Muscular carnitine palmitoyltransferase II deficiency in infancy

Pediatr Neurol. 2000 Feb;22(2):148-50. doi: 10.1016/s0887-8994(99)00125-3.

Abstract

An 8-month-old female presented with febrile myoglobinuria. The activity of carnitine palmitoyltransferase (CPT) II was decreased to 16% of the control mean, and the oxidation of the long-chain fatty acids was reduced to 25% of the mean in the fibroblasts. Homozygosity for the common mutation, S113L, was identified in the CPT II gene. Residual CPT II activity of more than 10% of the mean and homozygosity for the common mutation S113L are usually associated with a milder reduction of long-chain fatty acid oxidation to about 80% of the control and with a later age of clinical onset. The early clinical presentation in the present patient is unique and was associated with a marked impairment of long-chain fatty acid oxidation, possibly because of other genetic factors. CPT II deficiency should be included in the differential diagnosis of isolated myoglobinuria in infancy.

Publication types

  • Case Reports

MeSH terms

  • Carnitine O-Palmitoyltransferase / genetics*
  • Carnitine O-Palmitoyltransferase / metabolism
  • Creatine Kinase / blood
  • Diagnosis, Differential
  • Electrolytes / administration & dosage
  • Fatty Acids / blood*
  • Fatty Acids / genetics
  • Female
  • Fever / etiology
  • Fluid Therapy
  • Homozygote
  • Humans
  • Infant
  • Metabolism, Inborn Errors / complications
  • Metabolism, Inborn Errors / diagnosis*
  • Metabolism, Inborn Errors / enzymology
  • Metabolism, Inborn Errors / genetics*
  • Myoglobinuria / enzymology
  • Myoglobinuria / etiology*
  • Myoglobinuria / genetics*
  • Oxidation-Reduction
  • Treatment Outcome

Substances

  • Electrolytes
  • Fatty Acids
  • Carnitine O-Palmitoyltransferase
  • Creatine Kinase