Pilot safety trial of deferiprone in 10 subjects with superficial siderosis

Michael Levy, Rafael Llinas

Research output: Contribution to journalArticlepeer-review

45 Scopus citations


Background and Purpose-: Superficial siderosis is a neurodegenerative disease caused by toxic accumulation of hemosiderin on the surface of the brain and spinal cord for which there is no known effective treatment. Methods-: Oral deferiprone, a lipid-soluble iron chelator with ability to cross the blood-brain barrier, at a dose of 30 mg/kg per day was tested for safety in an open pilot study in 10 subjects with superficial siderosis. Results-: Over a 90-day period, deferiprone had no significant adverse effects on hematologic, liver, or neurological function. Ad hoc MRI assessments of the brain indicated a reduction in hemosiderin deposition in some subjects. Conclusions-: Deferiprone proved safe in this small population of superficial siderosis subjects. There was MRI evidence of reduced hemosiderin deposition with deferiprone. Prospectively designed efficacy studies are necessary to determine the clinical efficacy of deferiprone in superficial siderosis.

Original languageEnglish (US)
Pages (from-to)120-124
Number of pages5
Issue number1
StatePublished - Jan 2012


  • deferiprone
  • hemosiderosis
  • superficial siderosis

ASJC Scopus subject areas

  • Clinical Neurology
  • Cardiology and Cardiovascular Medicine
  • Advanced and Specialized Nursing


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