Medium-chain acyl-CoA dehydrogenase deficiency in Saudi Arabia: incidence, genotype, and preventive implications

J Inherit Metab Dis. 2010 Dec:33 Suppl 3:S263-7. doi: 10.1007/s10545-010-9143-1. Epub 2010 Jun 22.

Abstract

Medium-chain acyl-CoA dehydrogenase deficiency (MCADD), caused by mutated ACADM gene, is a potentially fatal fatty acid oxidation defect. Detection of MCADD is now part of tandem mass spectrometry (MS-MS)-based newborn screening programs worldwide. To date, more than 67 mutations have been reported to cause MCADD with a single allele, c.985A>G, being the most common in patients of northwestern European descent. In Saudi Arabia, the Newborn Screening Program, officially launched in 2005, screens for 16 disorders including MCADD. Over a period of 3 years, 237,812 newborns were screened; 13 were identified to have MCADD giving an incidence of 1:18,293. Since the introduction of MS-MS to our institution, however, a total of 30 patients were detected to have MCADD. These cases were either newborns, at high-risk family members, or clinically suspected. The C8-carnitine levels (median 3.31, range 0.81-16.33 µM) were clearly diagnostic in all analyzed samples. Sequencing ACADM in 20 DBS revealed two novel mutations: c.362C>T (p.T121I) and c.347G>A (p.C116Y) substitutions, neither of which were detected in 300 chromosomes from controls. Eighteen (90%) patients were homozygous for the T121I mutation and two (10%) were compound heterozygous (T121I/C116Y). Our molecular data lend further support to MS-MS biochemical screening for MCADD and provide evidence for the relatively high incidence of MCADD in the Arab population. The identification of a founder mutation for MCADD has important implications for the preventive screening programs not only in Saudi Arabia but potentially also in other countries in the region.

MeSH terms

  • Acyl-CoA Dehydrogenase / blood
  • Acyl-CoA Dehydrogenase / deficiency
  • Acyl-CoA Dehydrogenase / genetics*
  • Biomarkers / blood
  • Carnitine / blood
  • DNA Mutational Analysis
  • Dried Blood Spot Testing
  • Founder Effect
  • Gene Frequency
  • Genetic Predisposition to Disease
  • Genetic Testing / methods
  • Humans
  • Incidence
  • Infant, Newborn
  • Lipid Metabolism, Inborn Errors / blood
  • Lipid Metabolism, Inborn Errors / diagnosis
  • Lipid Metabolism, Inborn Errors / enzymology
  • Lipid Metabolism, Inborn Errors / epidemiology
  • Lipid Metabolism, Inborn Errors / genetics
  • Lipid Metabolism, Inborn Errors / prevention & control
  • Mutation*
  • Neonatal Screening / methods
  • Phenotype
  • Predictive Value of Tests
  • Saudi Arabia / epidemiology
  • Tandem Mass Spectrometry

Substances

  • Biomarkers
  • Acyl-CoA Dehydrogenase
  • Carnitine

Supplementary concepts

  • Medium chain acyl CoA dehydrogenase deficiency