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01-07-2019 | Magnetic Resonance Imaging | Original Article

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

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

Published in: Neurological Sciences | Issue 7/2019

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

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Title: Efficacy and safety of deferiprone for the treatment of superficial siderosis: results from a long-term observational study
Authors: Giovanni Cossu
Giovanni Abbruzzese
Gian Luca Forni
Gildo Matta
Valeria Pinto
Uberto Ruffinengo
Valentina Oppo
Roberta Marchese
Publication date: 01-07-2019
Publisher: Springer International Publishing
Keywords: Magnetic Resonance Imaging
Magnetic Resonance Imaging
Published in: Neurological Sciences / Issue 7/2019
Print ISSN: 1590-1874
Electronic ISSN: 1590-3478
DOI: https://doi.org/10.1007/s10072-019-03847-x

At a glance: The STEP trials

Springer Medicine

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

Abstract

Background

Methods

Results

Conclusions

Clinicaltrials.gov identifier

At a glance: The STEP trials

Springer Medicine

Abstract

Background

Methods

Results

Conclusions

Clinicaltrials.​gov identifier

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