Progression despite replacement of a myopathic form of coenzyme Q10 defect

Neurology. 2004 Aug 24;63(4):727-9. doi: 10.1212/01.wnl.0000134607.76780.b2.

Abstract

The authors report 7 years of follow-up evaluation of a patient with coenzyme Q10 (CoQ10) deficiency. Initial symptoms of exercise intolerance and hyperlactatemia improved markedly with substitutive treatment. However, CoQ(10) supplementation did not prevent the onset of a cerebellar syndrome. A switch to idebenone treatment resulted in clinical and metabolic worsening, which disappeared with subsequent CoQ10 treatment. CoQ10 defects may cause progressive neurologic disease despite supplementation.

Publication types

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

MeSH terms

  • Benzoquinones / adverse effects
  • Benzoquinones / therapeutic use
  • Carnitine / therapeutic use
  • Cerebellar Ataxia / genetics*
  • Cerebellum / pathology
  • Child, Preschool
  • Disease Progression
  • Drug Therapy, Combination
  • Exercise Tolerance / drug effects
  • Exercise Tolerance / genetics*
  • Female
  • Follow-Up Studies
  • Humans
  • Lactates / blood*
  • Magnetic Resonance Imaging
  • Mitochondria, Muscle / chemistry
  • Mitochondrial Myopathies / complications
  • Mitochondrial Myopathies / drug therapy
  • Mitochondrial Myopathies / genetics*
  • Mitochondrial Myopathies / metabolism
  • Muscle, Skeletal / pathology
  • Treatment Failure
  • Ubiquinone / deficiency*
  • Ubiquinone / pharmacokinetics
  • Ubiquinone / therapeutic use
  • Vomiting / etiology

Substances

  • Benzoquinones
  • Lactates
  • Ubiquinone
  • idebenone
  • Carnitine