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一例诊断为罕见病(果糖血症)的腹痛患者的病例研究。

A Case Study of a Rare Disease (Fructosemia) Diagnosed in a Patient with Abdominal Pain.

作者信息

Garbowski Leszek, Walasek Marzena, Firszt Rafał, Chilińska-Kopko Ewelina, Błażejewska-Gała Paulina, Popielnicki Daniel, Dzięcioł-Anikiej Zofia

机构信息

Public Independent Healthcare Services of the Ministry of Internal Affairs and Administration in Białystok, 15-471 Białystok, Poland.

Department of Human Anatomy, Medical University of Białystok, 15-089 Białystok, Poland.

出版信息

J Clin Med. 2024 Jun 10;13(12):3394. doi: 10.3390/jcm13123394.

Abstract

Hereditary fructose intolerance is a rare genetic disorder that is inherited in an autosomal recessive manner, with mutations sometimes occurring spontaneously. Consuming fructose triggers biochemical abnormalities, disrupting liver processes like glycogenolysis and gluconeogenesis. Recent studies have revealed elevated intrahepatic fat levels in affected individuals. Symptoms include aversion to fructose-containing foods, hypoglycemia, liver and kidney dysfunction, and growth delays, with severe cases leading to liver enlargement, fatty liver disease, kidney failure, and life-threatening hypoglycemia. In this case study, we present a 20-month-old child with symptoms including difficulty passing stool, abdominal rigidity, abdominal pain with bloating and hypoglycemia. Initial clinical findings revealed elevated liver enzymes, a mildly enlarged hyperechoic liver, hypercholesterolemia, and borderline alpha-fetoprotein values. Diagnostic assessments identified hereditary fructose intolerance (HFI) with pathogenic variants in the ALDOB gene, along with a diagnosis of celiac disease. Genetic testing of the parents revealed carrier status for pathological aldolase B genes. This case underscores the importance of comprehensive clinical evaluation and genetic testing in pediatric patients with complex metabolic presentations.

摘要

遗传性果糖不耐受是一种罕见的遗传性疾病,以常染色体隐性方式遗传,有时会自发发生突变。摄入果糖会引发生化异常,扰乱肝脏的糖原分解和糖异生等过程。最近的研究表明,受影响个体的肝内脂肪水平升高。症状包括厌恶含果糖食物、低血糖、肝肾功能障碍和生长发育迟缓,严重的病例会导致肝脏肿大、脂肪肝疾病、肾衰竭和危及生命的低血糖。在本病例研究中,我们介绍了一名20个月大的儿童,其症状包括排便困难、腹部僵硬、腹胀腹痛和低血糖。最初的临床检查发现肝酶升高、肝脏轻度增大且回声增强、高胆固醇血症以及甲胎蛋白值临界。诊断评估确定为遗传性果糖不耐受(HFI),伴有ALDOB基因的致病变异,同时诊断为乳糜泻。对父母的基因检测显示为病理性醛缩酶B基因的携带者状态。该病例强调了对具有复杂代谢表现的儿科患者进行全面临床评估和基因检测的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/20e9/11204229/6dadeec75ada/jcm-13-03394-g001.jpg

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