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Neurological Sciences

, Volume 40, Issue 7, pp 1357–1361 | Cite as

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

  • Giovanni CossuEmail author
  • Giovanni Abbruzzese
  • Gian Luca Forni
  • Gildo Matta
  • Valeria Pinto
  • Uberto Ruffinengo
  • Valentina Oppo
  • Roberta Marchese
Original Article

Abstract

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

Keywords

Superficial siderosis Iron accumulation Deferiprone Magnetic resonance imaging 

Notes

Compliance with ethical standards

The trial was approved by the E.O. Ospedali Galliera Ethics Committee, and all participants gave written informed consent before entering the study (Clinicaltrials.gov identifier: NTC00907283).

Conflict of interest

The authors declare that they have no conflict of interest.

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Copyright information

© Fondazione Società Italiana di Neurologia 2019

Authors and Affiliations

  1. 1.Neurology Service and Stroke Unit, Department of NeuroscienceAO BrotzuCagliariItaly
  2. 2.Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics and Maternal and Child HealthUniversity of GenovaGenoaItaly
  3. 3.IRCCS Ospedale Policlinico San MartinoGenoaItaly
  4. 4.Center for Congenital Anemias and Iron DysmetabolismGalliera HospitalGenoaItaly
  5. 5.Department of RadiologyG. Brotzu General HospitalCagliariItaly
  6. 6.Neuroradiology UnitGalliera HospitalGenoaItaly

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