Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study

Giovanni Cossu, Giovanni Abbruzzese, Gian Luca Forni, Gildo Matta, Valeria Pinto, Uberto Ruffinengo, Valentina Oppo, Roberta Marchese

Research output: Contribution to journalArticle

Abstract

© 2019, Fondazione Società Italiana di Neurologia. Background: Superficial siderosis (SS) of the central nervous system is a rare and heterogeneous condition due to deposition of hemosiderin on the surface of the brain and spinal cord. The usually progressive clinical course is characterized by a combination of hearing loss, cerebellar ataxia, and myelopathy. There is no known treatment for SS, but the iron chelator deferiprone (DFP) has been proposed as a potentially useful treatment. Methods: We present a long-term (average 3.7 years) evaluation of four cases of SS treated with DFP (15 mg/kg po bid). Results: Treatment with DFP proved safe and well tolerated. Two out of the four subjects were unchanged while the other two presented a clinical improvement with reduction of postural instability and cerebellar signs. Blinded evaluation of magnetic resonance imaging (performed every 6 months during follow-up) showed a reduction of the abnormal iron deposition for all patients. Conclusions: This long-term observational study suggests that DFP may be effective in the management of the neurological manifestations associated with iron accumulation in SS. Clinicaltrials.gov identifier: NTC00907283.
Original languageEnglish
Pages (from-to)1357-1361
Number of pages5
JournalNeurological Sciences
Volume40
Issue number7
DOIs
Publication statusPublished - Jul 1 2019

Keywords

  • Deferiprone
  • Iron accumulation
  • Magnetic resonance imaging
  • Superficial siderosis

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