Mitochondrial DNA deletion in a child with megaloblastic anemia and recurrent encephalopathy

J Child Neurol. 2004 Apr;19(4):258-61. doi: 10.1177/088307380401900403.

Abstract

A 3 1/2-year-old boy presented with megaloblastic anemia and recurrent episodes of severe lactic acidosis and coma. At age 4 years, he developed sepsis and died; postmortem examination failed to show any gross abnormality in any tissue. Biochemical analysis of muscle showed decreased activities for all respiratory chain enzymes except complex II. Muscle histochemistry revealed diffuse cytochrome c oxidase deficiency. Southern blot analysis of mitochondrial DNA from muscle, liver, and blood showed a heteroplasmic single mitochindrial DNA deletion of 2.4 kb, which removed the genes for cytochrome c oxidase I and II and the transfer ribonucleic acid genes for serine and aspartic acid. Single large-scale deletions in mitochondrial DNA have been associated with Pearson's syndrome, Kearns-Sayre syndrome, and progressive external ophthalmoplegia. This patient's presentation is unusual and suggests an overlap between Pearson's syndrome and Kearns-Sayre syndrome.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Acidosis, Lactic / genetics
  • Acidosis, Lactic / metabolism
  • Anemia, Megaloblastic / complications
  • Anemia, Megaloblastic / genetics*
  • Blotting, Southern
  • Brain Diseases, Metabolic / complications
  • Brain Diseases, Metabolic / genetics*
  • Brain Diseases, Metabolic / metabolism
  • Child, Preschool
  • Coma / genetics
  • Coma / metabolism
  • Cytochrome-c Oxidase Deficiency / diagnosis
  • Cytochrome-c Oxidase Deficiency / genetics*
  • Cytochrome-c Oxidase Deficiency / metabolism
  • DNA, Mitochondrial / genetics*
  • Diagnosis, Differential
  • Fatal Outcome
  • Humans
  • Kearns-Sayre Syndrome / genetics
  • Liver / metabolism
  • Male
  • Mitochondrial Diseases / diagnosis
  • Mitochondrial Diseases / genetics*
  • Mitochondrial Diseases / metabolism
  • Muscle, Skeletal / metabolism
  • Recurrence
  • Sepsis
  • Sequence Deletion*

Substances

  • DNA, Mitochondrial