Carnitine membrane transporter deficiency: a rare treatable cause of cardiomyopathy and anemia

Pediatr Cardiol. 2008 Jan;29(1):163-5. doi: 10.1007/s00246-007-9051-9. Epub 2007 Oct 10.

Abstract

Carnitine transporter defect is an autosomal recessive disorder caused by mutations in the SLC22A5 gene that encodes the high-affinity carnitine transporter OCTN2. Affected patients can present with predominant metabolic or cardiac manifestations. Early recognition of this disorder in a context of life-threatening cardiac failure and treatment with carnitine can be lifesaving in this inborn error of fatty acid oxidation. Here we describe a boy with a severe cardiomyopathy and severe anemia who improved with carnitine therapy. Physiopathology of anemia, a probably less recognized symptom of carnitine deficiency, is also discussed.

Publication types

  • Case Reports

MeSH terms

  • Anemia, Iron-Deficiency / drug therapy
  • Anemia, Iron-Deficiency / etiology*
  • Anemia, Iron-Deficiency / physiopathology
  • Cardiomyopathies / drug therapy
  • Cardiomyopathies / etiology*
  • Carnitine / therapeutic use
  • Humans
  • Infant
  • Male
  • Metabolism, Inborn Errors / complications*
  • Metabolism, Inborn Errors / drug therapy
  • Organic Cation Transport Proteins / deficiency*
  • Solute Carrier Family 22 Member 5
  • Vitamin B Complex / therapeutic use

Substances

  • Organic Cation Transport Proteins
  • SLC22A5 protein, human
  • Solute Carrier Family 22 Member 5
  • Vitamin B Complex
  • Carnitine