡而被送往当地儿童医院进行评估。入院时,她最初被发现氨水平为329微摩尔/升,并有胆总管结石。通过静脉输注葡萄糖、静脉输注脂肪乳和口服乳果糖进行饮食调整后,她的实验室检查结果有所改善。然而,尽管采取了这些干预措施,她的氨水平仍升至381微摩尔/升。需要重症监护来使她的血清氨水平正常化,并为她进行磁共振胰胆管造影(MRCP)检查。我们报告了一例杂合子I型瓜氨酸血症的独特病例,其具有一些与II型瓜氨酸血症特征重叠的表现。需要进一步研究以更好地了解观察到的独特表现以及与该疾病相关的长期临床意义。

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