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遗传性酪氨酸血症合并高胰岛素血症性低血糖症:一例罕见病例的诊断挑战

Hereditary Tyrosinemia Compounded With Hyperinsulinemic Hypoglycemia: Challenging Diagnosis of a Rare Case.

作者信息

Nasir Sharmeen, Raza Mohammad, Siddiqui Samrah I, Saleem Ayesha, Abbas Awais

机构信息

Pediatrics, Dow University of Health Sciences, Karachi, PAK.

Pediatrics, Civil Hospital Karachi, Karachi, PAK.

出版信息

Cureus. 2020 Nov 18;12(11):e11541. doi: 10.7759/cureus.11541.

Abstract

Hereditary tyrosinemia type 1 (HT-1) is a rare autosomal recessive disorder caused by a deficiency in the enzyme fumarylacetoacetate hydrolase (FAH), which catalyzes the final step in the tyrosine degradation pathway. Hereditary tyrosinemia is a heterogeneous disease with a wide spectrum of clinical manifestations involving hepatic, renal, or nervous systems. It has grave consequences if left untreated. Some of the late complications of hereditary tyrosinemia include cirrhosis, liver nodules, hepatocellular carcinoma, hypophosphatemic rickets, nephrocalcinosis, glomerulosclerosis, and chronic renal failure. Rarely, infants with hereditary tyrosinemia may present with persistent hypoglycemia, which may be a result of acute liver failure or hyperinsulinism. Hyperinsulinemic hypoglycemia (HH), caused by dysregulation of insulin secretion from pancreatic β-cells, leads to insulin driven glucose entry into the tissues and inhibits glycolysis, gluconeogenesis, fatty acid release, and ketone body synthesis. Hyperinsulinemic hypoglycemia can cause severe, persistent hypoketotic hypoglycemia. Diagnosing tyrosinemia type 1 can be a challenge as it is a heterogeneous disorder with a wide variety of clinical manifestations and complications. We herein report a rare case of a three-day-old male neonate with HT-1 compounded with HH.

摘要

1型遗传性酪氨酸血症(HT-1)是一种罕见的常染色体隐性疾病,由延胡索酰乙酰乙酸水解酶(FAH)缺乏引起,该酶催化酪氨酸降解途径的最后一步。遗传性酪氨酸血症是一种异质性疾病,临床表现广泛,累及肝脏、肾脏或神经系统。如果不治疗,会产生严重后果。遗传性酪氨酸血症的一些晚期并发症包括肝硬化、肝结节、肝细胞癌、低磷性佝偻病、肾钙质沉着、肾小球硬化和慢性肾衰竭。遗传性酪氨酸血症婴儿很少会出现持续性低血糖,这可能是急性肝衰竭或高胰岛素血症的结果。由胰腺β细胞胰岛素分泌失调引起的高胰岛素血症性低血糖(HH),会导致胰岛素驱动葡萄糖进入组织,并抑制糖酵解、糖异生、脂肪酸释放和酮体合成。高胰岛素血症性低血糖可导致严重的持续性低酮性低血糖。由于1型酪氨酸血症是一种具有多种临床表现和并发症的异质性疾病,因此诊断可能具有挑战性。我们在此报告一例罕见的3日龄男性新生儿,患有HT-1并伴有HH。

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