Organic acidurias in Egyptian children: The urge for high-risk screening

Dina A Mehaney1,2, Zeinab S Seliem3, Laila A Selim4

  • 1Clinical and Chemical Pathology Department, Cairo University, Cairo, Egypt.

Insights

Organic acidurias, a group of metabolic disorders, were frequently found in high-risk Egyptian children. Early detection through high-risk screening is crucial for timely intervention and improved outcomes.

Area of Science:

  • Biochemistry
  • Genetics
  • Pediatrics

Background:

  • Organic acidurias are inborn errors of metabolism posing diagnostic challenges.
  • These disorders are common in high-risk pediatric populations, leading to significant morbidity and mortality.
  • Gas chromatography-mass spectrometry (GC-MS) is a key diagnostic tool for organic acidurias.

Purpose of the Study:

  • To determine the frequency of organic acidurias in high-risk Egyptian children.
  • To emphasize the importance of high-risk screening for early detection of these metabolic disorders.
  • To identify common organic acidurias in the studied pediatric cohort.

Main Methods:

  • A cohort of 150 high-risk children was evaluated.
  • Urine organic acid analysis was performed using gas chromatography-mass spectrometry (GC-MS).
  • Patients were recruited from Cairo University Children Hospital's inherited metabolic disease and pediatric intensive care units.

Main Results:

  • Thirty percent (45/150) of patients exhibited altered organic acid profiles.
  • Neurological manifestations were the most frequent clinical presentation.
  • Glutaric aciduria type I and maple-syrup urine disease were the most prevalent identified disorders.

Conclusions:

  • GC-MS detection of organic acids is vital for diagnosing metabolic disorders.
  • High-risk screening is recommended for early identification and intervention in Egyptian children.
  • Establishing a national expanded newborn screening program is essential.
Abstract

Related Concept Videos

Diagnosing Acidosis and Alkalosis01:24

Diagnosing Acidosis and Alkalosis

Diagnosing acid-base imbalances involves systematically analyzing arterial blood samples, focusing on three key measurements: pH, bicarbonate (HCO3−) concentration, and carbon dioxide partial pressure (PCO2). This analysis follows a four-step process that helps identify the imbalance's underlying cause and nature.
First, the pH level is assessed to determine whether the blood pH is normal (7.35–7.45), low (acidosis), or high (alkalosis).
Next, the PCO2  and...
384
Inborn Errors of Metabolism01:20

Inborn Errors of Metabolism

Phenylketonuria (PKU) is a protein metabolism disorder characterized by high blood levels of the amino acid phenylalanine. This results from a mutation in the gene responsible for phenylalanine hydroxylase, an enzyme that converts phenylalanine into tyrosine. When this enzyme is deficient, phenylalanine builds up in the blood, leading to symptoms such as vomiting, rashes, seizures, growth deficiency, and severe mental retardation. An early diagnosis and a diet restricting phenylalanine intake...
221
Serum Laboratory Studies, Stool Test, Breath Test01:30

Serum Laboratory Studies, Stool Test, Breath Test

Gastrointestinal (GI) diagnostic studies are pivotal in confirming, ruling out, diagnosing, or staging various diseases, including cancers. Following diagnosis, allocating time for discussions with the patient and providing informational resources is crucial. Diagnostic assessments of the GI tract often occur in outpatient settings like endoscopy suites or GI labs. Preparation for these tests may include dietary restrictions, fasting, liquid bowel preparations, laxatives, enemas, and the...
448
Urine Studies I: Urinalysis01:29

Urine Studies I: Urinalysis

Urinalysis is a widely used diagnostic test that analyzes urine's physical, chemical, and microscopic characteristics. Healthcare providers use it to detect and monitor various health conditions, including renal disease, urinary tract infections (UTIs), diabetes, and metabolic or systemic disorders.Components of UrinalysisUrinalysis consists of three primary components: physical, chemical, and microscopic examination. Each provides unique insights into the urine sample and, by extension, the...
65