A Case Study of a Rare Disease (Fructosemia) Diagnosed in a Patient with Abdominal Pain.
Leszek GarbowskiMarzena WalasekRafał FirsztEwelina Chilińska-KopkoPaulina Błażejewska-GałaDaniel PopielnickiZofia Dzięcioł-AnikiejPublished in: Journal of clinical medicine (2024)
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.
Keyphrases
- abdominal pain
- type diabetes
- genome wide
- copy number
- clinical evaluation
- celiac disease
- single cell
- glycemic control
- oxidative stress
- gene expression
- early onset
- case report
- metabolic syndrome
- cardiovascular disease
- intellectual disability
- coronary artery disease
- young adults
- cardiovascular events
- insulin resistance
- drug induced
- transcription factor
- sleep quality