Experience on therapy of adrenoleukodystrophy and adrenomyeloneuropathy

Dev Neurosci. 1991;13(4-5):274-9. doi: 10.1159/000112173.

Abstract

We report the biochemical and clinical results obtained during a dietary erucic acid (C22:1) therapy in 20 patients affected by X-linked adrenoleukodystrophy (ALD). Six patients were very severely affected, 9 had milder neurological symptoms and 5 were presymptomatic. Mean basal levels of plasma C26:0 were 1.41 +/- 0.48 micrograms/ml in ALD patients (control values: 0.33 +/- 0.12). In all patients C26:0 decreased to virtually normal values. In spite of good biochemical response and absence of consistent side effects of therapy, no encouraging data were observed during the clinical follow-up. The presymptomatic subjects were still free of symptoms after more than 1 year of therapy. The symptomatic patients, however worsened or did not show any improvement.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adrenoleukodystrophy / diet therapy*
  • Adrenoleukodystrophy / metabolism
  • Adult
  • Child
  • Coenzyme A Ligases / deficiency
  • Coenzyme A Ligases / metabolism
  • Dietary Fats / administration & dosage
  • Dietary Fats / therapeutic use*
  • Diseases in Twins
  • Erucic Acids / therapeutic use*
  • Fatty Acids / metabolism*
  • Follow-Up Studies
  • Humans
  • Male
  • Microbodies / enzymology
  • Twins, Dizygotic

Substances

  • Dietary Fats
  • Erucic Acids
  • Fatty Acids
  • erucic acid
  • Coenzyme A Ligases
  • lignoceroyl-CoA ligase