Dhillon Neer, Stevens Andrew
Department of Internal Medicine, St. Matthews University School of Medicine, Orlando, FL, USA.
Department of Internal Medicine, Florida Hospital South, Orlando, FL, USA.
Am J Case Rep. 2019 Jul 24;20:1085-1088. doi: 10.12659/AJCR.914416.
BACKGROUND The urea cycle converts amino acids to urea and is excreted by the kidneys. Ornithine carbamoyltransferase (OTC) deficiency is a rare X-linked urea cycle disorder which results in hyperammonemia. Diagnosis is made based on a clinical presentation of poor feeding, hypotonia, biochemical profile, and genetic testing. Another genetic cause for hyperammonemia is hyperammonia hyperinsulinemia (HAHI) syndrome. A mutation coding for glutamate dehydrogenase (GDH) results in increased alpha-keto glutarate and ATP, triggering the secretion of pancreatic insulin. However, unlike OTC deficiency, these patients are asymptomatic but do have symptoms of hypoglycemia. The purpose of this article is to present the case of a 66-year-old woman with an unusual late-onset of OTC deficiency compounded with an underlying HAHI syndrome with co-disease management. CASE REPORT A 66-year-old female with a history significant for transient ischemic attack (TIA) and urea cycle disorder was admitted for new adverse symptoms. Further evaluation revealed hyperammonemia and hypoglycemia. Despite standard previous treatment for her underlying urea cycle disorder, high ammonia levels and hypoglycemia persisted. The contradicting values with continued hypoglycemia regardless of dextrose treatment was suspicious for underlying HAHI. Further genetic testing during her admission revealed a deletion in GLUD-1 gene concurrent with diagnosis of HAHI. After co-diagnosis was established, effective management required medications for both disorders in concordance with dietary restriction. CONCLUSIONS This is an extremely rare case of OTC deficiency, with a vague presentation in an elderly female. Exploring compounding genetic disorders in the presence of one that is already established and early recognition are crucial for prompt diagnosis and management.
尿素循环将氨基酸转化为尿素并由肾脏排出。鸟氨酸氨甲酰转移酶(OTC)缺乏症是一种罕见的X连锁尿素循环障碍,可导致高氨血症。诊断基于喂养困难、肌张力低下的临床表现、生化指标和基因检测。高氨血症的另一个遗传原因是高氨血症高胰岛素血症(HAHI)综合征。编码谷氨酸脱氢酶(GDH)的突变导致α-酮戊二酸和三磷酸腺苷增加,触发胰腺胰岛素分泌。然而,与OTC缺乏症不同,这些患者没有症状,但有低血糖症状。本文的目的是介绍一名66岁女性的病例,该患者患有不寻常的迟发性OTC缺乏症,并伴有潜在的HAHI综合征及合并疾病的管理情况。病例报告:一名有短暂性脑缺血发作(TIA)和尿素循环障碍病史的66岁女性因出现新的不良症状入院。进一步评估发现高氨血症和低血糖。尽管之前对其潜在的尿素循环障碍进行了标准治疗,但高氨水平和低血糖持续存在。无论葡萄糖治疗如何,持续低血糖的矛盾值提示可能存在潜在的HAHI。她住院期间的进一步基因检测发现GLUD - 1基因缺失,同时确诊为HAHI。确诊后,有效的管理需要针对两种疾病的药物治疗并配合饮食限制。结论:这是一例极为罕见的OTC缺乏症病例,在一名老年女性中表现不典型。在已确诊一种遗传疾病的情况下,探索合并的遗传疾病并早期识别对于及时诊断和管理至关重要。