Use of desferrioxamine in the treatment of aceruloplasminemia

Ann Neurol. 1997 Mar;41(3):404-7. doi: 10.1002/ana.410410318.


Aceruloplasminemia is a newly recognized autosomal recessive disorder of iron metabolism resulting in neurodegeneration of the retina and basal ganglia. We report here on the treatment of a patient who developed progressive extrapyramidal symptoms that included blepharospasm, grimacing, and rigidity associated with increased iron deposition in the brain and visceral organs. Treatment for 10 months with the iron chelator desferrioxamine decreased brain iron stores, prevented progression of the neurological symptoms, and reduced plasma lipid peroxidation. These data suggest that early treatment with this chelator may be useful in such patients to diminish central nervous system iron accumulation and to prevent or ameliorate neurological symptoms associated with neurodegeneration.

Publication types

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

MeSH terms

  • Ceruloplasmin / deficiency*
  • Deferoxamine / therapeutic use*
  • Diabetes Mellitus, Type 2 / etiology
  • Female
  • Humans
  • Iron / metabolism
  • Iron Metabolism Disorders / complications
  • Iron Metabolism Disorders / drug therapy*
  • Iron Metabolism Disorders / metabolism
  • Liver / metabolism
  • Middle Aged
  • Siderophores / therapeutic use*


  • Siderophores
  • Iron
  • Ceruloplasmin
  • Deferoxamine