Use of human somatotrophin in the treatment of a patient with methylmalonic aciduria

Eur J Pediatr. 1995 Oct;154(10):850-2. doi: 10.1007/BF01959796.

Abstract

Growth hormone (GH) insufficiency was demonstrated in a patient with methylmalonic aciduria. GH administration at 14-21 U/m2 per week accelerated linear growth, stimulated lipolysis, and produced clinical improvement and reduced urinary methylmalonate excretion. The clinical and metabolic benefits were lost as the dose of GH was increased to 28 U/m2 per week. CONCLUSION. The use of GH in other patients with methylmalonic aciduria (and other disorders of intermediary metabolism demonstrating similar clinical and metabolic features) warrants further study.

Publication types

  • Case Reports

MeSH terms

  • Amino Acid Metabolism, Inborn Errors / enzymology
  • Amino Acid Metabolism, Inborn Errors / therapy*
  • Body Height / drug effects
  • Child
  • Female
  • Follow-Up Studies
  • Growth Hormone / deficiency
  • Growth Hormone / therapeutic use*
  • Humans
  • Lipid Mobilization / drug effects
  • Methylmalonic Acid / urine*
  • Methylmalonyl-CoA Mutase / deficiency
  • Recombinant Proteins / therapeutic use

Substances

  • Recombinant Proteins
  • Methylmalonic Acid
  • Growth Hormone
  • Methylmalonyl-CoA Mutase